SR One

Leveraging personal data for custom-fit diagnosis and treatment

Precision Medicine

Philadelphia- and Israel-based FORE Biotherapeutics (formerly known as NovellusDx) is a next-generation oncology-focused precision medicine drug developer. The company uses advanced technology to target specific mutations in tumors that are unaddressed. The company strategy looks to identify (identifying new mutation targets through functional genomics), match, build (defines new patient subpopulations), and partner. Its Foresight engine synthesizes, transfects, and analyzes an image. In 2020, the company established its global headquarters in the US.In conjunction with financing in August 2023, FORE announced that CEO Matthew Ros would step down and board member Shawn Leland, formerly of Elevation Oncology, would take over as interim CEO.Plixorafenib (formerly known as FORE8394, PLX-8394) is an investigational, novel, orally available, small-molecule, next-generation selective inhibitor targeting mutated BRAF. By inhibiting ERK signaling, it shows promise for treating tumors driven by class I or II BRAF mutations and fusions. The US FDA has granted Orphan Drug designation to plixorafenib for the treatment of primary brain and CNS malignancies, and Fast Track designation for treating cancers with BRAF Class 1 (V600) and Class 2 (including fusions) alterations in patients who have exhausted prior therapies.

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 MiroBio is a clinical-stage biotechnology startup operating out of the UK that discovers and develops therapeutic antibodies to restore balanced immune function and prevent further tissue damage. Its proprietary I-ReSToRE (Receptor Selection and Targeting to Reinstate Immune Equilibrium) platform features a checkpoint atlas of receptor expressions and function, antibody discovery process, and advanced bioinformatics. The firm&rsquo;s technology was built on more than 15 years of checkpoint agonism research at the University of Oxford.
 
 
 The firm has two main antibody drug candidates that are in the preclinical stage of the drug development process. MB272, the firm&rsquo;s lead program, and MB151 are checkpoint receptor agonists targeting the proteins BTLA (B- and T-Lymphocyte Attenuator) &amp; PD-1 (Programmed cell death protein 1), respectively, found in autoimmune diseases. Miro is also leveraging its I-ReSToRE platform to advance its pipeline of drugs focused on restoring immune homeostasis in patients with autoimmune diseases.
 
 
 Key customers and partnerships
 
 
 In August 2022, global biopharmaceutical company Gilead Sciences s
 <a href="https://sp-edge.com/updates/12177">
 igned a definitive agreement to acquire
 </a>
 MiroBio for a cash consideration of USD 405 million. Post-acquisition, Gilead will have access to MiroBio's I-ReSToRE platform, and the entire portfolio of drug candidates. The pharma company plans to combine the I-ReSToRE platform with its existing R&amp;D to treat autoimmune and inflammatory diseases and to expand its pipeline in the coming years.
 
 
 Funding and Financials
 
 
 The firm raised USD 97 million (GBP 80 million) in a Series B funding round led by Medicxi in June 2022. The funds were allocated toward advancing its lead antibody drug candidates MB272 and MB151 into clinical trials. The funds were also directed toward advancing its preclinical pipeline and building out its proprietary I-ReSToRE discovery platform.
 
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 Lexicon Pharmaceuticals is a biopharmaceutical company focused on discovering and developing pharmaceutical products for treating various chronic diseases related to fields such as immunology, metabolism, cardiology, and ophthalmology. Lexicon scientists analyzed the role and function of roughly 5,000 genes as part of its Genome5000 initiative, and discovered more than 100 protein targets for treating various disorders.&nbsp;
 
 
 In May 2023, the company obtained 
 <a href="https://sp-edge.com/updates/18933">
 FDA approval for its drug INPEFA
 </a>
 (sotagliflozin), an oral tablet designed for treating heart failure, including in adults with type 2 diabetes. The company had plans to launch its drug to the US market in June 2023, with a wholesale price comparable to existing branded heart failure treatments (~USD 570 for a month&rsquo;s supply). Furthermore, market analysts predicted that the medicine, which would compete with Eli Lilly's Jardiance (licensed in February 2022 for the same indication), would generate USD 112 million in revenue by 2025 and USD 576 million by 2028.
 
 
 In addition, as of August 2023, the company had three drug candidates in its pipeline, including LX9211 in Phase IIb clinical trials to treat diabetic peripheral neuropathic pain.&nbsp;
 
 
 Key Customers and Partnerships
 
 
 The company has partnered with leading pharmaceutical companies like Bristol-Myers Squibb (since December 2003), Genentech (in December 2005), N.V. Organon (since May 2005), and Takeda Pharmaceutical (since August 2004). Furthermore, the company partnered with French drugmaker Sanofi SA to develop the diabetes drug, Zynquista. However, in September 2019, Sanofi terminated the alliance and paid USD 260 million as termination fee to Lexicon.
 
 
 Funding and Financials
 
 
 The company began trading on Nasdaq in February 1998 under the ticker symbol &ldquo;LXRX.&rdquo; In June 2023, the company closed an underwritten public offering and concurrent private placement with expected gross proceeds to the company of USD 71.5 million. The funds raised were used toward the commercial launch of INPEFA.
 
 
 For the three months ended March 2023, the company did not generate revenue. However, it reported a significant increase of 35% YoY in the net loss to USD 31.9 million. Its cash and cash equivalent as of March 2023 was at USD 105.9 million compared to USD 138.4 million in Q4 2022. 
 
