Invus Financial Advisors

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Bambusa Therapeutics is a biotechnology company dedicated to developing bispecific antibodies for treating immunological and inflammatory disorders. It focuses on building a diverse portfolio of molecules and leveraging antibody development capabilities to provide targeted therapies.

Coave Therapeutics specializes in developing gene therapies for rare ocular and central nervous system (CNS) diseases. Its platform enables targeted delivery of therapeutic genes to the CNS, enhancing autophagy and lysosomal function, with a focus on improving patient outcomes for conditions like ALS.

Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.

Ottimo Pharma is a biopharmaceutical company dedicated to developing innovative cancer therapies for solid tumors. Its primary focus is Jankistomig, a bi-functional antibody that simultaneously targets two critical pathways in cancer growth: immune checkpoint inhibition and angiogenesis. By addressing these pathways concurrently, Ottimo Pharma aims to improve treatment outcomes and reduce the burden of cancer on patients and the healthcare system.

Founded in 2019, Noema Pharma is a biotechnology company based in Basel, Switzerland. It specializes in developing innovative therapies for orphan neurological disorders characterized by imbalanced neuronal networks, aiming to improve patient outcomes and quality of life.

Tasca is a biotechnology company that offers small molecule drugs for hard-to-treat oncology indications.

AgomAb Therapeutics N.V., based in Gent, Belgium, specializes in the development of molecular therapies aimed at regenerating damaged tissues. The company focuses on creating agonistic monoclonal antibodies, known as agomAbs, which are designed to stimulate molecular and cellular repair mechanisms. These therapies have the potential to restore organ function in patients suffering from fibrotic, inflammatory, autoimmune, and degenerative diseases. AgomAb targets biologically validated pathways, including Transforming Growth Factor β and Hepatocyte Growth Factor, while employing specialized capabilities in organ-specific small molecules and high-affinity antibodies. The company boasts a diversified clinical pipeline addressing various fibrotic conditions and possesses comprehensive research and development expertise, alongside a strong track record in business development.

Seaport Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel neuropsychiatric medicines in areas of high patient need. Its innovative approach aims to improve oral bioavailability, thereby reducing side effects such as hepatotoxicity and enhancing patients' quality of life with neuropsychiatric disorders.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

City Therapeutics is a biopharmaceutical company specializing in the development of next-generation small-interfering RNAs (siRNAs) to enhance RNA interference (RNAi) therapies. The company's pipeline comprises innovative RNAi therapeutics targeting diverse disease indications. City Therapeutics aims to improve and expand RNAi technology to achieve better patient outcomes, led by a team of experienced scientists and industry professionals committed to advancing the field of RNA interference.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Egetis Therapeutics AB is a pharmaceutical drug development company based in Stockholm, Sweden, that specializes in late-stage projects targeting serious rare diseases with significant unmet medical needs, particularly within the orphan drug segment. The company is actively developing several innovative therapies, including PledOx, which is undergoing Phase III clinical trials to protect patients from chemotherapy-induced nerve damage while preserving the anticancer effects. Additionally, Egetis is working on Emcitate, a treatment for MCT8 deficiency, a rare genetic condition affecting thyroid hormone signaling, and Aladote, which aims to mitigate the risk of acute liver injury from acetaminophen poisoning. Founded in 2006, Egetis Therapeutics was previously known as PledPharma AB until its name change in December 2020.

Autobahn Therapeutics develops brain-targeting, small molecule therapies for central nervous system disorders. Its lead program ABX-002 is a thyroid hormone receptor beta agonist being developed as an adjunctive treatment for mood disorders, including major depressive disorder and bipolar depression. The company emphasizes precision tuning of CNS exposure, validated clinical and biologic targets, and biomarker-guided development across neuropsychiatry, neurodegeneration, and neuroinflammation to address high unmet medical needs and improve patient outcomes.

Cardurion Pharmaceuticals, LLC, established in 2017 and headquartered in Boston, Massachusetts, is a biotechnology company dedicated to developing innovative therapies for heart failure and other cardiovascular diseases. The company is advancing clinical programs that target PDE9 and CaMKII inhibition, utilizing its expertise in cardiovascular signaling pathways to address significant unmet patient needs.

Cartesian Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene immunotherapies to treat cancer and autoimmune diseases. The company designs chimeric antigen receptor therapies for multiple myeloma and other cancers and uses its ImmTOR immune tolerance platform to mitigate antigen-specific immune responses. It operates a proprietary technology and manufacturing platform to deliver one or more mRNA molecules into cells to enhance their function, aiming to improve therapeutic performance.

