Digitalis is a venture capital firm that that invests in solutions to complex problems in human and animal health.  Digitalis partners with entrepreneurs, inventors, and scientists across all stages of venture investing, and our investments span the full continuum of the healthcare ecosystem. It was founded in 2016 and is Headquartered in New York, New York with regional offices in Boston, Los Angeles, and San Francisco.

Digitalis Ventures

Wilson Sonsini Goodrich & Rosati is the premier legal advisor to technology, life sciences, and other growth enterprises worldwide. They represent companies at every stage of development, from entrepreneurial start-ups to multibillion-dollar global corporations, as well as the venture firms, private equity firms, and investment banks that finance and advise them. The firm's broad range of services and practice areas are focused on addressing the principal challenges faced by the management, boards of directors, shareholders, and in-house counsel of their clients.  The firm is nationally recognized for providing high-quality services to address the legal solutions required by its enterprise and financial institution clients. Their services include corporate law and governance, public and private offerings of equity and debt securities, mergers and acquisitions, securities class action litigation, intellectual property litigation, antitrust counseling and litigation, joint ventures and strategic alliances, technology licensing and other intellectual property transactions, tax, and employee benefits and employment law, among other areas. Their distinguished international roster of clients span a wide variety of industries, including information technology, life sciences, energy and clean technology, media and entertainment, communications, retail, and financial services.

Wilson Sonsini Goodrich & Rosati

Innovation Endeavors is an early-stage venture capital firm that invests in cutting-edge technologies to drive generational change in order to solve problems that matter. The firm’s central investment thesis, the Super Evolution, combines data, computational capacity, and advanced engineering to transform a range of industries including engineering health, climate, computing infrastructure, intelligent software, the physical economy, and more. Its investments include Afresh, AlphaSense, Atom Computing, Color, Gatik, Eikon Therapeutics, Fabric, Homelight, Kong, Formlabs, Panther, Planet, Replica, SoFi, Uber, Upstart, Vicarious Surgical, and more. For more information, visit www.InnovationEndeavors.com.

Innovation Endeavors

Rising Tide their technological advancements in big data analytics, computer-aided design, optical sensors, mobile applications in vertical markets, community-connected networks, and services.

Rising Tide

Heritage Provider Network (HPN) is a limited Knox-Keene licensed organization in California that provides affordable quality care to its members. Through innovative programs and services designed specifically for the managed care environment, HPN and its medical groups have been successful in delivering the goals it set out to achieve while establishing long-standing, favorable provider relationships.

Heritage Provider Network

The OS Fund finances and supports inventors and scientists who are working on audacious breakthroughs to solve the greatest issues and opportunities facing humanity today. Our mission is to partner with tech pioneers who aim to improve the lives of billions of people around the world for generations to come.  In the same way that computers have operating systems at their core — dictating the way a computer works and serving as a foundation upon which all applications are built — everything in life has an operating system (OS).

OS Fund

Pillar VC partners with unstoppable founders from the moment of inception, providing the first capital. The firm is committed to helping founders grow with their companies, investing in their personal and professional development with connections, coaching, and community. From university spinouts led by first-time founders to serial entrepreneurs, backing the next generation of big, meaningful companies in Boston. Pillar VC is open to high-potential ventures of any type, with the bulk of our portfolio related to machine intelligence, AI, hardware, enterprise software, blockchain, quantum, digital health, and synthetic biology.

Pillar VC

Dreamers VC they invest in early stage companies through the application of emerging technology in seed, A, or B rounds.

Dreamers VC

Green D Ventures is a community of accredited Dartmouth alums who pool money into a VC fund to invest in Dartmouth-connected companies. Simply put, they believe they can invest better together than they can alone. They create a diverse portfolio for their investors, co-investing with leading VC funds across industry, stage, and geography. Their goal is to raise a new fund each year that benefits entrepreneurs, the school's ecosystem, and their investors.

Green D Ventures

Redmile Group is a healthcare investment organization that makes venture, growth, and crossover investing across healthcare.

Redmile Group

Leaps by Bayer invests in paradigm-shifting advances in the life sciences and specializes in the fields of biotechnology, agriculture, and scientific breakthrough. The firm's approach is making significant and sustained investments in disruptive biotechnologies that could have the greatest impact on humanity. It was founded in 2015 and headquartered in Leverkusen, Nordrhein-Westfalen.

Leaps by Bayer

Alumni Ventures Group provides high-quality, diversified venture portfolios to individual investors who previously haven't had access to VC. It helps accredited alums from top entrepreneurial schools invest together in the ventures of fellow alums. The firm backs companies with an alumni connection and an institutional lead investor with expertise in the space. AVG also offers focused funds that allow any accredited investor access to a venture portfolio, diversified by type, sector, stage, and geography. Beth Obermiller, Michael Madden, and Mike Collins co-founded the firm in Manchester, New Hampshire in 2013.

Alumni Ventures

Atlas Venture is an early-stage venture capital firm that creates and invests in biotech startup companies led by promising entrepreneurs focusing on life sciences innovation. Its seed-led venture creation strategy rigorously selects and focuses investment on compelling opportunities to build scalable businesses and realize value.