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 Odyssey Therapeutics is a biotechnology company that focuses on discovering and developing precision oncology drugs and immunomodulators by building a functional genomics platform that&nbsp; leverages AI and machine learning (ML). The company employs an integrated platform approach that combines a drug discovery engine with ML, biology, and medicinal and structural chemistry. Its approach ensures optimal product profiles and biologic attrition for the respective therapeutic area.&nbsp;
 
 
 As of October 2022, the company had eight programs in the pipeline in two modalities, small molecule (orally administered) and protein therapeutics. It planned to submit multiple investigational new drug applications in 2023 and enter clinical trials by late 2023 or early 2024. In January 2022, the company acquired quantum machine learning company Rahko to expand the capabilities of its discovery platform.&nbsp;
 
 
 Funding and financials
 
 
 In December 2023, Odyssey Therapeutics has raised USD 101 million in Series C funding round led by Ascenta Capital. The company expected to use the funds to support the advancement of clinical studies of several programs and to add to investment in efforts to develop a sustainable model for therapeutic innovation. 
 
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Nomic Bio is a protein profiling company that has developed a proprietary platform, nELISA, which enables massive multiplexing with flexibility, full quantification, and cost-efficiency in protein measurement. Nomic's technology aims to change how drugs are developed and how diseases are diagnosed by providing a comprehensive solution for inflammation research that allows the high-throughput quantification of hundreds of secreted proteins.&nbsp;The nELISA platform miniaturizes immunoassays into small beads, isolating target-specific antibody pairs to their own color-coded beads, which prevents cross-reactive interactions and allows highly specific results at a fraction of the time and cost of traditional methods. As of September 2024, Nomic had profiled more than 25 million proteins. Also, the company was expanding its biological footprint to cover proteins across the entire proteome, aiming to unlock new possibilities for diverse applications, use cases, and disease areas.&nbsp;Key customers and partnershipsAs of September 2024, Nomic had served over 75 companies, including top 10 pharmaceutical companies, biotechs, and research institutes.Nomic Bio's customer base spans various sectors of the life sciences industry, profiling over 400,000 samples using Nomic's technology. The company's platform has been employed across different areas of biomedical research and pharmaceutical development.

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 California-based Alumis (previously Esker Therapeutics) is a clinical-stage precision medicine company focused on the discovery, development, and treatment of autoimmune disorders. The company leverages a precision analytics platform, powered by Foresite Labs, consisting of curated genetic, clinical, and medical record data, a systems immunology approach for prospective data collections, and tools for building patient registries.
 
 
 Alumis&rsquo; fully-owned lead pipeline product, ESK-001, is a Tyrosine Kinase 2 (TYK2) inhibitor in Phase 1 clinical trials for treating psoriasis. The company is also conducting preclinical studies of the drug for other autoimmune diseases. In addition, the company is conducting discovery programs for two other undisclosed targets.&nbsp;
 
 
 The company was founded in 2021, launching out of stealth a USD 70 million Series A funding round in May. In January 2022, the company raised USD 200 million in a Series B funding round led by AyurMaya, which will be used to develop its product pipeline with a focus on ESK-001 as well as other immunology programs targeting autoimmune diseases.&nbsp;
 
 
 
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Artificial intelligence is identifying and testing new therapies faster than ever.

AI Drug Discovery

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 Nimbus Therapeutics is a biotechnology company designing small molecule compounds targeted at various diseases. It utilizes the chemical simulation computational platform built by co-founder Schr&ouml;dinger Inc., which incorporates artificial intelligence and machine learning, and co-develops drugs by partnering with pharma companies. Its pipeline includes programs in metabolic diseases, cancer, and immune-inflammatory disorders.&nbsp;
 
 
 As of March 2024, the company was developing five targets, of which one is in the clinical stage, two are in the preclinical stage, and others remain at a discovery stage. The company initiated investigational new drug (IND)-enabling studies on two novel agents in 2022.
 
 
 Takeda Pharmaceuticals acquired Nimbus Therapeutics&rsquo;s fully owned subsidiary, Nimbus Lakshmi, and its tyrosine kinase 2 (TYK2) (intracellular enzyme) program 
 <a href="https://sp-edge.com/updates/16314">
 in February 2023
 </a>
 . Following the acquisition, Nimbus&rsquo; drug candidate, NDI-034858, for psoriasis was renamed to &ldquo;TAK-279.&rdquo;&nbsp;
 
 
 Key customers and partnerships
 
 
 Nimbus Therapeutics&rsquo;&nbsp; pharma partners include Roche&rsquo;s Genentech, Celgene, and Gilead.
 
 In 
 <a href="https://sp-edge.com/updates/24983">
 December 2023
 </a>
 , Nimbus Therapeutics partnered with Anagenex to discover small-molecule drugs for various complex targets. Further, in October 2022, the company partnered with Eli Lilly to progress the development of novel targeted therapies that activate AMPK to potentially treat a broad range of metabolic disorders.
 
 
 Funding and financials
 
 
 In September 2023, the company raised USD 210 million in a 
 <a href="https://sp-edge.com/updates/21624">
 private funding round
 </a>
 co-led by GV (Google Ventures) in September 2023 to advance Nimbus Therapeutics' pipeline of drugs for various disorders. 
 
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 HotSpot Therapeutics is a biotechnology company developing allosteric therapies for undrugged or poorly drugged targets. It focuses on targeting regulatory sites or pockets in proteins referred to as &ldquo;natural hotspots&rdquo; that have the ability to control cellular protein function. This approach is reportedly an overlooked method by drug developers, and so, the company boasts to be the first to follow it.&nbsp;
 
 
 The company leverages its platform Smart Allostery, which entails a broad range of technologies that combines AI, structure-function, and tailored chemistry, along with a chemical library tailored for hotspots.&nbsp;
 
 
 As of March 2024, the company had three drug candidates in its internal pipeline with two lead candidates, namely 1) HST-1011 to treat solid tumors in Phase I clinical trials and 2) HST-1021, a precision oncology candidate in the IND-enabling stage.
 