Bright Peak Therapeutics is a biotechnology company focused on developing a range of immunotherapies aimed at treating cancer and autoimmune diseases. The company specializes in creating immuno-cytokines that possess pharmacological properties enabling tissue and cell-specific targeting of cytokine payloads. This innovative approach is designed to enhance the efficacy of treatments, ultimately improving patient outcomes and quality of life. By leveraging its expertise in cytokine therapeutics, Bright Peak aims to advance the field of immunotherapy and provide effective solutions for challenging medical conditions.

Lycia Therapeutics operates as a biotechnology company focused on discovering and developing first-in-class therapeutics using its proprietary Lysosomal Targeting Chimeras (LYTACs) platform. This platform enables the degradation of extracellular and membrane-bound proteins associated with challenging-to-treat diseases like cancer and autoimmune disorders, aiming to provide effective treatments for patients.

Cullinan Therapeutics is a developer of an externally sourced cancer therapeutics intended to end a drug program quickly if the early research suggests it won't work. The company's therapeutics are developed via assets sourced internally through dry lab or externally through business, academic and pharma collaborations, enabling researchers to get a highly diversified portfolio of oncology therapeutics with a unique, cost-efficient business model.

Alterome Therapeutics is a precision oncology biotechnology company focused on developing alteration-specific therapeutics to target high-value, validated oncogenic drivers. It employs structure-guided drug discovery and advanced computational chemistry to create small-molecule therapies that selectively address oncogenic alterations, with the aim of improving patient outcomes in cancer.

Lexicon Pharmaceuticals is a biopharmaceutical company focused on discovering and developing drugs to treat human diseases. Its pipeline includes candidates for irritable bowel syndrome, type 2 diabetes, rheumatoid arthritis, carcinoid syndrome, and glaucoma. The company has collaborations with major pharmaceutical companies and other organizations.

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

GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Harpoon Therapeutics, Inc. is a clinical-stage immunotherapy company based in South San Francisco, California, focused on developing a novel class of T cell engagers to harness the body's immune system for treating cancer and other diseases. The company utilizes its proprietary TriTAC (Tri-specific T cell Activating Construct) platform to create engineered proteins that direct T cells to target and kill cells expressing specific antigens. Harpoon's lead product candidate, HPN424, is currently undergoing Phase I clinical trials for metastatic castration-resistant prostate cancer. Additionally, the company is advancing other candidates, including HPN536, which is in Phase I/IIa trials for ovarian cancer and MSLN-expressing tumors, as well as HPN217 for multiple myeloma and HPN328 for small cell lung cancer. Harpoon Therapeutics was founded in 2015 and is committed to addressing unmet medical needs in oncology through innovative therapies.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

ADARx Pharmaceuticals specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

Tisento Therapeutics, based in Massachusetts, specializes in developing innovative medicines to address diseases with substantial unmet medical needs. Its primary focus is on Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke (MELAS), a severe mitochondrial disorder. The company's therapies aim to treat mitochondrial diseases, both in the central nervous system and peripherally, by targeting key aspects of the disease's pathophysiology, including neuronal and mitochondrial function, cerebrovascular hemodynamics, and inflammatory processes. This approach enables patients to receive treatment promptly.

Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that specializes in the discovery, development, and commercialization of innovative therapeutic products targeting significant unmet medical needs. Founded by former executives from Receptos, the company focuses on areas such as immunology, inflammation, fibrosis, and oncology. Its pipeline includes several promising candidates: Seralutinib, aimed at addressing pulmonary arterial hypertension; GB004, for inflammatory bowel disease; GB1275, targeting various oncology indications; and GB001, designed for moderate-to-severe eosinophilic asthma. Gossamer Bio's strategy involves leveraging a rich in-licensing environment to enhance its therapeutic offerings, supported by a team recognized for their successful track record in drug development.

Septerna is a biotechnology company focused on discovering and developing oral small-molecule medicines that target G protein-coupled receptors (GPCRs). It operates a proprietary Native Complex Platform to accelerate GPCR drug discovery and aims to deliver therapies across endocrinology, immunology and inflammation, and metabolic diseases. The company is advancing a GPCR-focused pipeline with candidates such as SEP-786 (PTH1R) and SEP-631 (MRGPRX2), along with programs targeting TSHR, GLP-1R, GIPR, and GCGR, with the goal of translating GPCR biology into orally available medicines that address unmet patient needs.