Atlas Venture

Access Biotechnology is the life science investment arm of Access Industries. They invest in transformative therapies for conditions with high unmet needs and that have the potential to meaningfully impact human health. They invest in both private and public biopharma companies across therapeutic areas, modalities, and development stages – from discovery-stage technology platforms to late-stage clinical programs. The company is based in New York.

Access Biotechnology

GRO Biosciences

GRO Biosciences is leveraging breakthrough technologies from computational protein design and synthetic biology to develop best-in-class protein therapeutics with enhanced properties. The company has established multiple genomically recoded strains of bacteria that can incorporate non-standard amino acids into proteins to confer increased potency, stability, and targeted delivery.  GRO Biosciences is applying its technology platform to develop improved protein therapeutics for diabetes, growth disorders, and autoimmunity.

GRO Biosciences (GRObio) is a biotechnology company focused on developing protein therapeutics with enhanced properties. The company leverages breakthrough technologies from computational protein design and synthetic biology to develop best-in-class protein therapeutics. GRObio is utilizing genomically recoded organisms (GROs) to expand beyond the 20 natural amino acids typically found in proteins and introduce non-standard amino acids (NSAAs) that can customize the shape and chemical properties of proteins.

GRO Biosciences to Present at 2022 BIO CEO & Investor Conference

GRO Biosciences Appoints Rakhshita Dhar to its Board of Directors

Protein Tx Startup Cofounded by George Church Raises $2.1M in Seed Funding

GRO Biosciences Presents Preclinical Data on Two ProGly™ Programs for Non-Standard Amino Acid Therapies

GRO Biosciences CEO, Daniel J. Mandell, PhD, Awarded 2023 Termeer Fellowship

GRO Biosciences Appoints Rakhshita Dhar to Board

GRO-ing the Alphabet: George Church Spinout Engineers Bespoke Protein Therapeutics

Targeting complex diseases with complex molecules

Large-molecule Therapeutics

GRO Biosciences (GRObio) is a biotechnology company leveraging synthetic biology to expand the amino acid alphabet and develop enhanced protein therapeutics. Founded in 2016 and headquartered in Cambridge, Massachusetts, GRObio utilizes genomically recoded organisms (GROs) to incorporate non-standard amino acids (NSAAs) into proteins, enabling capabilities not achievable with conventional amino acids. The company's flagship technology, ProGly, incorporates glycan-containing NSAAs to precisely control immune responses to proteins. GRObio's lead program, ProGly-Uricase, is being developed for severe, refractory gout. This proprietary uricase enzyme incorporates ProGly NSAAs to prevent the emergence of anti-drug antibodies, potentially enabling long-term control of serum uric acid levels in patients who do not respond to existing therapies. As of July 2024, GRObio had raised over USD 90 million in funding to advance its pipeline and expand its GRO platform for scalable production of therapeutics with multiple NSAAs. The company is also exploring applications of its technology in treating autoimmune diseases without broad immunosuppression by inducing antigen-specific tolerance.

GRO Biosciences appoints Rakhshita Dhar to its Board of Directors

GRO Biosciences Closes $60.3M Series B Financing

GRO Biosciences raises $60.3m to advance amino acid-based therapeutics

GRO Biosciences Closes $60M Series B Financing Co-Led by Atlas Venture and Access Biotechnology

GRO Biosciences raises Series B funds to advance gout treatment

Aiming to Top Amgen in Gout, Startup GRObio Gets $60M to Enter the Clinic

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

Tissue Targeting Therapeutics

GRO Biosciences (GRObio) is a biotechnology company leveraging synthetic biology to expand the amino acid alphabet and develop innovative protein therapeutics. Its core technology is based on genomically re-coded organisms (GROs) that enable the incorporation of non-standard amino acids (NSAAs) into proteins. The company's platform allows the precise placement of NSAAs within proteins to achieve previously unattainable therapeutic capabilities.&nbsp;

Leveraging personal data for custom-fit diagnosis and treatment

Precision Medicine

GRO Biosciences Closes $60M Series B

GRO Biosciences (GRObio) is a biotechnology company focused on developing protein therapeutics with enhanced properties. The company leverages breakthrough technologies from computational protein design and synthetic biology to develop best-in-class protein therapeutics. GRObio is utilizing genomically recoded organisms (GROs) to expand beyond the 20 natural amino acids typically found in proteins and introduce non-standard amino acids (NSAAs) that can customize the shape and chemical properties of proteins.
GRObio's platform allows for the systematic alteration of the genetic code to incorporate NSAAs into proteins. This approach enables the company to address fundamental limitations of protein therapeutics, such as instability, immunogenicity, and delivery challenges. GRObio has developed two primary platform chemistries: DuraLogic, designed to enhance protein stability and enable flatter pharmacodynamic profiles with relaxed dosing schedules, and ProGly (programmable glycosylation), designed to enable precise regulation of the immune system's response to protein therapeutics.
In September 2023, GRObio presented preclinical data demonstrating the potential of its ProGly platform for reversing autoimmune diseases like myasthenia gravis without systemic immunosuppression and preventing immunogenic responses to protein therapeutics. The company has constructed a proprietary biofoundry with computational protein design and robotics capabilities to streamline the development of NSAAs and NSAA protein products.
GRObio was co-founded by Dr. George Church of Harvard Medical School in 2016 and is headquartered in Cambridge, MA. In November 2021, the company raised USD 25 million in Series A financing co-led by Leaps by Bayer and Redmile Group.