 
 Key customers and partnerships
 
 
 The company has entered several key partnerships including 1) Caris Life Sciences to develop its pipeline of precision medicine drugs and companion diagnostic tests (
 <a href="https://sp-edge.com/updates/7709">
 January 2022
 </a>
 ), 2) Excelra to leverage its Global Online Structure Activity Relationship Database (GOSTAR) platform to identify new drug targets (
 <a href="https://sp-edge.com/updates/10722">
 May 2022
 </a>
 ), and 3) AbbVie to develop the first small-molecule IRF5 inhibitor for treating autoimmune diseases (
 <a href="https://news.abbvie.com/2022-12-06-AbbVie-Launches-Strategic-Collaboration-with-HotSpot-Therapeutics-to-Further-Expand-Immunology-Pipeline">
 December 2022
 </a>
 ).&nbsp;
 
 
 Funding and financials
 
 
 In 
 <a href="https://sp-edge.com/updates/7003">
 November 2021
 </a>
 , HotSpot Therapeutics raised USD 100 million in a Series C funding round led by Pivotal BioVenture Partners. New investors, including Revelation Partners and Pavilion Capital, as well as existing investors Atlas Venture and Sofinnova Partners, also participated in the round. The funds were used to further develop its platform and advance its clinical pipeline.
 
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Odyssey Therapeutics is a biotechnology company focused on discovering, developing and commercializing next-generation precision immunomodulators and oncology medicines.The company’s platform integrates AI/ML, computational chemistry, and functional genomics to drive novel target discovery and molecular design. The company has proprietary covalent libraries targeting multiple amino acids, molecular glues, and natural products for its chemistry efforts.Odyssey's key product offerings include small molecule and protein therapeutics initially focused on immunology and oncology indications. The company’s portfolio comprises eight programs, with several candidates potentially entering clinical trials.In January 2022, Odyssey acquired Rahko, a quantum machine learning company, to further strengthen its AI and computational capabilities for drug discovery.Key customers and partnershipsIn <a href="https://sp-edge.com/updates/28236">April 2024</a>,Odyssey entered into a strategic research collaboration with Janssen Pharmaceutica NV to jointly discover and optimize small molecule medicines against select therapeutic targets by combining their expertise in AI, machine learning, and computational chemistry.

Targeting complex diseases with complex molecules

Large-molecule Therapeutics

F-star Therapeutics was a clinical-stage biopharmaceutical company pioneering bispecific antibodies for cancer immunotherapy. Its proprietary platform technology enabled the development of tetravalent (2+2) bispecific antibodies, which could simultaneously target two different antigens and elicit potent and focused immune activation in the tumor microenvironment. F-star had four clinical programs based on this technology: FS118, FS222, FS120, and SB 11285 (a next-generation STING agonist). These programs aimed to address unmet medical needs in various cancers by offering potentially first- and best-in-class drug candidates.
F-star's modular antibody technology also facilitated collaborations with major biopharmaceutical companies across oncology, immunology, and neurology. In June 2022, F-star entered into a definitive agreement to be acquired by invoX Pharma, a subsidiary of Sino Biopharmaceutical Limited, for approximately USD 161 million. The acquisition was completed in 2023, and F-star's assets, including its tetravalent bispecific antibody platform and clinical programs, became part of invoX Pharma's oncology research and development efforts.

Elektrofi is a Boston-based biotechnology company focused on developing innovative drug formulation and delivery technologies. The company's breakthrough formulation platform addresses the long-standing need for stabilized, highly concentrated biologics through a proprietary microparticle technology. This technology enables small-volume injections for monoclonal antibodies, therapeutic proteins, and other large molecule drugs, offering significant advantages over traditional intravenous administration methods.
Elektrofi's platform has the potential to revolutionize the treatment landscape across various disease areas and indications. By enabling patient-friendly subcutaneous administration of biologic therapies, the company aims to enhance convenience, reduce healthcare costs, and improve access to life-changing medicines. The technology can transform intravenously administered therapies into convenient, ready-to-use formulations suitable for self-administration at home.
In June 2022, Elektrofi raised USD 40 million in a Series B financing round, which will be used to establish a centralized GMP manufacturing line to support upcoming clinical trials and expand the company's development pipeline. As of June 2022, Elektrofi aimed to double its headcount within the next 12-18 months to accelerate the development of its licensed programs with pharmaceutical and biotechnology partners.
Key customers and partnerships
Elektrofi has formed strategic partnerships and collaborations with several leading pharmaceutical companies, including Takeda Pharmaceutical Company, Argenx, and Janssen biotech. In January 2024, Elektrofi and Janssen biotech entered into a worldwide collaboration and license agreement to develop up to five programs utilizing Elektrofi's formulation technology, with an initial focus on an oncology asset for at-home self-administration.

Long-term demographic changes are finally bringing fertility technologies into focus.

Fertility Tech

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 Progyny works with employers (primarily large enterprises in the US) to optimize the fertility benefits they offer for their employees. It connects employees to fertility specialists and clinics, and also offers extensive support to patients through their Progyny Patient Care Advocates, who provide clinical guidance, education, and emotional support.
 
 
 The company was founded as a fertility education platform and eventually included in vitro fertilization (IVF) referral network Fertility Authority and reproductive health company Auxogyn, into its network. The company pivoted into offering fertility benefit solutions in 2016, and launched Progyny Rx (fertility medication benefit) in 2018.
 
