Sanofi Ventures

Leveraging personal data for custom-fit diagnosis and treatment

Precision Medicine

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 Nucleai is an AI-powered drug discovery platform that leverages spatial biology mapping technology and pathology data to predict patient response to cancer treatments and support precision medicine drug development. The firm's software uses computer vision and machine learning algorithms to predict cancerous tumor progression and immune system response in order to determine the most effective course of treatment. The model replicates a patient's immune system and tumor characteristics by analyzing large datasets of tissue images. The technology is used to discover novel therapies, support clinical trials, stratify patients, and provide therapeutic insights.
 
 
 Key customers and partnerships
 
 
 Nucleai collaborates with pharmaceutical companies, licensing its research platform for internal use and developing biomarkers. It has partnered with 10 pharmaceutical companies, including Merck and Debiopharm Group, on various phase 2 and 3 clinical trials for treating non-small cell lung, breast, and cervical cancer as of February 2022. In July 2022, the firm partnered with multiomics analytical service provider Sirona Dx to identify new spatial biomarkers in solid tumors. Sirona Dx will provide Nucleai and its customers with a contract research organization partner in order to launch its diagnostic algorithm for use in biopharma clinical trials.&nbsp;
 
 
 Funding and Financials
 
 
 The firm&rsquo;s latest funding took place in March 2022, when it raised USD 33 million in a Series B funding round led by Section 32 and Sanofi Ventures. A portion of the funds was allocated for the further development of the company's spatial biology analysis platform. In addition, the funds were earmarked for the expansion of its commercial footprint among biopharma companies and contract research organizations.
 
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UK-based biotechnology startup OMass Therapeutics offers OdyssION, a proprietary drug discovery platform based on breakthrough research in native mass spectrometry. Mass spectrometry was traditionally used to identify a chemical by measuring its component masses and relative concentrations. OMass’ technology platform aims to provide a high-resolution view of the potential impact of experimental drugs on a protein target, taking into account the way drugs interact with proteins in their native environment. This allows users to identify the drugs that will bind to protein targets and those that will get rejected.Using this technology, OMass is developing a pipeline of small-molecule therapeutics focused on treating difficult-to-drug proteins that are linked to rare diseases and immunological conditions. Its portfolio consists of five novel drug programs in preclinical development for treating endocrine disorders, inflammatory diseases, and inflammatory bowel disease (IBD) as well as two early-stage programs targeting epilepsy and lupus.I

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 Matchpoint Therapeutics is a biotechnology company that focuses on the development of small molecule drugs for the treatment of immune diseases. The firm utilizes its proprietary Advanced Covalent Exploration (ACE) platform, which combines chemoproteomics, machine learning algorithms, and covalent chemistry. This platform allows for the identification of disease-causing proteins and the development of targeted medicines.
 
 
 
 
 The company prioritizes genetically or clinically validated targets that are best suited for therapeutic intervention through covalent chemistry. The use of covalent chemistry enables enhanced potency, selectivity, and reduced systemic exposure in the developed treatments. While the company had undisclosed drug candidates in the discovery stage as at May 2023, it was also planning to establish a strategic collaboration aimed at advancing its internal pipeline while also exploring new programs through partnerships.
 
 
 Funding and financials
 
 
 The company launched out of stealth in October 2022 and raised USD 70 million in a 
 <a href="https://sp-edge.com/updates/13810">
 Series A
 </a>
 funding round led by Sanofi Ventures. The new funds were used to advance the company&rsquo;s proprietary Advanced Covelent Exploration platform and support the development of the company&rsquo;s undisclosed drug candidates. 
 
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 Avilar Therapeutics is a biopharmaceutical company focused on the discovery and development of drugs using a precision medicine approach by targeting protein degraders called ATACs (Asialoglycoprotein receptor Targeting Chimeras) to selectively shuttle disease-causing proteins. The company uses its proprietary discovery platform to design and synthesize ATAC in the drug development process. The drug discovery platform comprises key components such as ASGPR ligand chemistries, modular assembly techniques, extracellular degradation modeling, and proteome mapping.&nbsp;
 
 
 Furthermore, the company&rsquo;s pipeline of ATAC-targeting drugs is focused on treating autoimmune, neurological, and other diseases.&nbsp;
 
 
 Funding and Financials
 
 
 In February 2023, the company raised USD 15 million via its 
 <a href="https://sp-edge.com/updates/16489">
 seed funding 
 </a>
 round from new investors including Sanofi Ventures. The funds were used toward advancing its pipeline drug candidates targeting extracellular proteins and expanding its proprietary technologies.
 
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 QurAlis is a biotech company that develops precision medicine for amyotrophic lateral sclerosis (ALS) and related neurodegenerative diseases. The creators of QurAlis are a group of neurodegenerative biologists from Harvard University and Harvard Medical School. The company has two proprietary platforms: 1) QR43 Platform which supports the discovery and development of TDP-43 diseases and 2) QurAlis FlexASO Splice Modulator Platform to support discovery and development of different disease targets. In July 2023, the company released data that claimed Antisense oligonucleotides (ASO) generated from this platform is 3x better in terms of the potency and biodistribution of standard ASOs.&nbsp;
 
 
 As of July 2023, the company had four drugs in development: 1) QRL-201 aimed at restoring the production of the STATHMIN-2 (STMN2) protein in individuals with ALS and was
 <a href="https://sp-edge.com/updates/19148">
 in Phase I clinical trials
 </a>
 , 2) QRL-101 aimed at addressing the progression of hyperexcitability-related diseases in ALS patients and was in Phase I clinical trials, 3) QRL-203 also targeting the STMN2 protein and was in the preclinical stage, and 4) QRL -204 targeting the UNC13A gene which was also in the preclinical stage. Furthermore, in July 2020, the company obtained a license from Eli Lilly and Company for a preclinical small-molecule compound to treat ALS.&nbsp;
 
 
 Key Customers and Partnerships
 
 
 The company partnered with digital twin company Unlearn in June 2023 to collaborate on clinical-stage drugs with Unlearn&rsquo;s generative AI technology.&nbsp;
 
 
 Funding and Financials
 
 
 In March 2023, the company raised USD 88 million in an oversubscribed 
 <a href="https://sp-edge.com/updates/17009">
 Series B 
 </a>
 funding round led by EQT Life Sciences. The new funds were used to expedite the clinical development of QRL-201 and QRL-101 and to support the company's research projects and advance the development of new therapies targeting specific components of ALS and related frontotemporal dementia (FTD) pathology.
 