Innovent Biologics is a leading biopharmaceutical company founded in 2011 with the mission to develop and commercialize innovative and affordable medicines. The company focuses on oncology, cardiovascular and metabolic diseases, autoimmune disorders, and ophthalmology. As of March 2024, Innovent has ten approved products in the Chinese market, including TYVYT (sintilimab injection), BYVASDA (bevacizumab injection), SULINNO (adalimumab injection), and others. The company's core product, TYVYT, is an anti-PD-1 monoclonal antibody approved across seven indications, making it the most widely approved PD-1 inhibitor in China.
Innovent has a robust pipeline with three new drug applications under review, four assets in Phase III or pivotal trials, and 19 molecules in early clinical development. The company's innovative pipeline spans various modalities, including bispecific antibodies, antibody-drug conjugates (ADCs), and RNA therapeutics. In oncology, Innovent is advancing the next-generation immunotherapy combinations with ADCs, such as the CLDN18.2 ADC (IBI343) and the EGFR/B7H3 bispecific ADC (IBI3001). The company's metabolic pipeline includes mazdutide, a first-in-class GLP-1R/GCGR dual agonist in late-stage development for weight management.
Innovent has a strong emphasis on global innovation, with its Innovent Academy delivering eight novel molecules into IND-enabling stage in 2023. The company has established large-scale manufacturing facilities in Suzhou and Hangzhou to support global supply. Innovent collaborates with over 30 global healthcare leaders, reinforcing its commitment to advancing biopharmaceutical innovation worldwide.

Innovent Biologics

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 ADC Therapeutics is a clinical-stage biotechnology company focused on developing and commercializing next-generation antibody-drug conjugates (ADCs) for treating cancer. The company's ADCs leverage highly potent and targeted pyrrolobenzodiazepine (PBD) dimer technology, which causes the ADCs' antibodies to bind to specific tumor cell surface antigens. Once internalized, the potent PBD dimer is released inside the cell, creating covalent cross-links between the DNA strands, disrupting cell division without triggering DNA repair mechanisms.
 
 
 
 
 ADC Therapeutics' lead product candidate, Zynlonta, received FDA approval in April 2021 for treating adult patients with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy. The company is also advancing a deep pipeline of other PBD-based ADCs targeting hematologic malignancies and solid tumors. As of May 2021, ADC Therapeutics reported revenues of USD 38.2 million, reflecting initial sales of Zynlonta.
 
 
 
 
 In March 2022, ADC Therapeutics acquired US commercial rights to Overrkey Biological's anti-HER2 ADC Overena, broadening its cancer treatment portfolio. The acquisition aligned with the company's strategic focus on expanding globally with its PBD-based ADC technology.
 
</div>

ADC Therapeutics

Sirnaomics is a biopharmaceutical company engaged in the discovery and development of RNA therapeutics. The company focuses on innovative drugs for indications with unmet medical needs and large market opportunities. Sirnaomics utilizes two proprietary delivery platforms: the Polypeptide Nanoparticle Formulation (PNP) and the second generation of GalNAc conjugates called GalAhead.
Sirnaomics has established a robust pipeline of drug candidates, with several products in clinical and preclinical stages. The company's lead clinical product, STP705, has demonstrated positive results in Phase II clinical trials for the treatment of squamous cell carcinoma in situ (isSCC) and basal cell carcinoma (BCC). Additionally, STP705 is being explored for focal fat reduction in the medical aesthetics field. Another promising candidate, STP707, is a PNP formulation consisting of two siRNAs targeting TGF-β1 and COX-2 for intravenous treatment of solid tumors. STP707 has shown promising results in a Phase I basket clinical study, exhibiting stable disease response in 74% of evaluable patients.
Sirnaomics achieved a significant milestone by advancing STP122G, the first candidate utilizing the GalAhead technology, into clinical development. STP122G targets Factor XI for anticoagulation therapeutics and is currently undergoing Phase I clinical trials.
In March 2023, Sirnaomics' subsidiary, RNAimmune, received IND clearance from the FDA to initiate a Phase I clinical trial for RV-1770, an mRNA vaccine targeting respiratory syncytial virus (RSV). Additionally, RNAimmune obtained FDA clearance for a SARS-CoV-2 vaccine booster candidate, RV-1730.
Sirnaomics has established its manufacturing facility in China, enabling the transition from a biotech company to a biopharmaceutical corporation. The facility has successfully produced a full GMP batch of STP707 for human injection and is expected to support the company's clinical pipeline.
In July 2024, Sirnaomics announced the completion of IND-enabling studies for STP125G, an siRNA therapeutic targeting Apolipoprotein C3 (ApoC3) for the treatment of cardiovascular diseases. The positive results from non-human primate studies support the filing of an IND with the FDA.