 
 Progyny connects patients to a network of 800 fertility specialists and nearly 600 clinic locations.&nbsp;
 
 
 Key customers and partnerships
 
 
 As of March 2022, the company served 264 enterprise customers (including Facebook, Microsoft, Google, and Nasdaq), and had 3.9 million active members.
 
 
 Funding and financials
 
 
 In October 2019, Progyny raised USD 130 million through its IPO, where it was listed on the Nasdaq.
 
 
 In FY2021, the company reported a revenue of USD 500.6 million reflecting a 45% YoY growth and a net income of USD 65.8 million. The company&rsquo;s revenue for 
 <a href="https://sp-edge.com/updates/14396">
 Q3 FY2022
 </a>
 amounted to USD 205.4 million, representing 68% YoY growth. The management&rsquo;s revenue guidance for FY2022 ranges between USD 775 million and USD 785 million, which implies an YoY growth of 55% to 57%.
 
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Reinventing gene therapies with next-generation technologies

Human Gene Editing

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 CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, the company had 18 drug candidates in its pipeline. 
 
 
 In a milestone event in November 2023, the company&rsquo;s gene-editing therapy exagamglogene autotemcel, CASGEVY 
 <a href="https://sp-edge.com/updates/24093">
 (exa-cel)
 </a>
 , developed with Vertex Pharmaceuticals to treat sickle-cell disease and beta-thalassemia, received the world&rsquo;s first authorization from the UK's Medicines and Healthcare products, Regulatory Agency (MHRA). In December 2023, it received FDA approval for patients aged 12 and older. The drug for beta-thalassemia patients is also nearing its market entry in the US, with the FDA&rsquo;s decision expected on March 30, 2024.&nbsp;
 
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured strategic collaborations with several companies to co-develop and co-commercialize gene editing medicines that include 
 <a href="https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-and-crispr-therapeutics-amend">
 Vertex Pharmaceuticals
 </a>
 (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and 
 <a href="https://viacyte.com/press-releases/crispr-therapeutics-and-viacyte-announce-strategic-collaboration-to-develop-gene-edited-stem-cell-derived-therapy-for-diabetes/">
 ViaCyte
 </a>
 (September 2018) to develop VCTX210, a regenerative medicine for diabetes.
 
 
 Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.&nbsp;&nbsp;
 
 
 
 
 
 Funding and financials
 
 
 
 
 Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.
 
 
 
 
 
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Novel therapies to reboot cells and organs

Cell & Gene Therapy

UK- and US-based Akamis Bio (previously known as PsiOxus) is a developer of therapeutics used to treat cancer by using gene therapy platforms. The company develops tumor-specific therapeutics and is curating a library of Tumor-Specific Immuno-Gene (T-SIGn) therapeutics that help a patient’s immune system recognize, attack, and clear cancer cells. Akamis is developing its T-SIGn therapeutics in such a way that the company claims it can enhance the effectiveness of other cell-based therapeutics such as chimeric antigen receptor T-cell (CAR-T)-based therapies.As of May 2024, the company had one therapeutic using CD40 agonistic platforms for tumor-specific targeting called NG-350A in Phase I of clinical trials. Akamis had another therapeutic candidate using T-cell and Natural Killer cell activation (NK) for tumor-specific targeting called NG-7xx in investigational new drug application (IND)-enabling stage of clinical trials. Akamis was also developing a therapeutic using CAR-T cell targeting and activation for specific tumor-targeting for an undisclosed party. The solution was in the research stages. Key customers and partnershipsAkamis has listed several collaborations that it has carried out with other pharmaceutical providers such as Bristol Myers Squibb, Dana-Farber Cancer Institute, MSD, and the University of Oxford.Further to the above, the company partnered with the Parker Institute for Cancer Immunotherapy (PICI) and Cancer Research Institute (CRI) in January 2023 to assess Akamis’ cancer therapeutics. Through the partnership, PICI and CRI would assess Akamis’ therapeutic candidates NG-350A and CTLA-4.

Managing employee health-related benefits

Health Benefits Platforms

Progyny works with employers (primarily large enterprises in the US) to optimize the fertility benefits they offer to their employees. It connects employees to fertility specialists and clinics and also offers extensive support to patients through its Progyny Patient Care Advocates, who provide clinical guidance, education, and emotional support. As part of its solution, the company offers Smart Cycle, a benefit currency that enables physicians to customize treatment plans according to the needs of a diverse user population, including single parents and the LGBTQ+ community.The company was founded as a fertility education platform and eventually included in vitro fertilization (IVF) referral network Fertility Authority and reproductive health company Auxogyn into its network. The company pivoted into offering fertility benefit solutions in 2016 and launched Progyny Rx (fertility medication benefit) in 2018.As of July 2024, Progyny connected patients to a network of over 1,000 fertility specialists across over 650 clinic locations. Key customers and partnershipsAs of July 2024, the company had served over 460 enterprise customers (including Nike, Microsoft, Google, and Genetec) and had 6.7 million members.

RNA Therapeutics

ADARx Pharmaceuticals is a clinical-stage biotechnology company focused on developing next-generation RNA therapeutics. The company utilizes its proprietary RNA targeting platform, which includes technologies for oligonucleotide inhibition, degradation, and editing, along with novel oligonucleotide delivery methods. ADARx is advancing a pipeline of RNA-targeting therapeutics to treat diseases across various therapeutic areas including genetic, cardiometabolic, complement-mediated and central nervous system disorders.
The company's lead product candidate, ADX-324, represents an innovative approach for treating hereditary angioedema, a rare genetic disease that causes painful, rapid, and potentially life-threatening swelling attacks. Early phase 1 clinical data has shown promising results for ADX-324. ADARx is also developing ADX-038 for the treatment of multiple complement-mediated diseases. The company's proprietary targeted delivery PLR platform and broad SPE technology have been validated by clinical data. In August 2023, ADARx announced it had raised USD 200 million in Series C financing to further advance its clinical programs and support development of its pipeline of mRNA silencing or editing candidates.