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Sudo Biosciences is a biopharmaceutical company developing drug candidates targeting the tyrosine kinase 2 (TYK2) inhibitor which plays a crucial role in cytokine signaling pathways associated with various immune-mediated inflammatory conditions. The company's pipeline of next-generation TYK2 inhibitors features a promising brain-penetrant candidate, potentially the first and best-in-class, for treating multiple sclerosis and neurodegenerative diseases with underlying neuroinflammation. Additionally, it includes a potential first and best-in-class topical candidate for immune-mediated dermatologic diseases. As of June 2024, the company had 4 drug candidates in preclinical stages in its pipeline.

New technologies are opening up mental health services to previously overbooked and underserved populations.

Mental Health Tech

Click Therapeutics develops software that provides prescription digital therapeutics (PDTs) for individuals diagnosed with mental health and chronic conditions. It's only nonprescription therapeutic, a smoking cessation product called Ckickotine, is commercially available and clinically validated despite not requiring FDA approval.&nbsp;The company leverages adaptive data science and machine learning algorithms to personalize content and ensure user engagement. Click’s programs attempt to change individual behavior through cognitive and neurobehavioral mechanisms and can be used independently or in conjunction with biomedical treatments. Users can download the app after being prescribed by a physician and activate it using a pharmacist-issued activation code. The treatments (missions for users) are usually between eight and 12 weeks long, and patients can interact with physicians during or after completing therapy.&nbsp;In March 2024, the company, in collaboration with Otsuka Pharmaceutical, launched Rejoyn, the first FDA-cleared prescription digital therapeutic for the adjunctive treatment of major depressive disorder (MDD) symptoms in adults aged 22 and older who are on antidepressant medication. Rejoyn utilizes cognitive emotional brain training exercises and cognitive behavioral therapy to help reduce depression symptoms.Additionally, as of September 2024, the company had several PDTs in the pipeline across a variety of therapeutic areas that were still in the development stage, including insomnia, schizophrenia, migraines, acute coronary syndrome, overactive bladder, obesity, and chronic lower back pain. The company's pipeline includes CT-155, a PDT for treating negative symptoms of schizophrenia, which received FDA Breakthrough Device designation in January 2024. Click is also developing CT-132, a PDT for episodic migraine prevention that received FDA Breakthrough Device designation in December 2022.&nbsp;In <a href="https://sp-edge.com/updates/29687">May 2024</a>, Click acquired assets from Better Therapeutics, including AspyreRx, an FDA-authorized PDT for type two diabetes, to accelerate its development initiatives in obesity and cardiometabolic diseases.Key customers and partnershipsClick partnered with Magellan Health for clinical coaching and pharmacy benefits management in 2016. Additionally, the company has collaborated with Otsuka (December 2018) in a deal worth USD 302 million to develop and commercialize a PDT for the treatment of MDD, as well as a partnership with Boehringer Ingelheim (September 2020) to develop and commercialize a PDT to aid in the treatment of schizophrenia.Furthermore, in September 2023, Click initiated a partnership with Indivior to develop a prescription digital therapeutic for opioid use disorder.&nbsp;

Artificial intelligence is identifying and testing new therapies faster than ever.

AI Drug Discovery

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 Nucleai is an AI-powered drug discovery platform that leverages spatial biology mapping technology and pathology data to predict patient response to cancer treatments and support precision medicine drug development. The company&rsquo;s ATOM platform uses computer vision and machine learning algorithms to predict cancerous tumor progression and immune system response in order to determine the most effective course of treatment. The model replicates a patient's immune system and tumor characteristics by analyzing large datasets of tissue images.&nbsp;
 
 
 The technology is used to discover novel therapies, support clinical trials, stratify patients, and provide therapeutic insights. Further it has access to millions of annotated cells across 12 cancer indications. 
 
 
 Key customers and partnerships
 
 
 Nucleai collaborates with pharmaceutical companies, licensing its research platform for internal use and developing biomarkers. It has partnered with 10 pharmaceutical companies, including Merck and Debiopharm Group, on various Phase II and III clinical trials for treating non-small cell lung, breast, and cervical cancer as of February 2022.
 
 
 As of March 2024, the company has over 50 data partners. In July 2022, the firm partnered with Sirona Dx to identify new spatial biomarkers in solid tumors. Sirona Dx will provide Nucleai and its customers with a contract research organization partner in order to launch its diagnostic algorithm for use in biopharma clinical trials. Further, in 
 <a href="https://www.businesswire.com/news/home/20230604005025/en">
 June 2023
 </a>
 , it partnered with Mayo Clinic to focus on several distinct offerings to support drug development.&nbsp;
 
 
 Funding and financials
 
 
 In 
 <a href="https://sp-edge.com/updates/28170">
 April 2024
 </a>
 , the company raised USD 14 million in a funding round led by M Ventures, the venture capital arm of Merck, with participation from Section 32, Sanofi Ventures, and others. The funds were allocated to further implement Nucleai's AI algorithms in prospective patient enlistment for clinical trials. 
 
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Membership-based, non-insurance healthcare companies helping maintain a steady state of well being.

Preventive Healthcare

Omada is a prevention-focused digital therapeutics startup that helps members achieve health goals through lifestyle changes. It offers four evidence-based, virtual prevention programs for managing hypertension, musculoskeletal (MSK) issues, prediabetes, and diabetes.Omada offers real-time data tracking through connected devices (such as wireless scales, blood pressure, and glucose monitors) and access to personalized coaching sessions via a mobile app as part of its prevention program. It also provides an online community of peers with similar health conditions to discuss challenges and provide support. In May 2021, Omada became the first fully-virtual healthcare provider to earn the National Committee for Quality Assurance’s Population Health Program Accreditation for its type 2 diabetes and type 2 diabetes + hypertension programs.Unlike its peers, Omada operates a performance-based business model where it charges clients an enrollment fee based on the number of users and a performance fee based on how much weight their employees lose. In May 2020, the company acquired virtual physical therapy startup Physera.Key customers and partnershipsAs of May 2024, Omada had more than 1 million members from more than 1,900 enterprise clients across all 50 US states. Its B2B employer and health plan client base include key clients such as American Eagle, Kaiser Permanente, Costco Wholesale, 3M, and Marvin. Additionally, the company has partnered with health systems and hospitals such as Intermountain Healthcare to provide its preventive programs as a covered benefit.In <a href="https://sp-edge.com/updates/15587">January 2023</a>, the company partnered with health service company Castell (a subsidiary of Intermountain Healthcare) to provide its diabetes programs to members from Intermountain Healthcare’s primary care providers in its Utah Medical Group. Further in <a href="https://sp-edge.com/updates/25245">January 2024</a>, the company partnered with Amazon to expand access to its cardiometabolic program.