Sirnaomics

Sesen Bio is a late-stage clinical company that previously focused on developing targeted fusion protein therapeutics for cancer treatment. Its lead product candidate, Vicineum, is a locally administered targeted fusion protein composed of an anti-epithelial cell adhesion molecule (EpCAM) antibody fragment tethered to a truncated form of Pseudomonas exotoxin A for the treatment of non-muscle invasive bladder cancer (NMIBC).
In February 2021, the FDA accepted Sesen Bio's Biologics License Application (BLA) for Vicineum to treat BCG-unresponsive NMIBC, granting Priority Review. However, in August 2021, the company received a Complete Response Letter from the FDA, requiring an additional Phase 3 clinical trial for potential resubmission. Following discussions with the FDA in March 2022, Sesen Bio aligned on most elements for the trial design and planned to meet again in mid-2022 to finalize outstanding items.
On July 15, 2022, Sesen Bio made the strategic decision to voluntarily pause further development of Vicineum in the US due to the incremental timeline and costs associated with the additional Phase 3 trial. The company shifted its primary focus to consummating a strategic transaction to maximize shareholder value while seeking a partner for Vicineum's further development.
In September 2022, Sesen Bio announced a pending merger with Carisma Therapeutics, a clinical-stage biopharmaceutical company developing immunotherapies based on engineered macrophages. This merger was approved by Sesen Bio's stockholders in March 2023, with the combined company operating under the name Carisma Therapeutics Inc. and trading on the Nasdaq under the ticker "CARM".
Upon closing the merger in March 2023, Sesen Bio declared a special cash dividend of USD 75 million (around USD 0.36 per share) to its stockholders and issued them Contingent Value Rights (CVRs) entitling them to potential proceeds from the sale of Vicineum and other assets, as well as a USD 30 million milestone payment under the Roche Asset Purchase Agreement.

Sesen Bio

ACELYRIN, INC. is a late-stage clinical biopharmaceutical company focused on developing transformative medicines for immunology and inflammatory diseases. The company's lead product candidate is izokibep, a next-generation inhibitor of interleukin-17A (IL-17A) designed to overcome limitations of monoclonal antibodies. Izokibep is being evaluated in multiple late-stage trials for moderate-to-severe hidradenitis suppurativa (HS), psoriatic arthritis (PsA), and noninfectious uveitis.
In March 2024, ACELYRIN reported positive top line data from a global Phase 2 billion/3 clinical trial of izokibep in PsA, meeting the primary endpoint of ACR50 at 16 weeks with high statistical significance. The trial also demonstrated robust clinical responses for higher hurdle endpoints like ACR70, PASI100, and composite measures, indicating izokibep's potential to provide clinically meaningful benefits. Additionally, ACELYRIN announced differentiated long-term 32-week data from a global Phase 2 billion trial of izokibep in HS, showing rapid, dose-ordered improvement across multiple disease manifestations, including HiSCR100 achieved earlier than reported by other IL-17 inhibitors.
ACELYRIN's other late-stage asset is lonigutamab, a subcutaneously delivered monoclonal antibody targeting IGF-1R being investigated for the treatment of thyroid eye disease (TED). In March 2024, ACELYRIN announced positive proof-of-concept data from a Phase 1/2 trial, demonstrating rapid improvements in proptosis and clinical activity score within three weeks after the first dose.
As of December 31, 2023, ACELYRIN had cash, cash equivalents, and short-term marketable securities totaling USD 721.3 million, providing a runway to support key late-stage data milestones and registrational studies across multiple indications. In January 2023, ACELYRIN acquired ValenzaBio, adding lonigutamab and other immunology assets to its pipeline.

ACELYRIN

Companies that develop engineered proteins that can target specific proteins or cells in the body

Companies that develop large molecules that can be used to target specific RNA molecules in the body, either to silence or replace them

Companies that develop large molecules that combine the targeting ability of monoclonal antibodies with the cytotoxic effects of chemotherapy drugs

Companies that develop large molecules that can be used to replace or supplement deficient or malfunctioning proteins in the body

Companies specializing in peptide-based treatments leverage short amino acid chains, known as peptides, to target and modulate various biological processes for therapeutic applications

AL/ML used to identify potential drug targets, and support protein engineering and optimization

GRO Biosciences (GRObio) is a biotechnology company leveraging synthetic biology to expand the amino acid alphabet and develop enhanced protein therapeutics. Founded in 2016 and headquartered in Cambridge, Massachusetts, GRObio utilizes genomically recoded organisms (GROs) to incorporate non-standard amino acids (NSAAs) into proteins, enabling capabilities not achievable with conventional amino acids. The company's flagship technology, ProGly, incorporates glycan-containing NSAAs to precisely control immune responses to proteins. GRObio's lead program, ProGly-Uricase, is being developed for severe, refractory gout. This proprietary uricase enzyme incorporates ProGly NSAAs to prevent the emergence of anti-drug antibodies, potentially enabling long-term control of serum uric acid levels in patients who do not respond to existing therapies. As of July 2024, GRObio had raised over USD 90 million in funding to advance its pipeline and expand its GRO platform for scalable production of therapeutics with multiple NSAAs. The company is also exploring applications of its technology in treating autoimmune diseases without broad immunosuppression by inducing antigen-specific tolerance.