Nomic is a bioengineering company working to make the human proteome as broadly and easily accessible as the human genome. They are building the nELISA, a next-generation ELISA platform for measuring proteins at scale and at high throughput. They offer scientists protein profiling solutions to empower them to decide the scope and scale of their experiments.

Elektrofi is a preclinical-stage company dedicated to redefining the standard of drug delivery by transforming the patient experience. Elektrofi’s formulation system can effectively process a wide range of therapeutic molecules. Their technology has the capacity to process large, increasingly complex and delicate proteins including monoclonal antibodies, bispecifics and fusion proteins. Founded in 2016, Elektrofi is based in Boston, Massachusetts, United States.

Renalys Pharma is a private, late-stage clinical biopharmaceutical firm in Japan dedicated on developing new therapies that address unmet needs in the management of renal illness in Japanese and Asian patients.

Asceneuron develops effective therapeutics for orphan tauopathies and Alzheimer’s disease. By focusing on areas of high unmet medical need, Asceneuron aspires to become a leading biotech company specialized in small molecule drug discovery for neurodegenerative diseases.  Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as e.g. neurofibrillary tangles in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major contributor to neurodegeneration and mutations in the tau gene are causative of the tauopathy fronto-temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17).  Due to increasing life expectancy, Alzheimer’s disease is viewed as one of the largest healthcare problems of this century, imposing a major economic burden on societies in the Western and developing world. Current treatment options provide limited benefits supporting the urgent need for more efficacious and better tolerated medicines that address symptomatic relief as well as disease progression.

Kala Pharmaceuticals is developing novel treatments for diseases that affect mucosal tissues. The company's proprietary mucus penetrating particle technology dramatically improves drug distribution and pharmacokinetics by uniformly coating the mucosal surface with biocompatible, drug-loaded particles. Kala's formulations achieve high, sustained local drug concentrations with reduced systemic exposure and are delivered in a patient-friendly format. The company's research efforts have demonstrated successful in vivo proof-of-concept in mucosal organs such as the respiratory tract, eye, and cervicovaginal tract, enabling diverse product opportunities that target significant clinical unmet need. Kala is focused on advancing an internal pipeline of products through human proof-of-concept while establishing R&D collaborations with select partners to improve products in their pipelines.

Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.

Nkarta seeks to improve upon the efficacy of cell therapy, making it more potent, better tolerated and more rapidly available to a broad population of patients with a variety of hematologic and solid tumor malignancies. While T-cell therapy, often known as CAR-T, has proven successful in certain cancer indications, its promise has been limited to a few hematologic malignancies and the side effects can be severe. Natural Killer cells are the immune system’s first responders against diseases and pathogens. Unlike T cells, they have an inherent ability to target tumor cells without genetic alteration, and therefore hold significant promise in the development of potent therapies that can be used more readily for diverse tumor types. Nkarta’s differentiated cell therapy approach is designed to boost and enhance the inherent power of Natural Killer cells to target and destroy tumor cells, while also offering a better tolerated safety profile.

MISSION Therapeutics is a biotechnology company specializing in developing drugs that target the cellular process of deubiquitylation for the treatment of cancer and other diseases. Their focus is on developing small molecule drugs that selectively inhibit specific deubiquitylating enzymes (DUBs), which play crucial roles in regulating protein degradation and cellular processes. These drugs aim to intervene in disease mechanisms where DUB dysregulation is implicated, offering potential therapeutic benefits.

Alumis develops precision medications to treat autoimmune illnesses. The company's analytics platform and understanding of immunologic disorders aid in the development of treatments and medications for autoimmune diseases, allowing patients to alleviate suffering and modify their way of life.

CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.

Odyssey Therapeutics is a biotechnology company pioneering the efficient development of next-generation immunomodulators and oncology medicines. Odyssey is making a transformational impact on the field of drug discovery and is accelerating the path to clinical development to drive the creation of more effective precision medicines. The company's integrated drug-hunting engine is designed to accelerate drug discovery by unlocking targets with great potential and minimizing attrition.

Avalyn Pharma is a biopharma company that advances therapies for the treatment of Idiopathic Pulmonary Fibrosis (IPF) and other severe respiratory diseases. Its therapies rapidly advance AP01 for the treatment of IPF, provides additional pipeline candidates for IPF, improve the care and outcomes of severe respiratory diseases, and develop novel inhaled therapeutics. Avalyn Pharma was established in 2011 and is headquartered in Seattle, Washington.

Nimbus Discovery is now Nimbus Therapeutics to reflect our transition into a clinical-stage company. Nimbus Therapeutics is a biotechnology company headquartered in Cambridge, Mass., that is pioneering a new computational technology-driven paradigm to rapidly advance a diverse pipeline of novel small molecule product candidates into clinical development. Nimbus Therapeutics designs highly selective and potent medicines to disrupt known drivers of serious diseases, including metabolic disease, cancer, and immune-inflammatory disorders.

Fore Biotherapeutics is a precision oncology company specializing in cancer therapies guided by functional genomics. The company is creating a pipeline of precision oncology treatments aimed at patients with unaddressed mutations across well-established oncology targets.