More important now than ever, telehealth will long outlast the pandemic that drove its growth.

Telehealth

Atropos Health provides its clients with insights and reports into clinical queries based on anonymized patient records. The company refers to this as their “prognostogram informatics consult”, which has a 24-hour turnaround time. The company is working on a similar consult service for oncology where treatment pathways will also be suggested based on previous patient data. Its main clients are physicians.&nbsp;In <a href="https://www.atroposhealth.com/blog/atropos-health-launches-new-geneva-os-and-chatrwd-application-for-rapid-real-world-evidence-with-generative-ai">October 2023</a>, Atropos Health launched the Geneva OS (Generative Evidence Acceleration Operating System) and ChatRWD (Real World Data) application, aimed at helping healthcare and life sciences leaders advance evidence generation. The new offering allows non-technical users to generate publication grade observational research studies using real world data from the Atropos Evidence Network. The company claimed that the new offering can expedite decision-making, generate evidence in minutes instead of weeks to months, and result in cost savings while not affecting clinical outcomes.Key customers and partnershipsIn <a href="https://www.prweb.com/releases/atropos-health-partners-with-norstella-to-expand-the-atropos-evidence-network-and-structured-clinical-trial-intelligence-302173899.html">June 2024</a>, the company partnered with Norstella, an intelligence solution provider, to expand the Atropos Evidence Network. Through this partnership, Norstella’s platform was used to expand the adoption of Atropos Health's GenAI platform GENEVA OS.In <a href="https://www.mobihealthnews.com/news/data-companies-atropos-health-arcadia-announce-partnership#:~:text=Through%20the%20partnership%2C%20Arcadia%20joins,conduct%20real%2Dworld%20evidence%20studies.">January 2024</a> the company partnered with healthcare data platform Arcadia to integrate Atropos' evidence generation capability into Arcadia's data platform, enabling value-based healthcare services and decision-making. The partnership allows Arcadia’s clients to apply Atropos’s suite of tools, which turn medical data into individual evidence supporting comprehensive patient care and performance in value-based care models. Furthermore, the collaboration will facilitate healthcare providers in creating relevant data-driven evidence to optimize care pathways and clinical guidelines and choose the best interventions for value-based care.Also in <a href="https://www.atroposhealth.com/blog/atropos-health-leverages-google-cloud">January 2024</a>, the company partnered with Google Cloud to accelerate real-world evidence generation for healthcare systems. Through the partnership, Atropos’ GENEVA OS platform was integrated with Google Cloud's Healthcare Data Engine (HDE) and BigQuery. This enabled company's using Atropos' services to simplify access to data, streamline analytics, and democratize insights.The company also partnered with SEQESTER in <a href="https://www.businesswire.com/news/home/20231214292956/en/Atropos-Health-and-SEQSTER-Partner-to-Accelerate-Patient-Registries-in-Clinical-Research-Saving-Health-Systems-Millions-and-Accelerating-Evidence-Generation">December 2023</a> to save time, money, and accelerate drug development. Through the partnership, SEQESTER incorporated Atropos' solution to its own, allowing SEQESTER to deliver real-time and real-world data layered in AI to accelerate drug discovery.In <a href="https://www.prnewswire.com/news-releases/atropos-health-and-datavant-partner-to-bring-connected-fit-for-purpose-real-world-evidence-to-the-point-of-care-301857602.html">June 2023</a>, the company partnered with healthcare data ecosystem provider Datavant to exchange patient-level data. Through the partnership, Atropos Health users gained access to Datavant’s services, while Datavant’s ecosystem gained access to the Atropos Evidence Platform. The partnership also enabled Atropos Health to grade data present in Datavant’s ecosystem to assign a “Real World Data Score,” based on the quality of data with regard to size, completeness, longitudinality, and level of standardization and a “Real World Fitness” score, based on the data’s real-world applications. Additionally, the partnership allowed for the conversion of Datavant’s ecosystem data into real-world evidence through Atropos Health’s “Green Button Informatics Consult Service,” which creates publication-grade analytics in 48 hours.

Reinventing gene therapies with next-generation technologies

Human Gene Editing

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 Based in France, Eligo Bioscience develops novel microbiome gene editing therapies across various therapeutic areas including autoimmunology, dermatology, and oncology using the CRISPR technology including base editing. The company&rsquo;s approach entails editing microbiome gene ​​repertoire to add or remove genes to support disease treatment. For this, it designs small biotherapeutics dubbed eligobiotics, made out of DNA and proteins to target specific parts of the bacteria.
 
 
 The company has not revealed its complete pipeline of therapeutic programs. It leverages its three proprietary eligobiotics platforms: 1) Sequence-Specific Anti-Microbials (SSAM) to deliver CRISPR-Cas9 nucleases and the corresponding guide RNA into the genome, which will only eliminate the unfavorable parts of the bacteria and leave the favorable ones unaffected; 2) Gene-Editing of the Microbiome (GEM) platform to deliver base editors into the bacteria to modify the genome, without destroying the bacteria; and 3) Function Addition to the MicrobiomE (FAME) to deliver a therapeutic gene into the microbiome to trigger existing microbial populations to stimulate a therapeutic impact in the body.
 
 
 Key customers and partnerships
 
 
 Eligo entered into a USD 224 million partnership with 
 <a href="https://www.nutraingredients.com/Article/2021/01/19/GSK-Eligo-Bioscience-targets-skin-microbiome-in-185m-acne-deal">
 GlaxoSmithKline 
 </a>
 in January 2021 to leverage its eligobiotics technology to prevent acne vulgaris, a skin condition, and received funding for its acne program EB0005. Both parties also planned to enter into a licensing and collaboration agreement.&nbsp;
 
 
 In June 2020, Eligo Bioscience secured USD 7.1 million investment&mdash;including milestone payments&mdash;from 
 <a href="https://carb-x.org/carb-x-news/carb-x-funds-eligo-bioscience-to-develop-crispr-based-therapeutics-derived-from-bacteriophages-to-kill-superbugs-and-prevent-deadly-infections-in-transplant-patients/">
 CARB-X
 </a>
 , a global non-profit organization, to develop highly specific antimicrobials to prevent multi-drug-resistant bacterial infections in organ transplant patients.&nbsp;
 
 
 Funding and financials
 
 
 In December 2023, Eligo Bioscience raised USD 30 million in a Series B funding round led by Sanofi Ventures, with participation from new investor Bpifrance (through its InnoBio 2 fund) and existing investors Khosla Ventures and Seventure Partners. The proceeds were set aside to accelerate the development of Eligo&rsquo;s primary program, EB005, designed for acute acne and to facilitate pre-Investigational New Drug (IND) and IND activities to achieve early human data readouts in a Phase Ib/IIa clinical trial. The funding was also set aside to support Eligo&rsquo;s expansion into other chronic diseases, including oncology. 
 