<div>
 
 Generation Bio is a biotechnology company developing non-viral genetic medicines for rare and prevalent diseases. The company's proprietary platform combines immune-quiet DNA (iqDNA), a novel variant of closed-ended DNA, with a cell-targeted lipid nanoparticle (ctLNP) delivery system and a rapid enzymatic synthesis (RES) manufacturing process. This platform is designed to create gene therapies with drug-like properties, offering durable gene expression, the ability to re-dose for individual patient titration, and the potential to sustain effect over a lifetime. Generation Bio's approach aims to enable multi-year durability from a single dose, deliver large genetic payloads, and allow titration and redosing to adjust or extend expression levels in each patient. The company's pipeline includes programs for liver diseases such as hemophilia A, phenylketonuria (PKU), and Wilson's disease, as well as eye disorders like Stargardt disease and Leber congenital amaurosis type 10. In October 2023, Generation Bio announced the development of iqDNA, which demonstrated cytokine levels and tolerability comparable to chemically modified messenger RNA in mice and non-human primates. Generation Bio's RES manufacturing process has the potential to produce ceDNA at a scale of hundreds of millions of doses.
 
 
 
 
 Key customers and partnerships
 
 
 
 
 In March 2023, Generation Bio entered into a strategic partnership with Moderna to develop non-viral genetic medicines. The collaboration combines Moderna's expertise with Generation Bio's platform technologies to expand their respective non-viral genetic medicines pipelines. Under this partnership, Moderna is using Generation Bio's ctLNP delivery system and iqDNA technology to advance two immune cell programs and two liver programs. The deal included a USD 40 million upfront cash payment and a USD 36 million equity investment from Moderna. Generation Bio will also receive future development, regulatory, and commercial milestone payments, along with royalties on worldwide net sales of immune cell-targeted and liver-targeted products. The partnership aims to develop new lipid nanoparticles and expand in vivo immune cell targeting as a new class of genetic medicines.
 
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Generation Bio

Oncopeptides AB is a biotech company focused on developing therapies for difficult-to-treat hematological diseases, particularly multiple myeloma. The company's lead product, PEPAXTO (melphalan flufenamide, also known as melflufen), received FDA approval in February 2021 for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy. PEPAXTO is the first anticancer peptide-drug conjugate approved by the FDA for this indication. The drug works by leveraging aminopeptidases, which are overexpressed in multiple myeloma cells, to release cytotoxic agents.
In August 2022, the European Commission granted marketing authorization for Pepaxti (melphalan flufenamide) in combination with dexamethasone for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies. Oncopeptides' research is based on two technology platforms: Peptide Drug Conjugates (PDC) and Small Polypeptide based Innate Killer Engagers (SPiKE). The PDC platform allows for efficient drug distribution in the body, enabling a wide therapeutic window and an optimized benefit-risk profile. The SPiKE platform utilizes the immune system to fight cancer by targeting natural killer cells, potentially reducing dose-limiting side effects while maintaining clinical efficacy.
As of 2023, Oncopeptides was conducting one clinical phase 3 study and six clinical phase 2 studies. The company's pipeline includes innovative drug candidates for difficult-to-treat hematological diseases, with the potential to expand into new indications. Oncopeptides' approach is science and data-driven, focusing on bringing innovation to patients with rare hematological diseases.

Oncopeptides

Neurona Therapeutics is a clinical-stage biotherapeutics company focused on developing regenerative cell therapy candidates for the treatment of neurological disorders. The company's lead investigational candidate, NRTX-1001, is being evaluated in an ongoing Phase I/II clinical trial for drug-resistant mesial temporal lobe epilepsy (MTLE). NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells. It consists of fully-differentiated neural cells called interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, these human interneurons are intended to integrate on-target and durably silence seizure activity in the epileptic region of the brain. In December 2023, Neurona presented early data from five participants in the ongoing study of NRTX-1001. Two patients who received the therapy more than a year prior experienced at least a 95% reduction in overall seizure frequency. NRTX-1001 is manufactured in Neurona's in-house GMP facility using proprietary methods. Beyond epilepsy, Neurona is also evaluating NRTX-1001 for potential application in Alzheimer's disease. The company's pipeline includes other cell therapy candidates such as investigational myelinating glial cells and gene-edited cells for undisclosed indications.

Neurona Therapeutics

Evox Therapeutics is a biotechnology company specializing in exosome therapeutics. The company utilizes its proprietary DeliverEX technology to engineer exosomes for drug delivery to specific organs, including the brain and central nervous system. Evox's approach aims to overcome limitations of genetic medicines such as gene therapy, gene editing, and RNA therapeutics by enabling safe, non-immunogenic repeated delivery to cells and tissues that are challenging to reach with other drug delivery technologies.
The company's technology focuses on harnessing and engineering the natural delivery capabilities of extracellular vesicles, known as exosomes, to develop a new class of therapeutics for treating severe rare diseases. Evox's intellectual property portfolio includes more than 100 granted patents across key jurisdictions, covering various aspects of exosome-based drug design, development, and manufacturing. In May 2023, Evox was granted a US patent for a method of purifying engineered exosomes using ultrafiltration and size-based separation steps, which is widely used in exosome therapeutics.
In June 2023, Evox acquired Codiak Biosciences' engEx-AAV technology platform, which enables active loading of adeno-associated virus (AAV) into exosomes. This technology has demonstrated improved AAV delivery, increased expression breadth in preclinical models, and shielding of AAVs from neutralizing antibodies. The acquisition aligns with Evox's strategy to enhance exosome-mediated delivery for genetic medicines such as gene therapy and genome editors.