Convergent Therapeutics is a biotechnology company developing radiopharmaceutical therapies for prostate cancer. Its proprietary platform links monoclonal antibodies targeting prostate-specific membrane antigen (PSMA) with alpha-emitting radionuclides, enabling precise delivery of radiation to cancer cells while minimizing exposure to healthy tissue.

Orbital Therapeutics develops innovative RNA-based medicines to enhance global health. Its platform integrates RNA technology, delivery techniques, data analytics, and automation to create a broad portfolio of treatments for various diseases.

Harpoon Therapeutics, Inc. is a clinical-stage immunotherapy company based in South San Francisco, California, focused on developing a novel class of T cell engagers to harness the body's immune system for treating cancer and other diseases. The company utilizes its proprietary TriTAC (Tri-specific T cell Activating Construct) platform to create engineered proteins that direct T cells to target and kill cells expressing specific antigens. Harpoon's lead product candidate, HPN424, is currently undergoing Phase I clinical trials for metastatic castration-resistant prostate cancer. Additionally, the company is advancing other candidates, including HPN536, which is in Phase I/IIa trials for ovarian cancer and MSLN-expressing tumors, as well as HPN217 for multiple myeloma and HPN328 for small cell lung cancer. Harpoon Therapeutics was founded in 2015 and is committed to addressing unmet medical needs in oncology through innovative therapies.

Flare Therapeutics is a biotechnology company that develops small-molecule medicines by targeting transcription factors. It focuses on identifying switch sites—druggable regions that regulate transcription factor activity—to address disease-causing mutations. Leveraging insights into transcription factor biology, Flare aims to translate this understanding into precision medicines, building an early pipeline that is initially focused on precision oncology with potential expansions into neurology, rare genetic disorders, immunology, and inflammation. The company combines a drug-discovery platform with a biology-led approach to discover therapies that alter the trajectory of diseases driven by transcription factor dysregulation.

Ring Therapeutics is a biotechnology company developing gene therapies using Anellovector, a redosable and targetable DNA therapy platform based on the biology of the human commensal virome. The platform aims to address limitations of current DNA therapies, including limited tissue access, inability to redose, potential genomic integration, and tolerability concerns. Founded in 2017 and based in Cambridge, Massachusetts, Ring Therapeutics was established by Flagship Pioneering to pursue a multi-product platform. It focuses on discovering and developing Anellovector therapeutics to unlock the full potential of gene therapy, moving beyond gene replacement to a broader set of modalities and mechanisms. The approach targets disease sites with tissue and cellular specificity, aiming to treat conditions across genetic disorders, ophthalmology, oncology, and metabolic disorders and to expand therapeutic options for previously inaccessible diseases.

Founded in 2019, Noema Pharma is a biotechnology company based in Basel, Switzerland. It specializes in developing innovative therapies for orphan neurological disorders characterized by imbalanced neuronal networks, aiming to improve patient outcomes and quality of life.

Hemab is a company that creates preventative treatments for bleeding and thrombosis problems. The medicines developed by the business aggregate, modify, balance, and at times recruit endogenous clotting components already present in the blood to the site of injury, allowing patients to produce hemostatic plugs or clots to avert life-threatening complications. Hemab Therapeutics was established in 2017 in Copenhagen, Hovedstaden by Johan H. Faber and Mads Behrndt.

Fusion Pharmaceuticals is a clinical-stage biopharmaceutical company specializing in developing radiopharmaceuticals as precision medicines. It focuses on creating targeted alpha therapeutics using its proprietary linker technology to enhance the safety and efficacy of these drugs, ultimately improving cancer patient outcomes.

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

FogPharma is a biotechnology company focused on developing innovative therapies for cancer treatment through its proprietary cell-penetrating mini proteins (CPMPs). Founded by Greg Verdine, a leader in drug discovery, the company aims to target cancer-causing proteins within cells that are typically inaccessible to traditional drugs. This novel approach allows for the neutralization of these proteins, potentially transforming the treatment landscape for cancer-related diseases. FogPharma collaborates with leading experts in cancer biology and therapy, fostering a network of researchers, investors, and cancer patients dedicated to advancing its mission. The company is committed to delivering this new class of medicines to improve patient outcomes and enhance quality of life for individuals affected by cancer.