ADARx Pharmaceuticals is a genetic medicine company focusing on the base editing of mRNA transcripts. It discovers and develops innovative therapeutics that can utilize a family of endogenous enzymes called adenosine deaminase acting on RNA (ADAR) to precisely target and correct single point mutations on an mRNA, thus the production of desired and functional protein is restored.

Avenzo Therapeutics specializes in the development of oncology therapies for cancer treatment.

Pulmocide Ltd, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.

Rezo Therapeutics identifies targets and therapies by combining distinct, unbiased technologies with artificial intelligence. Rezo's proprietary platform can be used to treat a wide range of diseases. Rezo's initial focus is oncology, with plans to expand into other therapeutic areas through collaborations and partnerships.

MiroBio's platform is based on ground-breaking research by their scientific founders at the University of Oxford delineating how immune cells communicate and how they are activated. These insights have enabled MiroBio to create antibodies that can leverage natural signalling mechanisms to restore immune system balance and control.

Developer of antibody biotherapeutics drugs intended to develop a treatment for several life-threatening diseases. The company focuses on clinical development support for the treatment of B cell cancers, inflammatory and metabolic disorders, and chronic hepatitis B infections, enabling healthcare workers to treat patients with various life-threatening diseases.

Dren Bio is a biotechnology company with a portfolio of attractive projects focused on powerful protein engineering technologies for the depletion of cells, protein aggregates, and other disease-causing agents. It is a holding company business model aimed at creating value with multiple projects as stand-alone assets.

Mineralys Therapeutics is a biopharmaceutical company that provides hypertension treatment. They also bring a targeted approach to the management of hypertension.

Simcha Therapeutics is a biopharmaceutical company developing engineered cytokine immunotherapy for cancer. The company uses directed evolution to engineer novel cytokines designed to unlock the precision and power of the immune system. Simcha’s lead program, ST-067, is a designer IL-18 cytokine that has shown potent antitumor effects in animal models, both as a monotherapy and when combined with anti-PD-1 checkpoint inhibitors, as described in Nature in June 2020. It was founded in 2018 and is headquartered in New Haven, Connecticut, USA.

HotSpot Therapeutics is a biotech startup that focuses on the development of allosteric therapies targeting regulatory sites on proteins. It develops a therapy platform to discover medicines and provide proper treatment to identify hotspots and locate pathway treatments for cancer, metabolic and rare diseases, leverages a database of hotspot structures, harnessing the power of data science in its multidisciplinary approach to drug discovery, and helps patients to get treatment against several diseases. HotSpot Therapeutics was founded in 2017 and is headquartered in Boston, Massachusetts.

ARS Pharmaceuticals is a stealth pharmaceutical company that developed a novel intranasal epinephrine spray with a unique absorption technology that could be easy-to-use, convenient, and more reliable for patients and loved ones at risk of severe allergic reactions to food, medications and insect bites that could lead to life-threatening anaphylaxis.

eFFECTOR is a clinical stage biopharmaceutical company focused on pioneering the development of a new class of oncology drugs referred to as selective translation regulator inhibitors (STRIs). eFFECTOR’s STRI product candidates target the eIF4F complex and its activating kinase, mitogen-activated protein kinase 1/2 (MNK 1/2). The eIF4F complex is a central node where two of the most frequently mutated signaling pathways in cancer, the PI3K-AKT and RAS-MEK pathways, converge to activate the translation of select messenger RNA into proteins that are frequent culprits in key disease driving processes. Each of eFFECTOR’s product candidates is designed to act on a single protein that drives the expression of multiple functionally related proteins, including oncoproteins and immunosuppressive proteins in T cells, that together control tumor growth, survival and immune evasion. eFFECTOR’s lead product candidate, tomivosertib, is a MNK 1/2 inhibitor.  KICKSTART, a randomized, double-blind, placebo controlled Phase 2b trial of tomivorsertib in NSCLC in combination with pembrolizumab is currently open for enrollment. Zotatifin, eFFECTOR’s inhibitor of eIF4A, is currently in the dose-escalation portion of a Phase 1/2 trial, with Phase 2a expansion cohorts expected to initiate in the second half of 2021. eFFECTOR has a global collaboration with Pfizer to develop inhibitors of a third target, eIF4E.

Biotheryx is a clinical stage biopharmaceutical company discovering and developing a portfolio of innovative small molecule targeted protein degraders (TPDs) for the treatment of a wide range of diseases with an initial focus on cancer.  Members of our founding and scientific teams previously developed the first FDA approved modulators of Cereblon, the most widely validated E3 ligase involved in protein degradation, and have applied their expertise in Cereblon binding to build our proprietary PRODEGY platform. Our platform leverages an extensive patent protected and rationally designed library of Cereblon binders that we believe provides us with a differentiated starting point for drug design of molecular glues, bifunctional degraders and hybrid degraders.

Arcellx is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the forward pillars of medicine and Arcellx’s mission is to advance humanity by developing cell therapies that are safer, more effective, and more broadly accessible. Arcellx’s lead product candidate, CART-ddBCMA, is being developed for the treatment of relapsed or refractory multiple myeloma (r/r MM) in an ongoing Phase 1 study. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations by the U.S. Food and Drug Administration. Arcellx is also advancing its dosable and controllable CAR-T therapy, ARC-SparX, into the clinic through two programs: ACLX-001 in r/r MM and ACLX-002 in relapsed or refractory acute myeloid leukemia.

Design Therapeutics is a biotechnology company developing a new class of therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company’s lead program is focused on the treatment of Friedreich’s ataxia and discovery efforts are ongoing in other degenerative diseases.