Evox Therapeutics

Aro Biotherapeutics is a clinical-stage biotechnology company based in Philadelphia that specializes in developing tissue-targeted genetic medicines. The company's core technology is a proprietary protein platform called Centyrins, which enables efficient and tissue-specific delivery of siRNA and other genetic drugs. Aro's lead candidate, ABX1100, is being developed for the treatment of Pompe disease, a rare genetic disorder characterized by toxic glycogen accumulation in muscles. ABX1100 comprises a CD71 receptor-binding Centyrin conjugated to a small interfering RNA (siRNA) that targets the Gys1 gene, reducing levels and activity of the Gys1 enzyme in muscle tissues. The therapy has shown promising results in preclinical studies, demonstrating potent and muscle-specific reductions in Gys1 mRNA and protein in the Pompe mouse model. In October 2023, Aro initiated a Phase 1 clinical trial of ABX1100 in Canada, with initial data expected in 2024. The US Food and Drug Administration granted ABX1100 Orphan Drug Designation and Rare Pediatric Disease status. Beyond Pompe disease, Aro is exploring the potential of its Centyrin-siRNA conjugate programs for developing new treatments for autoimmune disorders.

Aro Biotherapeutics

Therapeutics that are designed to target specific cells to reach designated target sites

Therapeutics that are designed to target specific protein or protein receptors to reach designated target sites

Therapeutics that use different targeting approaches, such as bone-targeting or pressure-based, to help therapeutics identify specific target sites

Companies that offer contract research and manufacturing facilities that focus on tissue-targeting therapeutics for life sciences organizations

GRO Biosciences (GRObio) is a biotechnology company leveraging synthetic biology to expand the amino acid alphabet and develop innovative protein therapeutics. Its core technology is based on genomically re-coded organisms (GROs) that enable the incorporation of non-standard amino acids (NSAAs) into proteins. The company's platform allows the precise placement of NSAAs within proteins to achieve previously unattainable therapeutic capabilities.&nbsp;As of October 2024, GRObio's lead program, ProGly-Uricase, was being developed for the treatment of severe, refractory gout. This proprietary uricase enzyme incorporates the company's ProGly NSAAs to prevent the emergence of anti-drug antibodies (ADAs) and enable long-term effective control of serum uric acid levels in patients.&nbsp;The company's technology portfolio includes two key platforms: DuraLogic and ProGly. DuraLogic integrates NSAAs to enhance and maintain the three-dimensional structure of proteins, potentially improving their resistance to degradation and increasing their half-life. ProGly comprises glycan-containing NSAAs designed to induce or inhibit specific immune reactions. In September 2023, GRObio presented data claiming that its ProGly platform produced significant improvements in an animal model of myasthenia gravis and demonstrated the ability to prevent immunogenic responses to enzyme replacement therapies.

<div>
 
 Praxis Precision Medicines is a clinical-stage biopharmaceutical company that develops precision medicine for central nervous system disorders based on neuronal imbalance caused by genetic mutations.
 
 
 The company has four clinical-stage product candidates in its pipeline for treating disorders such as depression, epilepsy, movement disorders, and pain syndromes. The company&rsquo;s drug candidates include 1) PRAX-944, which is an inhibitor for essential tremor disorder linked to Parkinson&rsquo;s disease and is set to begin Phase 3 in mid-2023, 2) PRAX-222 in Phase 2 of clinical trials targets the SCN2A gene most commonly associated with early-onset epilepsy, 3) PRAX-562 was designed to block persistent sodium for patients suffering from rare diseases such as headache disorders and severe seizures and completed Phase 1 of clinical trials, and 4) PRAX-628 in Phase 2 clinical trials for treating focal epilepsy that originates in one side or area of the brain and affects one side of the body. Additionally, the firm has six pre-clinical drug candidates.
 
 
 Funding and financials
 
 
 The company went public in October 2020, trading on the Nasdaq Global Select Market under the ticker symbol &ldquo;PRAX&rdquo;. 
 <a href="https://sp-edge.com/updates/19453">
 In June 2023 
 </a>
 , the company announced an underwritten public offering of its common shares and pre-funded warrants, to raise approximately USD 59.1 million. The capital raised was used to support the company&rsquo;s ongoing operations and strategic initiatives, furthering the development of its pipeline and potential future endeavors.
 