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Pushing the envelope in MedTech with emerging technologies

Next-gen Medical Devices

i2o Therapeutics is a biotechnology company focused on developing oral formulations of biologic and peptide-based drugs using its proprietary ionic liquid platform technology. Founded in 2019 as a spinout from Harvard University, i2o's platform aims to enable oral delivery of therapeutics ranging from peptides to monoclonal antibodies that typically require injection. The company's technology uses ionic liquids - essentially liquid salts - to protect drug molecules from the harsh environment of the digestive system and help them cross the epithelial lining of the gastrointestinal tract.
i2o's lead program is an oral GLP-1 agonist for diabetes and obesity that is in preclinical development. The company has five other preclinical programs targeting type two diabetes, obesity, and non-alcoholic steatohepatitis. In August 2023, i2o acquired assets from Intarcia Therapeutics, including ITCA 650, an investigational twice-yearly implantable GLP-1 agonist for type two diabetes that is scheduled for an FDA advisory committee review. The company also acquired Intarcia's Medici implant technology platform and other pipeline assets in the cardiometabolic space.
i2o has raised significant funding to advance its programs, including a USD 4 million seed round in 2020 and a USD 46 million Series A financing in 2023. The company is led by Kurt Graves, who was appointed Chairman, President and CEO in August 2023, succeeding co-founder Ravi Srinivasan.
Key customers and partnerships
i2o has established collaborations with major pharmaceutical companies to explore applications of its ionic liquid technology. In 2021, the company announced a partnership with Sanofi to enable oral delivery of nanobody-based medicines across multiple therapeutic areas. In January 2022, i2o entered into a research collaboration and option agreement with Janssen biotech to evaluate the use of ionic liquid technology for oral delivery of macromolecules. Additionally, i2o has a 4-year sponsored research and licensing agreement with the Mitragotri Lab at Harvard University to further advance ionic liquid applications and strengthen its intellectual property position.

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Bluebird Bio is a gene therapy provider that is developing therapeutics to treat conditions associated with cell disease, genetic diseases, and neurological diseases. Specifically, the company focuses on developing therapies for thalassemia, cerebral adrenoleukodystrophy (CALD), and sickle cell disease. Bluebird develops lentiviral vectors (LVVs) designed to address the causes of specific diseases and are delivered as single-dose treatments to patients. The company is listed on the Nasdaq and is publicly traded under the ticker symbol BLUE, and claims to have the largest ex-vivo gene therapy data set in the world. 2seventy bio was spun out of Bluebird Bio in November 2021 and functions as an oncology cell therapy company.The company has three commercialized therapeutics: SKYSONA for the treatment of CALD in patients less than 18 years, ZYNTEGLO for the treatment of thalassemia in adults, and LYFGENIA for the treatment of sickle cell disease. As of April 2024, the company had two candidates for the treatment of sickle cell disease, SCD (HGB-210) and SCD (HGB-206), which were in Phase III clinical trials and Phase I/II clinical trials, respectively. Key customers and partnershipsBluebird Bio partnered with biotechnology company Magenta Therapeutics in December 2020 to create a research collaboration to carry out a Phase II clinical proof-of-concept study for Magenta’s novel mobilization regimen, MGTA-145, for mobilization and collection of stem cells in patients with sickle cell disease (SCD).Bluebird Bio also partnered with biotechnology company National Resilience, Inc in July 2021 to accelerate research, development, and delivery of cell therapies. Through the partnership, National Resilience acquired Bluebird Bio’s Research Triangle manufacturing facility in North Carolina, along with more than 100 technical staff. Resilience was expected to continue to support vector supply for both Bluebird Bio and 2seventy bio.

Redefining hospital care with the comfort of home

Hospital-at-Home

Medisafe is a digital health platform focused on medication management and adherence. Founded in 2012, the company offers a smartphone app that helps patients track and manage their medication schedules. The Medisafe app provides features such as medication reminders, drug interaction warnings, and health measurement tracking. It allows users to set up multiple profiles to manage medications for family members as well.
Medisafe utilizes artificial intelligence and behavioral science principles to deliver personalized interventions aimed at improving medication adherence. The platform offers Just-In-Time-Interventions technology to provide targeted support when patients are most likely to skip doses. In addition to the consumer app, Medisafe provides digital solutions for pharmaceutical companies and healthcare providers to support patient engagement throughout the treatment journey.
The company's technology has been validated through clinical studies. A randomized control trial published in February 2024 found that Medisafe's app significantly increased medication adherence and self-efficacy among medically underserved populations with chronic illnesses. As of September 2022, Medisafe reported over 10 million app installs worldwide and over 7 billion doses managed through its platform. The app is available in over 15 languages and has received high ratings on both iOS and Android app stores.

RNA Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company developing mRNA and gene correction therapeutics using its proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform. The company's technology enables precise delivery of genetic medicines to targeted organs and cells beyond the liver, potentially improving efficacy and potency. ReCode's lead programs focus on primary ciliary dyskinesia (PCD) and cystic fibrosis (CF).RCT1100, ReCode's lead candidate for PCD caused by DNAI1 gene mutations, is an inhaled mRNA therapy designed to restore proper ciliary function. In January 2024, ReCode dosed the first patient in a Phase 1 clinical trial of RCT1100. The company is also developing RCT2100 for CF patients with Class I CFTR mutations who do not respond to current modulators, representing 10-13% of CF patients. Both therapies utilize ReCode's SORT LNP platform for targeted delivery.preclinical data presented in 2022 showed that ReCode's CFTR mRNA encapsulated in SORT LNPs could be nebulized, delivered as an aerosol, and rescue CFTR function in cells from CF patients to levels comparable with approved modulators. In June 2022, ReCode published research in Science demonstrating its SORT LNPs could facilitate high levels of persistent gene editing in lung stem cells when given intravenously, achieving over 70% editing efficiency sustained for up to 660 days in mice.Key customers and partnershipsIn January 2023, ReCode announced a strategic investment of up to USD 15 million from the Cystic Fibrosis Foundation to support development of its inhaled mRNA-based CF therapeutic. The investment includes USD 10 million upfront to fund preclinical studies and early clinical development, with an additional USD 5 million upon meeting milestones.ReCode entered a multi-year research collaboration with Asklepios BioPharmaceutical (AskBio) in January 2023 to develop a novel platform for full gene insertion using single vector delivery of gene editing and DNA cargoes. The partnership aims to combine AskBio's synthetic DNA and gene editing expertise with ReCode's SORT LNP technology to enable precision delivery of both gene editing tools and DNA in a single LNP to desired targets.