River 3 Renal is a developer of novel therapies designed to target orphan diseases in the renal space.  The company's technology has the potential to address the unmet need in several kidney diseases, by further improving the standard of care, enabling medical practitioners to treat patients in a better way. River 3 Renal was established in 2020 in New York, USA.

River 2 Renal is a developer of novel therapies intended to cure orphan diseases.

F-Star Therapeutics is a biopharmaceutical company dedicated to developing novel bispecific antibody products that provide a significant improvement over the current standard of care. Given its strong patent position, it is the only biopharmaceutical company with the ability to create and develop Fcab:tm: antibody fragments, and bispecific antibodies, by modifying the constant region of an antibody. In particular, F-star’s Modular Antibody Technology:tm: enables rapid discovery and development of bispecific antibodies by introducing additional binding sites to the constant region of an antibody and offers unprecedented ease in the development and manufacturing of bispecific monoclonal antibodies. Using the Modular Antibody Technology, F-star generates bispecific antibodies (mAb2:tm:) that possess the favourable characteristics of traditional monoclonal antibodies, without the production challenges often associated with other antibody formats. F-star is now applying its proprietary technology to the development of a pipeline of product candidates.  Since its founding in 2006 the company has secured funding and support from leading VC investors: Aescap Venture, Atlas Venture, Novo Ventures and TVM Capital; as well as from renowned strategic corporate investors: Merck Serono Ventures, MP Healthcare Venture Management and SR One. In 2013, F-star established an asset-centric vehicle structure through the formation of F-star Alpha Ltd., which included out-licensing of FS102 from F-star to F-star Alpha Ltd. In October 2014 Bristol-Myers Squibb obtained an exclusive option to buy F-star Alpha. In addition the company has alliances with Boehringer Ingelheim and Merck Serono, each covering multiple targets. In 2011, F-star was selected by FierceBiotech as one of the Fierce 15 winners. F-star currently employs over 35 people at its research site in Cambridge, UK.

Decibel Therapeutics is a developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.

Palleon Pharmaceuticals is a developer of a biotechnology platform designed to offer glycoimmune checkpoint inhibitors to treat cancer.The company's biotechnology platform integrates technologies and insights from scientific leaders all over the world in the fields of glycoscience and human immunology to create a novel approach to treating cancer by targeting multiple immune cell types, providing physicians with a wider range of rational combination therapies to treat cancer and tackle resistance to first-generation immuno-oncology agents.

Tranquis Therapeutics is discovering and developing innovative medicines with the potential to revolutionize the management of neurodegenerative and aging-related diseases and to dramatically reduce the burden these illnesses place on patients, families and societies worldwide. Founded on groundbreaking neuro-immunology research from the laboratory of Professor Edgar Engleman, MD, at Stanford University, Tranquis’ novel therapeutic approach targets underlying myeloid immune cell dysfunction that has been linked to a variety of nervous system disorders. The company’s portfolio is comprised of small molecules which cross the blood-brain barrier, and the lead therapeutic candidate has demonstrated highly encouraging effects in challenging preclinical models of Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), Parkinson’s Disease (PD), and age-related cognitive impairment. Tranquis plans to initiate development in orphan neurodegenerative diseases such as ALS and FTD and later expand into more prevalent diseases such as PD and Alzheimer’s Disease.

Pandion Therapeutics is a biotechnology company developing bispecific antibody therapeutics to achieve localized immunomodulation at the site of disease for durable, tissue-specific treatment of patients with autoimmune and inflammatory diseases, and organ transplants. Pandion’s approach has the potential to more effectively induce and sustain response and remission in patients with many autoimmune and inflammatory conditions. They aim to shift the paradigm and create a new generation of drugs with transformative efficacy and improved safety.

Turning Point Therapeutics is a clinical-stage structure-based drug design company for the discovery and development of precision medicines for cancer and other diseases.  They focus on the design of novel chemical identities for established oncogene drivers with secondary resistant mutations, newly identified disease-driven targets, and potential targets regulating the tumor microenvironment and tumor immunity.

Gotham Therapeutics is discovering a novel drug class targeting the epitranscriptomics machinery.The company offers treatment options for patients suffering from cancers, auto-immune and neurodegenerative diseases.

Morphic Therapeutic is a biotechnology company that develops oral integrin therapies to treat serious chronic diseases, including autoimmune, cardiovascular, and metabolic diseases, fibrosis, and cancer. The company offers the development of therapies for patients with immunological, fibrotic, neoplastic, and vascular diseases, focusing on creating small molecule inhibitors of various integrin targets.

Propeller Health is a mobile platform that offers sensors, mobile apps, analytics, and services to support respiratory health management. The HIPAA compliant platform stores users’ data in a cloud computing infrastructure.   The Propeller sensor and mobile application help users learn more about and better manage their asthma, COPD, and respiratory disease. The Propeller sensor attaches to users’ inhalers and wirelessly syncs with their smartphones. It tracks their triggers and symptoms and sends personalized feedback and education to their phones based on the symptoms. Users can see how often they use the medication, and set up medication reminders and alerts.   Propeller community enables users to share their Propeller data with their physician and family members.   Propeller Health is backed by Safeguard Scientifics, The Social+Capital Partnership, California HealthCare Foundation, and other investors.  The platform works with organizations such as Dignity Health, Wyckoff Heights Medical Center, Amerigroup Florida, and the City of Louisville. The system has been featured in The Economist, Washington Post, Fast Company, Wired, and Scientific American.  Propeller Health has received TEDMED Innovation Showcase, White House Champion of Change, and Bluetooth Breakthrough Product awards.  Propeller Health was launched by David Van Sickle, Greg Tracy, and Mark Gehring and its operations are based in Wisconsin, United States.