Aravive Biologics is a clinical-stage biotechnology company based in Houston, Texas, founded in 2008. The company specializes in developing novel cancer therapeutics aimed at treating various types of malignancies, including solid tumors and hematologic cancers. Aravive's approach involves targeting critical survival pathways, with a key therapeutic candidate designed to act as a decoy that binds to the GAS6 pathway with high affinity, thereby preventing the activation of the AXL signaling pathway. This strategy is intended to inhibit cancer cell growth and survival.

Neumora Therapeutics is a clinical-stage biotechnology company focused on developing precision medicines for brain diseases by combining data science with neuroscience. Founded to address the global brain disease crisis, Neumora takes an innovative approach to treatment development. The company has established a robust therapeutic pipeline that includes seven clinical and preclinical neuroscience programs targeting novel mechanisms of action for various underserved neuropsychiatric and neurodegenerative disorders. Neumora plans to advance its pipeline further by initiating multiple clinical trials across its programs in the upcoming 12 to 18 months.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies to treat chronic inflammatory and autoimmune diseases. The company's pipeline includes three small molecule products. The lead program, IMU-838, is a selective immune modulator that blocks the enzyme DHODH, inhibiting the metabolism of activated immune cells and exhibiting antiviral effects. It is being developed for multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. IMU-935, a selective inverse agonist of the transcription factor RORγt, targets psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Additionally, IMU-856 aims to restore intestinal barrier function for diseases involving bowel barrier dysfunction.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Pretzel Therapeutics is a biotechnology company focused on developing therapies for mitochondrial dysfunction. Founded by leading experts in mitochondrial biology, the company aims to address the underlying causes of mitochondrial dysfunction through a thorough understanding of mitochondrial mechanisms. By creating innovative treatments, Pretzel Therapeutics seeks to provide effective solutions for a range of diseases, particularly those related to aging. The company is dedicated to advancing the field of mitochondrial medicine and improving patient outcomes through targeted therapeutic approaches.

Autobahn Therapeutics develops brain-targeting, small molecule therapies for central nervous system disorders. Its lead program ABX-002 is a thyroid hormone receptor beta agonist being developed as an adjunctive treatment for mood disorders, including major depressive disorder and bipolar depression. The company emphasizes precision tuning of CNS exposure, validated clinical and biologic targets, and biomarker-guided development across neuropsychiatry, neurodegeneration, and neuroinflammation to address high unmet medical needs and improve patient outcomes.

Lexicon Pharmaceuticals is a biopharmaceutical company focused on discovering and developing drugs to treat human diseases. Its pipeline includes candidates for irritable bowel syndrome, type 2 diabetes, rheumatoid arthritis, carcinoid syndrome, and glaucoma. The company has collaborations with major pharmaceutical companies and other organizations.

AlloVir is a biopharmaceutical company focused on developing allogeneic T-cell therapies to combat viral diseases. Its proprietary virus-specific T cell (VST) therapy platform enables the creation of off-the-shelf VSTs, designed to restore immunity in patients with T cell deficiencies at risk from life-threatening viral infections.

Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that specializes in the discovery, development, and commercialization of innovative therapeutic products targeting significant unmet medical needs. Founded by former executives from Receptos, the company focuses on areas such as immunology, inflammation, fibrosis, and oncology. Its pipeline includes several promising candidates: Seralutinib, aimed at addressing pulmonary arterial hypertension; GB004, for inflammatory bowel disease; GB1275, targeting various oncology indications; and GB001, designed for moderate-to-severe eosinophilic asthma. Gossamer Bio's strategy involves leveraging a rich in-licensing environment to enhance its therapeutic offerings, supported by a team recognized for their successful track record in drug development.

Scholar Rock is a biopharmaceutical company dedicated to discovering and developing innovative therapies targeting dysregulated growth factors in disease microenvironments. Its proprietary platform enables the creation of monoclonal antibodies that selectively modulate these proteins at the cellular level, potentially avoiding historical challenges associated with inhibiting growth factors. The company's lead product candidate, SRK-015, is a selective inhibitor of myostatin activation for treating neuromuscular disorders like spinal muscular atrophy.

Founded in 2015, ImCheck Therapeutics specializes in developing immunotherapeutics targeting members of the butyrophilin super-family for treating a range of autoimmune diseases, with a primary focus on immuno-oncology.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

Founded in 2019 and based in Cambridge, Massachusetts, MoMa Therapeutics discovers precision medicines by targeting molecular machines underlying human diseases. The company's platform enables drug development from previously intractable enzyme classes.