Therapeutics that target specific areas/tissues to minimize treatment side effects.

Tissue Targeting Therapeutics

NodThera is a clinical-stage biotechnology company developing a new class of oral, small molecule NLRP3 inflammasome inhibitors to treat diseases driven by chronic inflammation. The company's lead candidate, NT-0796, has demonstrated positive results in Phase 1 studies, showing blood-brain barrier penetration and reduced inflammatory biomarkers. NT-0796 is designed as an orally bioavailable, brain-penetrant NLRP3 inhibitor, with potential applications in neurological and neurodegenerative diseases. In July 2023, NodThera reported initial data from a Phase Ib/IIa study of NT-0796 in elderly volunteers, showing rapid decreases in neuroinflammatory biomarkers in cerebrospinal fluid after just seven days of treatment.
The company's second clinical compound, NT-0249, is a peripherally restricted NLRP3 inflammasome inhibitor that has completed Phase 1 single ascending dose studies. NT-0249 demonstrated a pharmacokinetic profile consistent with once-daily dosing and showed significant anti-inflammatory effects in healthy volunteers. NodThera's third candidate, NT-0527, is a novel brain-penetrant NLRP3 inflammasome inhibitor advancing through IND-enabling studies.
NodThera's approach focuses on targeting chronic low-grade inflammation through selective modulation of the NLRP3 inflammasome, which plays a pivotal role in controlling inflammatory diseases. The company's strategy involves designing highly differentiated and brain-penetrant molecules, combining a deep understanding of NLRP3 inhibition with pharmaceutical neuroscience expertise and precision molecular design. NodThera's pipeline includes candidates for treating a range of inflammatory conditions, including non-alcoholic steatohepatitis, lung fibrosis, neurodegenerative diseases, and inflammatory bowel disease.

Nura Bio is a clinical-stage biopharmaceutical company focused on developing neuroprotective therapies to treat neurological diseases. Nura Bio’s drug candidate, NB-4746, is designed to inhibit the SARM1 protein, which plays a significant role in axon degeneration. This drug has shown promise in preclinical studies for preventing nerve damage and is currently advancing toward phase 1b/2 clinical trials in 2025. The company has raised $140 million in financing to support this and other neuroprotective drug programs.

Atropos Health develops an Atropos Evidence Platform that specializes in evidence-based healthcare. Its platform transforms health data into usable real-world evidence and combines electronic medical record (EMR) data to derive digital evidence for individual patients and helps healthcare institutions create value from previously siloed EMR data. Healthcare organizations, life science companies, and researchers leverage the cloud-based platform to close evidence gaps from bench to bedside, elevate clinical outcomes with data-driven care, expedite research, and more.

Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief. Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.

NextPoint Therapeutics is a clinical-stage biotechnology company that develops therapeutics to treat cancer patients. The company offers immunotherapies for a validated checkpoint axis that is independent of PD-1/L1 and modulates both adaptive and innate immune systems to find and destroy cancer cells. It advances therapeutic approaches utilizing the unique upregulation of HHLA2 in cancer as an anchor for tumor-targeting therapeutic modalities.

Sudo Biosciences is a biopharmaceutical company that designs and develops medicines that target the tyrosine kinase 2 (TYK2) pseudokinase domain, a crucial mediator in cytokine signaling pathways that have been linked to a broad range of immune-mediated inflammatory conditions. Its pipeline of next-generation TYK2 inhibitors includes a potential brain-penetrant candidate for treating multiple sclerosis and neurodegenerative diseases with underlying neuroinflammation and a potential topical candidate for immune-mediated dermatologic diseases. It helps patients receive the cure or the treatment of multiple sclerosis and neurodegenerative diseases with underlying neuroinflammation at a competitive cost.

Eligobiotics is a developer of antibiotics intended to treat microbiome precision and bacteria-associated diseases. The company's antibiotics combine CRISPR/Cas system with engineered phage capsids, to develop antimicrobials that are used to eradicate resistant pathogens or virulent bacteria within the human microbiome, enabling clients to get next-generation antimicrobials to treat diseases caused by superbugs.

T-Therapeutics is a expertise in mouse genome engineering, deep expertise in biopharmaceutical drug development, single cell genomics, machine-learning and structural biology, anchored in a culture of creativity and collaboration. .

Minervax is pursuing the development of a vaccine against Group B streptococcus (GBS) infections by utilizing a particular fusion protein, which has proved to elicit good protective immunity. A vaccine including this fusion protein will likely elicit protective immunity against many clinically relevant GBS strains.

ReCode Therapeutics is a genetic medicines company that specializes in mRNA and gene correction therapeutics. Its selective organ targeting (SORT) lipid nanoparticle (LNP) platform is a genetic medicines technology that enables precise delivery to target organs and cells beyond the liver.

ROME Therapeutics is developing novel therapies for cancer and autoimmune diseases by harnessing the power of the repeatome – vast stretches of uncharted genetic material that have long been dismissed as “junk DNA.” With several drug targets identified and multiple discovery programs underway, ROME is moving rapidly to leverage this new frontier in biology. To lead this exploration, ROME has assembled a team of world-class leaders across fields including oncology, immunology, virology and machine learning.

Abcuro’s mission is to develop immune-modulatory bio-therapeutics for treating both autoimmunity and cancer. They leverage the systematic target validation and clinical insights gained by their founders to identify new targets. Their intensive use of bioinformatics to interrogate the transcriptome of human disease together with ex-vivo validation in diseased tissue has uncovered new approaches to target key compartments of the immune system.

Tisento Therapeutics is a MA-based developer of novel medicines to treat diseases with significant unmet medical needs.

Veralox Therapeutics develops small molecule therapeutics that treat the underlying pathologies of thrombosis and type one diabetes. Based on an understanding of the molecular mechanisms of these diseases, these efforts will lead to new treatment paradigms and better outcomes for patients.

Therini Bio is a biotechnology company specializing in fibrin-targeting therapies to treat inflammatory neurological and retinal diseases. It develops therapeutics based on unique insights into the novel role of fibrin in driving chronic innate immune activation that characterizes a broad set of human diseases with high unmet needs. The current therapeutic target is based on novel findings by the Akassoglou lab at Gladstone/UCSF that implicate fibrin as a key driver of neuroinflammation.

QurAlis is a clinical-stage biotechnology company that is developing precision medicines with genetically validated targets for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Its proprietary platforms and biomarkers allow for the design and development of drugs that target disease-causing genetic alterations directly.