 
 For the year 
 <a href="https://sp-edge.com/updates/16211">
 2022
 </a>
 , net losses increased by 28% YoY to USD 214 million arising from a significant increase in R&amp;D expense (up by 29% YoY). For the six months ended June 2023, the company is yet to generate revenue from drugs, however received collaboration revenue of USD 1.5 million. Further, it reported a decline in net loss by 43.0% YoY to USD 34.3 million. Its cash and cash equivalent as of June 2023 was at USD 124.3 million, compared to USD 61.6 million in December 2022, which the company believes will be adequate to fund operations till Q1 2025. 
 
</div>

Praxis Precision Medicines

<div>
 
 Mirati Therapeutics is a commercial-stage biotechnology company developing oncology-targeted therapies to address KRAS mutations present in 25% of tumors, including lung, colorectal and pancreatic cancers. Mirati&rsquo;s lead product KRAZATI (adagrasib/ MRTX849) is an oral&nbsp; KRASG12C inhibitor approved in 2022 for certain non-small cell lung cancers.The company also has sitravatinib, a multi-kinase inhibitor, in Phase 3 trials for non-small cell lung cancer in combination with a Bristol-Myers Squibb checkpoint inhibitor.
 
 
 
 
 Additionally, Mirati is advancing multiple early-stage programs including KRAS G12D inhibitor MRTX1133 for pancreatic and colorectal cancers, which entered Phase 1 in 2023, as well as a synthetic lethal inhibitor MRTX1719. In December 2022, the company submitted an IND application for MRTX1133, a potent and selective KRAS G12D inhibitor, which was cleared by the FDA in January 2023. 
 
 
 Key partnerships and customers
 
 
 
 
 Mirati has multiple partnerships with leading biopharma companies to advance its pipeline, including discovery collaborations with Pfizer and clinical collaborations with Novartis, Boehringer Ingelheim, and 
 <a href="https://sp-edge.com/updates/14345">
 Incyte
 </a>
 . The company also has licensing agreements with BeiGene for sitravatinib and Zai Lab for MRTX849 in certain Asian markets.
 
 
 
 
 
 Funding and Financials
 
 
 
 
 The company went public in June 2013 to trade on Nasdaq under the ticker symbol &ldquo;MRTX&rdquo;. In August 2023, the company closed an 
 <a href="https://sp-edge.com/updates/20988">
 underwritten public offering
 </a>
 at USD 345 million. The new funds were used towards drug development and leadership expansion.&nbsp;
 
 
 
 
 <a href="https://sp-edge.com/updates/16725">
 For the year 2022 
 </a>
 , the firm's net losses increased to USD 740.9 million from USD 581.8 million. Net product revenue for the full year amounted to USD 0.7 million arising from the sale of KRAZATI, which received FDA approval for its commercial launch in December 2022. 
 
</div>

Mirati Therapeutics

<div>
 
 Tvardi Therapeutics is a clinical-stage biotechnology company developing precision medicine for different cancers, chronic inflammatory diseases, and fibrotic diseases. The company&rsquo;s pipeline focuses on the development of small-molecule inhibitors of STAT3, a regulatory protein that allows cancer cells to evade the immune system and survive.
 
 
 The company&rsquo;s orally administered monotherapy and lead product, TTI-101, is in Phase 1 clinical trials for patients with advanced solid tumors who have failed all other lines of therapy. Additionally, the drug is in preclinical trials for treating fibrosis and inflammation. The company is also developing TTI-102, a next-generation STAT3 inhibitor that is structurally similar but chemically different from TTI-101.&nbsp;
 
 
 Founded in 2017, the Texas-based company closed a Series B funding round in June 2021, which brought total funding to USD 81 million and the proceeds will be used to advance Tvardi&rsquo;s drug candidates through its clinical data readouts in mid-stage trials for cancer and fibrosis.
 
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Tvardi Therapeutics

<div>
 
 Tango Therapeutics is a clinical-stage biotech company developing precision oncology therapies targeting synthetic lethality in cancer which leads to cell death. Its discovery platform identifies novel targets to develop drugs focused on: 1) countering tumor suppressor gene loss to help the immune system recognize and kill cancer cells, and 2) reversing cancer cell evasion of the immune system.
 
 
 
 
 Tango has five pipeline programs, including lead candidate TNG908, an inhibitor for multiple cancer indicators. The FDA approved TNG908's IND in January 2022 and granted it Orphan Drug Designation. TNG908 is in Phase I/II trials, with preliminary efficacy data expected in H1 2023. Tango also plans to file INDs for TNG260 (STK11 mutant cancers) and TNG348 (BRCA1/2-mutant cancers). In 
 <a href="https://www.globenewswire.com/news-release/2023/01/25/2595034/0/en/Tango-Therapeutics-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-TNG462-Provides-Additional-Business-Updates.html">
 January 2023
 </a>
 , the FDA cleared the IND for TNG462, a next-gen PRMT5 inhibitor for cancers with MTAP deletion.
 
 
 
 
 Key partnerships and customers
 
 
 
 
 
 Outside its pipeline, the company also entered into a collaborative partnership with big pharma Gilead Sciences Inc., in October 2018 which was later expanded for two years in August 2020 to identify new immune evasion targets.
 