Onxeo is a clinical-stage biotechnology company based in Paris, France, focused on developing innovative oncology drugs that target tumor DNA repair mechanisms. The company offers Beleodaq, a treatment for relapsed or refractory peripheral T-cell lymphoma, and is advancing several other candidates, including Livatag, a nanoparticle formulation of doxorubicin currently in Phase III trials for primary liver cancer. Validive, a buccal tablet for preventing and treating severe oral mucositis in head and neck cancer patients, has completed Phase II trials. Additionally, Onxeo is developing AsiDNA, which is undergoing Phase 1 trials for metastatic melanoma, and OX401, which is in preclinical development for DNA damage response and immuno-oncology applications. The company has established partnerships with Spectrum Pharmaceuticals for Beleodaq, Monopar Therapeutics for Validive, and Gustave Roussy for the clinical trials of AsiDNA in relapsed ovarian cancer. Onxeo, previously known as BioAlliance Pharma, was founded in 1997 and has a commitment to advancing oncology treatments worldwide.

Septerna is a biotechnology company focused on discovering and developing oral small-molecule medicines that target G protein-coupled receptors (GPCRs). It operates a proprietary Native Complex Platform to accelerate GPCR drug discovery and aims to deliver therapies across endocrinology, immunology and inflammation, and metabolic diseases. The company is advancing a GPCR-focused pipeline with candidates such as SEP-786 (PTH1R) and SEP-631 (MRGPRX2), along with programs targeting TSHR, GLP-1R, GIPR, and GCGR, with the goal of translating GPCR biology into orally available medicines that address unmet patient needs.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

EQRx, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on re-engineering the drug discovery and delivery process to provide a market-based solution to the escalating costs of medications. EQRx aims to enhance the efficiency and affordability of high-quality, patent-protected medicines by collaborating with various stakeholders across the healthcare system and leveraging advancements in science and technology. The company has developed a robust pipeline with over ten programs, including clinical and preclinical projects targeting oncology and immune-inflammatory conditions, featuring compounds such as Aumolertinib, Lerociclib, Sugemalimab, and Nofazinlimab.

Neumora Therapeutics is a clinical-stage biotechnology company focused on developing precision medicines for brain diseases by combining data science with neuroscience. Founded to address the global brain disease crisis, Neumora takes an innovative approach to treatment development. The company has established a robust therapeutic pipeline that includes seven clinical and preclinical neuroscience programs targeting novel mechanisms of action for various underserved neuropsychiatric and neurodegenerative disorders. Neumora plans to advance its pipeline further by initiating multiple clinical trials across its programs in the upcoming 12 to 18 months.

Lycia Therapeutics operates as a biotechnology company focused on discovering and developing first-in-class therapeutics using its proprietary Lysosomal Targeting Chimeras (LYTACs) platform. This platform enables the degradation of extracellular and membrane-bound proteins associated with challenging-to-treat diseases like cancer and autoimmune disorders, aiming to provide effective treatments for patients.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Laronde is a biotechnology company that specializes in developing an eRNA-based therapeutic platform designed to express a variety of proteins within the body. Founded in 2017 by Flagship Labs, Laronde's innovative Endless RNA technology is engineered to be persistent and non-immunogenic, allowing for repeat dosing and offering flexibility in both formulation and delivery. This platform aims to enhance the predictability and efficacy of new medicines, facilitating the development of therapeutics that can address a wide range of diseases. As Laronde continues to scale its operations, it focuses on advancing multiple programs simultaneously, positioning itself as a key player in the future of medicine.

Vigil Neuroscience is a clinical-stage biotechnology company focused on developing therapeutics that restore the function of microglia, the brain's immune cells. The company aims to improve outcomes for patients with neurodegenerative diseases by advancing its lead monoclonal antibody TREM2 agonist through Phase 1 trials and progressing its small molecule TREM2 agonist towards an Investigational New Drug (IND) application.

Ring Therapeutics is a biotechnology company developing gene therapies using Anellovector, a redosable and targetable DNA therapy platform based on the biology of the human commensal virome. The platform aims to address limitations of current DNA therapies, including limited tissue access, inability to redose, potential genomic integration, and tolerability concerns. Founded in 2017 and based in Cambridge, Massachusetts, Ring Therapeutics was established by Flagship Pioneering to pursue a multi-product platform. It focuses on discovering and developing Anellovector therapeutics to unlock the full potential of gene therapy, moving beyond gene replacement to a broader set of modalities and mechanisms. The approach targets disease sites with tissue and cellular specificity, aiming to treat conditions across genetic disorders, ophthalmology, oncology, and metabolic disorders and to expand therapeutic options for previously inaccessible diseases.