Avilar Therapeutics is a biopharmaceutical company focusing on the discovery and development of extracellular protein degraders, a new frontier in targeted protein degradation.

Escient Pharmaceuticals is a provider of bio-technical research and development services intended to focus on the advancement of first-in-class G Protein-Coupled Receptor targeted drugs. Its services focus on offering therapies for neuro-immuno-inflammatory and autoreactive diseases.

Normunity is a biotechnology company creating a new class of precision immuno-oncology medicines, called immune normalizers, that target novel mechanisms that free the body’s normal immunity against cancer. Based on an ongoing, interactive academic-biotech alliance with the lab of Dr. Lieping Chen at Yale School of Medicine, Normunity is targeting newly-discovered mechanisms of immune disruption in cancer based on proprietary discovery platforms that elucidate the complex interactions between human cancer and the immune system. The company is advancing an emerging pipeline of immune normalizers, including initial drug programs that target mechanisms that drive the exclusion of T-cells from immune-sensitive tumors and other mechanisms that are barriers to normal immune function in cancer. Normunity is located in Boston, Massachusetts and West Haven, Connecticut.

Matchpoint is a biotechnology company harnessing the power of covalency to discover precision covalent medicines to transform the treatment of immune diseases and other serious illnesses. The company's proprietary Advanced Covalent Exploration (ACE) platform integrates advanced chemoproteomics, machine learning, and covalent chemistry library evolution. Matchpoint has an emerging pipeline of novel covalent medicines, initially focused on immunology.

Empatica designs and develops AI-enabled wearables, software, and digital biomarkers, to push the boundaries in health monitoring, forecasting, treatment, and research. Empatica’s platform uses a combination of biosensors to detect unique components of human physiology: providing AI-enabled tools advancing the forecasting, monitoring, research, and treatment of multiple conditions.

OMass Therapeutics is a drug discovery company exploiting novel mass spectrometry platforms to develop therapeutics against challenging disease targets.

Nucleai is a spatial biology company that has developed an AI-powered platform to enhance drug development and improve patient outcomes. Nucleai’s platform leverages unique tissue datasets to produce insights into cancer biology, increasing clinical trial efficacy and improving patient care. Nucleai works with leading pharmaceutical companies, licensing its research platform for internal use, and collaborating in biomarkers discovery projects.

Click Therapeutics develops and commercializes software as medical treatments for people with unmet medical needs. Through cognitive and neurobehavioral mechanisms, Click’s Digital Therapeutics™ enable change within individuals, and are designed to be used independently or in conjunction with biomedical treatments. The Clickometrics® adaptive data science platform continuously personalizes user experience to optimize engagement and outcomes. Following a groundbreaking clinical trial, Click’s industry-leading smoking cessation program is available nationwide through a wide variety of payers, providers, and employers. Click’s lead prescription program is entering into a multi-center, randomized, controlled, parallel-group, phase III FDA registration trial for the treatment of Major Depressive Disorder in adults.

Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases.

Muna Therapeutics is a private biopharmaceutical company that discovers and develops therapies that slow or stop devastating neurodegenerative diseases including Parkinson's, Alzheimer's, Frontotemporal Dementia, and Multiple Sclerosis.

Glycomine is early-stage biotech working on replacement therapies for rare diseases. There are approximately 7,000 rare disorders worldwide. Each day more are being discovered. 95% of rare diseases have not one single FDA-approved treatment. Glycomine develops therapeutics for diseases that have no treatment options.

Icosavax is a biopharmaceutical company leveraging its innovative virus-like particle (VLP) platform technology to develop vaccines against infectious diseases, with an initial focus on life-threatening respiratory diseases. Icosavax’s VLP platform technology is designed to enable multivalent, particle-based display of complex viral antigens, which it believes will induce broad, robust, and durable protection against the specific viruses targeted. Icosavax’s pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Icosavax was formed in 2017 to advance the breakthrough VLP technology from the Institute for Protein Design at the University of Washington with the goal to discover, develop, and commercialize vaccines against infectious diseases.

Medisafe is a digital patient engagement platform, empowering patients to stay on top of all their medications. Its approach leverages the billions of data points collected to personalize patient engagement and improve adherence and persistence to medications. By addressing the major challenges of medication management utilizing a data science-driven approach, Medisafe improves patient adherence and quality of care. The company provides its pharma and payer partners the opportunity to engage each patient on an individual level, supporting them throughout the entire patient journey, and provides unique insights on behavior patterns critical for improving the patient experience. Medisafe had shown over numerous studies, both retrospective with tens of thousands of patients as well as RCTs, that its solutions improve adherence by up to 20%. Founded following an accidental insulin overdose of the founder's father, the Medisafe solution currently has nearly 5 million registered users across iOS and Android smartphones, tablets, wearables, and Web solutions, who have recorded over 2 billion medication doses. Nearly 200,000 users have rated the app an average of 4.7 out of 5 stars in the App Store and Google Play store, making it the Top Rated solution in the category. Their accolades include Gartner Cool Vendors, Traction winner (Health 2.0 startup competition), Fast Company Most Innovative in Healthcare, Google and Apple Editors' Choice and Top Developer, etc.

DICE Therapeutics is a biopharmaceutical company leveraging its proprietary technology platform to build a pipeline of novel oral therapeutic candidates to treat chronic diseases in immunology and other therapeutic areas. DICE is initially focused on developing oral therapeutics against well-validated targets in immunology, with the goal of achieving comparable potency to their systemic biologic counterparts, which have demonstrated the greatest therapeutic benefit to date in these disease areas.  The Company’s DELSCAPE platform is designed to discover selective oral small molecules with the potential to modulate protein-protein interactions (PPIs) as effectively as systemic biologics. DICE’s lead therapeutic candidate, S011806, is an oral antagonist of the pro-inflammatory signaling molecule, interleukin-17 (IL-17), which is a validated drug target implicated in a variety of immunology indications. DICE is also developing oral therapeutic candidates targeting α4ß7 integrin and αVß1/αVß6 integrin for the treatment of inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.

Lava Therapeutics is a developer of a bispecific antibody platform used to engage gamma-delta T cells for the treatment of hematological and solid cancers. Its platform creates and develops next-generation γδ T cell engaging bispecific antibodies for the treatment of cancer as well as develop potent, safe, and cost-effective biopharmaceuticals that arm the immune system to recognize and destroy tumor cells that enable doctors to safely channel the immune system's response towards tumors.