Constellation Pharmaceuticals is the first biopharmaceutical company dedicated to the development of novel therapeutics in the emerging field of Epigenetics, a new field of science which is focused on selective regulators of epigenetic function, which are critical to controlling gene expression.

Zikani Therapeutics are among the most widely used, most effective, and safest antibiotics to reach clinical practice; in fact, azithromycin has been among the world’s most widely used antibiotics for the past several years. But, while current macrolides are highly effective against some major bacteria, they are ineffective against others, especially gram-negative bacteria, the cause of most serious infections in the US.  The first three generations of macrolides have very limited activity against gram-negative, and the sole drug in the current fourth-generation, solithromycin, has only slightly greater activity. Within other antibiotic classes, a limited number of compounds are in development to treat resistant gram-negative infections, and even fewer are available both intravenously and orally, as would be expected with macrolides. The company was founded in 2014 and is headquartered in Newton, Massachusetts.

Atox Bio is a biotechnology company that specializes in infectious diseases, immunomodulators, and critical care.It focuses on novel modulators of the immune response that target key multiple and interrelated pathways of both the adaptive and innate immune response that play critical roles in infectious and inflammatory diseases as well as ischemia/reperfusion injuries.  The company was founded in 2003 and headquartered in HaMerkaz, Israel.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines which are referred to as Bicycles. The Bicycles are synthetic short peptides constrained to form two loops which stabilize their structural geometry. Its initial internal programs are focused on oncology indications with high unmet medical need. The company's product candidate, BT1718, is a Bicycle Toxin Conjugate, or BTC.

Progyny (Nasdaq: PGNY) is a leading fertility benefits management company. We are redefining fertility and family building benefits, proving that a comprehensive and inclusive solution can simultaneously benefit employers, patients, and physicians.    Our benefits solution empowers patients with education and guidance from a dedicated Patient Care Advocate (PCA), provides access to a premier network of fertility specialists using the latest science and technologies, reduces healthcare costs for the nation’s leading employers, and drives optimal clinical outcomes. We envision a world where anyone who wants to have a child can do so.   Headquartered in New York City, Progyny has been recognized for its leadership and growth by CNBC Disruptor 50, Modern Healthcare’s Best Places to Work in Healthcare, Financial Times, INC. 5000, and Crain’s Fast 50 for NYC. For more information, visit www.progyny.com.

ZappRx is a digital health company that is modernizing and simplifying the way specialty drugs are managed. We provide a secure, collaborative online platform that allows patients, physicians, and pharmacists to work together to improve patients’ access to treatment and deliver medicine faster.

Spero Therapeutics is developing first in class therapeutics for treatment of gram-negative infections. The company leverages a top-tier chemistry and microbiology team, an efficient virtual approach to drug development, and a focus on high potential, novel mechanisms to fill the unmet need for early stage therapeutics targeting serious bacterial infections.  Its lead program addresses a novel target driving virulence and persistence of pseudomonas aeruginosa infections and other gram-negative pathogens.

Translate Bio is a biotechnology company that in biotechnology, RNA therapeutics, and rare diseases. Translate Bio is a therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction.

True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.

Formerly  Known as Vasculox. Arch Oncology is an immuno-oncology company developing anti-CD47 antibodies for the treatment of solid and hematologic cancers. The company's functionally diverse antibodies represent a new class of checkpoint inhibitors that harness both the adaptive and innate immune responses.

Second Genome’s mission is to transform lives with medicines developed through innovative microbiome science. Second Genome has built a novel platform for microbiome drug discovery. Second Genome has completed more than 400 microbiome studies, analyzing more than 75,000 samples, for internal R&D, as well as for external partners across government, academia, pharmaceutical, nutrition and industrial companies. The team leverages its microbiome analysis platform with its partners’ specific expertise to generate insightful findings that can accelerate research programs by elucidating the role of the microbiome in human health conditions, agriculture, animal health and other industries. The company has established a pipeline of therapeutic products for the treatment of inflammation and metabolic diseases.

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.

Puridify conducts research and development activities on bioprocessing technologies aimed at advancing industrial biomolecule manufacture. The company was founded by Tom Haywood, Oliver Hardick, and Iwan Roberts in March 2013 and is based in United Kingdom.

Principia Biopharma is a private, pre-clinical stage company initially focused on the discovery and development of differentiated small molecule drugs targeting inflammatory/autoimmune diseases as well as cancer. The company expects to submit an IND for its lead program in 2013 and continues to invest in additional programs and its reversible covalent platform.

Gladius Pharmaceuticals Inc., a Montréal-based biotechnology firm developing novel drugs to treat life-threatening, multi-drug-resistant bacterial infections.Gladius is a disruptive platform technology play, capable of developing multiple novel drug candidates

Akamis Bio is a clinical-stage oncology company whose mission is to use its groundbreaking Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform to improve the lives of cancer patients. To accomplish this goal, the company is developing a portfolio of solid tumor-targeted T-SIGn® therapeutics that will allow a patient's immune system to recognize, attack, and clear their cancer.

Thrasos is a bio-therapeutics company focused on the discovery and development of targeted therapies for the prevention and treatment of severe organ failure, with a principle focus on kidney disease. The Company's lead therapeutic program, THR-184, is focused on the prevention and treatment of acute kidney injury (AKI). The Company also has a preclinical development program for treating chronic kidney disease (CKD).

SR One is the corporate venture capital arm of GlaxoSmithKline. The firm invests globally in emerging life science companies that are pursuing innovative science which will significantly impact medical care. Since 1985, SR One has invested approximately 800 million in more than 180 companies.

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