 
 
 
 Funding and financials&nbsp;
 
 
 
 
 
 Tango went public in August 2021 via a SPAC deal, trading on Nasdaq under the ticker symbol &ldquo;TNGX&rdquo;, and the company received gross proceeds of USD 353 million. In August 2023, the company raised USD 80 million via a 
 <a href="https://sp-edge.com/updates/20955">
 private placement
 </a>
 , which was led by Nextech. The new funds were used toward expanding its TNG908 and TNG462 PRMT5 programs to various other indications.
 
 
 
 
 Tango has not yet generated any drug sales and does not expect to do so for several years. However, for the full-year the company reported collaboration revenue of USD 24.9 million (down 4.42% YoY) and a net loss of USD 108.2 million (up 85.9% YoY).
 
 
 
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Tango Therapeutics

<div>
 
 IDEAYA Biosciences is a clinical-stage biotech developing precision cancer therapies based on synthetic lethality - when specific gene mutations pair to cause cell death. The company also studies combining synthetic lethality with immunotherapies and DNA damage to enhance treatment efficacy. While current drugs target limited mutations like BRCA1/2, IDEAYA aims to treat broader genetic subpopulations.&nbsp;
 
 
 
 
 Its pipeline includes synthetic lethality-driven precision medicines across four potential first-in-class clinical-stage product candidates. Of these, the company owns/controls commercial rights of the three most-advanced of these product candidates: darovasertib, IDE397, and IDE161. As of May 2024, the company was also advancing Werner Helicase program for which a development candidate has been selected in collaboration with GlaxoSmithKline, and, subject to investigational new drug-, or IND-, enabling studies, are targeting an IND in 2024. In addition. the company also works on multiple earlier-stage preclinical programs. 
 
 
 By targeting specific genetic drivers, IDEAYA advances precision oncology using synthetic lethality approaches. The company is focused on developing novel targeted therapies for patient populations with high unmet need and limited treatment options.&nbsp;&nbsp;
 
 
 In December 2023, IDEAYA Biosciences teamed up with Gilead Sciences to test the combination of IDE397 and Trodelvy in a Phase I clinical trial for MTAP-deletion bladder cancer.
 
 
 
 
 Key partnerships and customers
 
 
 
 
 The company collaborates with pharmaceutical partners to advance its pipeline, including a strategic partnership with GSK in June 2020. The collaboration with GSK covers co-development and commercialization of two synthetic lethality programs. IDEAYA received USD 100 million upfront and USD 20 million in a direct private placement through the GSK deal. In June 2022, IDEAYA announced it would co-develop its Pol Theta candidate with GSK, with a Phase 1 trial starting in the first half of 2023.&nbsp;
 
 
 
 
 IDEAYA also holds an exclusive license from Novartis for LXS196 (IDE196), a PKC inhibitor for metastatic uveal melanoma. Additionally, the company is exploring IDE196 combinations with Pfizer and its potential in other rare skin diseases. In July 2022, IDEAYA signed an agreement with Amgen to evaluate IDE397 with Amgen's AMG 193 in solid tumors. Strategic pharma partnerships help advance and expand IDEAYA's pipeline of synthetic lethality precision oncology programs.
 
 
 
 
 Funding and financials
 
 
 
 
 
 The company was listed on the Nasdaq Global Select Market under the ticker symbol &ldquo;IDYA&rdquo; in May 2019, raising USD 50 million via an initial public offering. In September 2022, the company raised USD 92 million.&nbsp;
 
 
 
 
 For 2022, the company reported a net loss of USD 58.7 million (up 17.9% YoY) while collaboration revenue was USD 50.9 million (up 82.3% YoY). 
 
 
 On October 24, 2023, IDEAYA Biosciences priced an underwritten public offering of 5 million shares of common stock and pre-funded warrants to purchase 319,150 shares of common stock. The common stock offered was priced at USD 23.50 per share, while the exercise price of a pre-funded warrant was USD 0.0001. The offering was expected to bring in approximately USD 125 million in gross proceeds.
 
 
 
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IDEAYA Biosciences

Companies providing genetic tests to identify individuals with high risk or at-risk of having hereditary disease, aid physicians with a diagnosis, and support clinical decision making.

Secure cloud-based platforms and data sharing networks that collect, index, store, and analyze large amounts of health data and patient population data sets from sources including genomic, clinical, wearables, imaging, and population data.

Biopharma and biotechnology companies that develop precision medicine drugs based on novel disease biomarkers present in multi-omic patient population data and genetic insights.

Companies that study intergenomic variations in genes associated with drug response and metabolism to match patients with the appropriate drug and dosage.

Companies that develop an in-vitro medical device or imaging tools and tests that identify genetic mutations for both diagnostic (nature of the disease) and prognostic (likely course of the disease) purposes, allowing providers to match a patient to a specific drug or therapy.

Software analytics platforms that integrate multiple omics datasets (genomics, transcriptomics, proteomics, and metabolomics) to gain insights and support target discovery, drug development, clinical trials, and diagnostics.

Some companies leverage AI/ML to develop tailored medication

Considers individual variability in genes, environment, and lifestyle for each person

Genomics is used across all the segments of precision medicine from diagnostic solutions to treatments

AI/ML analyzes genomic data to find drug targets, predict disease progression, and treatment response

GRO Biosciences

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