Science 37 is making the promise of virtual trials the new reality. By engaging with patients from the comfort of their own home, the company provides access to patients who can never be reached by traditional site-based research models. Its virtual model has proven to enroll faster, retain patients at a higher rate, and reach a more representative patient population. Science 37 has conducted more virtual (or decentralized), interventional trials than any other company, using an expansive, in-house network of telemedicine investigators and home-health nurses. The company's research is powered by the industry’s most comprehensive, fully integrated clinical trial platform purpose-built to conduct virtual trials.

KaloBios Pharmaceuticals provides medicines for respiratory diseases and certain cancers. The company is a biopharmaceutical company focused on advancing medicines for patients with neglected and rare diseases. They designed to overcome marketed antibody products.

NodThera is a Cambridge (UK) headquartered biotech engaged in innate immune/ inflammasome research. Their leading project is focused on developing inhibitors of the NLRP3 inflammasome for the treatment of inflammatory and neuroinflammatory diseases.

i2o Therapeutics is a biotech company that develops safe and effective oral formulations of therapies traditionally limited to injections. Using an innovative ionic liquid technology, it leverages the benefits of protecting the drug cargo while also transiently enhancing permeation across the epithelial lining when administered orally. i2o is focused on creating the next generation of oral peptide and protein-based therapies. The company was founded in 2019 and based in Saratoga, California.

Omada Health is a digital care company that offers innovative and clinically effective digital health programs to empower individuals to engage in their health and lead healthier lives. The program is personalized to meet each participant’s unique needs as they evolve, ranging from diabetes prevention, type 2 diabetes management, hypertension, behavioral health, and musculoskeletal issues. Omada combines professional health coaching, connected health devices, real-time data, and personalized feedback to deliver clinically meaningful results. The company is the largest CDC-recognized provider of the National Diabetes Prevention Program.

Inozyme is a provider of therapies for rare diseases of calcification affecting soft tissues and bone created to offer potentially disease-modifying therapies to help children who are affected with rare, but severe and debilitating disorders of metabolism. The company's enzyme replacement therapy provides the treatment of Generalized Arterial Calcification of Infancy (GACI) and Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2), enabling patients to treat diseases characterized by mineral imbalances in the body.

Aetion delivers real-world evidence (RWE) and outcomes-based analytics solutions to life sciences companies, payers, and at-risk providers. With its patented rapid-cycle analytics technology, the Aetion Evidence Platform utilizes the everyday clinical and financial interactions of the healthcare system to unlock essential evidence about the effectiveness and value of medical treatments. Founded by Harvard Medical School faculty members and top big data technologists, Aetion enables payers and providers to collaborate with biopharmaceutical and medical device companies in real-time to develop therapeutic insights and make smart choices in inpatient care.  Aetion is based in New York City and backed by investors including New Enterprise Associates (NEA), Flare Capital Partners, Lakestar, Greenspring Associates, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, EDBI, Amgen Ventures, Johnson & Johnson Innovation — JJDC, Inc., UCB, and Horizon Health Services, Inc.

Kymera Therapeutics is a biotechnology company that specializes in the field of targeted protein degradation. It features a proprietary predictive modeling degradation platform with a novel small molecule modality to accelerate the drug discovery designed for disease-causing proteins.

Evidation measures health in everyday life and enables anyone to participate in groundbreaking research and health programs. Built upon a foundation of user privacy and control over permissioned health data, Evidation's Achievement platform is trusted by millions of individuals—generating data with unprecedented speed, scale, and rigor. We partner with leading healthcare companies to understand health and disease outside the clinic walls. Guided by our mission to enable and empower everyone to participate in better health outcomes, Evidation is working to bring people individualized, proactive, and accessible healthcare—faster.  Founded in 2012, Evidation Heath is headquartered in California. We collaborate as a distributed team with additional offices around the globe. To learn more, visit evidation.com, or follow us on Twitter @evidation

Imbria Pharmaceuticals’ mission is to translate our understanding of cellular metabolism into innovative medicines that improve the lives of patients with limited or inadequate treatment options. The company was founded by doctors and launched by an all doctor executive team motivated by the promise of bringing new therapies to people in need.  We are propelled by a deep scientific interest in cellular metabolism and the mitochondria, and are guided by both values and a code of business conduct built upon our commitment to always put the patients, families and communities we serve at the forefront of all that we do.

Common Sensing develops and manufactures data-driven hardware and software medical device solutions for diabetes care that foster education and communication for patients while delivering decision support for clinicians. Common Sensing has a team of talented designers and engineers to develop Gocap™, a smart cap that turns existing injector pens into smart injectors.

Curisium a healthcare technology and services company that provides innovative contracting solutions.

NeuroVia, Inc. is committed to addressing unmet medical needs in neurological diseases with the primary goal to arrest the onset of devastating neurological deficits associated with X-ALD.

ImmuneXcite has developed a proprietary platform technology for enhancing the efficacy of therapeutic monoclonal antibodies for oncological and infectious disease targets.  ImmuneXcite was founded based on research originating from the Whitehead Institute at MIT, where a unique carbohydrate was shown to activate neutrophils.   Chemically linking the carbohydrate to monoclonal antibodies to improve their effector function is a bold concept that has been supported by the Massachusetts Technology Transfer Center, Deshpande Center for Technological Innovation at the Massachusetts Institute of Technology (MIT), the Department of Defense, Massachusetts Life Science Center, National Cancer Institute, and private investor.  Immunexcite, Inc., a biopharmaceutical company, develops monoclonal antibodies for cancer. The company develops monoclonal antibodies by employing its mAbXcite, an immunotherapy platform technology that targets cancer cells for destruction by engaging neutrophils. Immunexcite, Inc. was founded in 2007 and is based in Lexington, Massachusetts.

Yumanity Therapeutics is transforming drug discovery for neurodegenerative diseases caused by protein misfolding. Founded in December 2014 by award-winning protein folding expert, Susan Lindquist, and renowned biotech industry leader, Tony Coles, Yumanity is working to identify and develop new, disease-modifying therapies that address several illnesses with critical unmet medical needs. The initial focus of the company is neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS).  The company’s proprietary platforms have already identified one potential new target for treating Parkinson’s disease, and Yumanity is actively advancing its new chemical lead series for this condition, as well as identifying additional compounds for Alzheimer’s disease and ALS.

Navitor Pharmaceuticals is a developer of novel medicines designed to target specific nutrient-sensing proteins that selectively regulate mTORC1 activity to treat diseases. The company's medicines selectively modulate the activity of mTORC1, a master regulator of cell growth and metabolism, and unlock the therapeutic potential of the mTOR pathway by targeting the amino acid and other nutrient sensors that selectively regulate the activity of mTORC1, enabling healthcare practitioners to address a wide range of metabolic, neurodegenerative, autoimmune and musculoskeletal diseases. It was founded in 2009 and headquartered in Cambridge, Massachusetts.

Selecta Biosciences is a biopharmaceutical company developing the first generation of nanoparticle immunomodulatory drugs for the treatment and prevention of human diseases. Their proprietary product platform technology combines recent advances in immunobiology with cutting-edge nanotechnology to develop targeted immunomodulatory nanoparticles. It was founded in 2008 and is headquartered in Watertown, Massachusetts.

Proteostasis Therapeutics is discovering and developing novel small molecule therapeutics designed to control the body's protein homeostasis, or Proteostasis Network. The Proteostasis Network maintains the body's natural balance of proteins to protect us from numerous diseases. These novel therapies, or Proteostasis Regulators, are designed to treat multiple genetic and degenerative disorders associated with deficiencies of the Proteostasis Network, such as emphysema, type II diabetes, Alzheimer's Disease and Huntington's Disease.

Ovid is a pure play neurology company focused on rare and orphan diseases of the brain. They were founded in 2014 to leverage recent advances in science and medicine to find optimal therapies. They seek to identify and develop late stage, de-risked medicines with significant potential.   Their rationale for developing these medicines is based on key insights from their scientific founders and confirmed in discussions with leading experts in the field. Their products are protected by strong intellectual property. They apply the most efficient processes to bring safe and effective therapies to patients.   They are based in New York City but have collaborators and are working with patients across the United States and the globe.

Cogent Biosciences uses proprietary T-cell engineering technology in combination with tumor-targeting antibodies to activate the body’s own immune system to fight cancer. Unum’s lead program, based on its antibody-coupled T-cell receptor technology, is expected to enter Phase I clinical testing in the coming months to assess safety and efficacy.

Lysosomal Therapeutics is dedicated to innovative small-molecule research and development in the field of neurodegeneration, yielding new treatment options for patients with severe neurological diseases. Their strategy leverages the clinically-validated link between lysosome-based genetic disorders and neurodegenerative diseases to establish a unique and effective molecular platform for novel drug discovery. LTI’s lead program targets Gaucher-related neurodegeneration, Parkinson’s disease, and other synucleinopathies.

Immune Design Corp. (IDC) uses breakthrough technology to develop safe and effective vaccines and therapeutics, with emphasis on the prevention and treatment of infectious disease. This goal is being achieved through dedicated research aimed towards a greater understanding of the immune system, and by developing a new generation of immune-based products for the pharmaceutical industry. IDC is passionate about its mission to alleviate human suffering and pursues this goal with high energy and an unsurpassed standard of excellence

KAHR Medical (''KAHR'') is pioneering the development of ''third generation biological drugs,'' a unique class of proprietary fusion-protein molecules with clear and far-reaching advantages. KAHR's technology represents a paradigm shift in protein-based pharmaceuticals that will significantly expand available treatment options for cancer and autoimmune disorders.

Edimer Pharmaceuticals: Working toward improving the health and quality of life of families living with XLHED.  Edimer Pharmaceuticals is dedicated to developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED). XLHED is a rare, orphan disease that causes a range of symptoms including lack of sweat glands, poor temperature control, respiratory problems, and hair and tooth malformations.  Edimer is led by a team of seasoned biotechnology industry veterans with deep experience in drug development complemented by a strong network of world-class clinical and scientific advisers.

Lumena Pharmaceuticals is developing oral therapeutics for rare liver diseases to improve liver function, relieve disease symptoms and dramatically impact patient health. The company’s clinical-stage product candidates carry a reduced risk of systemic toxicities by selectively targeting a transporter in the intestine and are designed to be minimally absorbed in the body. Lumena’s lead candidate, LUM001, has been extensively evaluated in other indications across 12 clinical studies in more than 1,400 subjects. Because of the extensive preclinical and clinical data package for a compound at this stage in development, LUM001 is positioned to rapidly progress through the clinic in pediatric and adult patients with several types of cholestatic liver disease. While Lumena’s primary focus is to develop novel treatments for patients with rare liver diseases, the company’s therapeutic approach also has promising potential in the treatment of metabolic diseases affecting the liver, a significant and growing health problem in children and adults.

bluebird bio is a clinical-stage biotechnology company that develops gene therapies for severe genetic and rare diseases. The company's gene therapy procedures aim to genetically modify a patient's cells to fundamentally correct or address the genetic basis underlying a disease. Bluebird's revenue is derived from collaboration arrangements, research fees, license fees, and grant revenue. The company works with industry peers, strategic partners, and nonprofit organizations for the development and commercialization of its product candidates.

Fate Therapeutics is using the fundamental biological mechanisms that guide cell fate to develop stem cell therapeutics. The company has brought together the foremost scientists from the nation's research hotbeds (Boston, San Francisco, San Diego, and Seattle) who have demonstrated the potential to create and modulate stem cells to restore health.  The backstory is that while others were working towards therapies based on transplanting stem cell-derived cells into patients, Randall Moon (HHMI and the University of Washington) and Alex Rives (Arch Venture Capitol) envisioned a different approach to develop regenerative therapies. The concept, based on data from hematopoietic progenitor cells, was that one could modulate the properties of stem cells in vivo using small molecules or biologics. They presented this idea to Arch Venture Capitol in Seattle, which launched the company, soon to be joined by other investors.

GlycoMimetics, Inc., a biopharmaceutical company, engages in the design and development of small molecule therapeutics to mimic the functions of bioactive carbohydrates. Its products include GMI-1070, a synthetic glycomimetic molecule designed to inhibit selectin types, such as E, P, and L-selectin; and GMI-1051, a small molecular weight compound for the treatment or prevention of infections caused by pseudomonas aeruginosa. The company was founded in 2003 and is based in Gaithersburg, Maryland.

TcLand Expression is a fully integrated company displaying high expertise in all stages of biomarker development: R&D, bioinformatics & biostatistics, clinical & regulatory , market access and production.   TcLand Expression is a pioneer and European leader in personalized medicine in immunology (transplantation and auto-immune disorders) with a rich pipeline of biomarkers in development.

[Sirtris Pharmaceuticals](http://www.sirtrispharma.com) (NASDAQ: SIRT) is a biopharmaceutical company focused on discovering and developing proprietary, orally available, small molecule drugs with the potential to treat diseases associated with aging, including metabolic diseases such as Type 2 Diabetes.   via: [Sirtris](http://www.sirtrispharma.com)

Sanofi Ventures is the corporate venture capital arm of Sanofi. We have the ability to seed and to lead financings in early-stage companies with innovative ideas and transformative new products and technologies that are of strategic interest to Sanofi. Among these areas are rare diseases, oncology, immunology and inflammation, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health and data science solutions.

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