Lyndra Therapeutics develops ultra‑long‑acting oral medicines that release drugs over periods of up to a month while temporarily residing in the stomach. The company focuses on sustained, steady‑state delivery for conditions such as Alzheimer’s disease, cardiovascular and metabolic disorders, psychiatric and opioid use disorders, immunology, HIV, malaria vector control, and oral contraceptives. Founded in 2015 and headquartered in Watertown, Massachusetts, Lyndra’s platform aims to improve medication adherence and reduce healthcare costs by enabling once‑weekly or monthly dosing of approved drugs and novel therapeutics.

Abata Therapeutics is a biotechnology company dedicated to developing innovative cell therapies for patients suffering from progressive multiple sclerosis and other severe autoimmune and inflammatory diseases. The company specializes in the manipulation of regulatory T cells (Tregs) to address these conditions, aiming to restore balance within the immune system. By engineering Tregs to precisely target disease-related antigens, Abata Therapeutics creates therapies that can effectively treat diseases such as multiple sclerosis and type 1 diabetes without imposing systemic immune suppression. Founded by experts in immunology and cell therapy, Abata is committed to translating advanced scientific research into meaningful therapies that improve the lives of individuals facing chronic autoimmune challenges.

Bright Peak Therapeutics is a biotechnology company focused on developing a range of immunotherapies aimed at treating cancer and autoimmune diseases. The company specializes in creating immuno-cytokines that possess pharmacological properties enabling tissue and cell-specific targeting of cytokine payloads. This innovative approach is designed to enhance the efficacy of treatments, ultimately improving patient outcomes and quality of life. By leveraging its expertise in cytokine therapeutics, Bright Peak aims to advance the field of immunotherapy and provide effective solutions for challenging medical conditions.

Cyclerion Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on discovering, developing, and commercializing treatments for serious and orphan diseases. The company leverages soluble guanylate cyclase (sGC) pharmacology to create next-generation sGC stimulators. Its pipeline includes Olinciguat, an orally administered vascular sGC stimulator in Phase II studies for sickle cell disease; Praliciguat, another oral sGC stimulator in Phase II trials for diabetic nephropathy and heart failure with preserved ejection fraction; and IW-6463, a CNS-penetrant sGC stimulator in Phase I trials for neurodegenerative diseases. Additionally, Cyclerion is exploring liver-targeted and lung-targeted sGC stimulators. The company was incorporated in 2018.

Flare Therapeutics is a biotechnology company that develops small-molecule medicines by targeting transcription factors. It focuses on identifying switch sites—druggable regions that regulate transcription factor activity—to address disease-causing mutations. Leveraging insights into transcription factor biology, Flare aims to translate this understanding into precision medicines, building an early pipeline that is initially focused on precision oncology with potential expansions into neurology, rare genetic disorders, immunology, and inflammation. The company combines a drug-discovery platform with a biology-led approach to discover therapies that alter the trajectory of diseases driven by transcription factor dysregulation.

ViaCyte, Inc. is a regenerative medicine company based in San Diego, California, that specializes in developing cell replacement therapies for diabetes. The company’s primary product candidates, VC-01 and VC-02, aim to treat type 1 diabetes by implanting pancreatic beta-cell precursors derived from stem cells within an encapsulation device. This innovative approach allows the implanted cells to produce insulin in response to blood glucose levels while minimizing the risk of immune rejection, thereby eliminating the need for immunosuppressants. ViaCyte's goal is to provide a long-term solution that can free both type 1 and type 2 diabetes patients from dependence on insulin and reduce associated complications such as hypoglycemia and cardiovascular issues. Founded in 1999, ViaCyte was previously known as Novocell, Inc., and changed its name in 2010. The company also operates an additional facility in Athens, Georgia.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Founded in 2017, ElevateBio operates a portfolio of companies focused on developing, manufacturing, and commercializing transformative cell and gene therapies for severe diseases. Through strategic partnerships with leading scientists and inventors, along with a centralized facility for efficient translation of R&D into viable therapies, the company aims to deliver life-changing medicines to patients.

Evox Therapeutics Limited is a biotechnology company based in Oxford, United Kingdom, that specializes in developing exosome-based therapeutics for the treatment of severe diseases with limited treatment options. Founded in 2016, the company focuses on harnessing and engineering extracellular vesicles, known as exosomes, to facilitate targeted delivery of nucleic acids and proteins. Evox's innovative platform allows for the modification of exosomes using advanced molecular engineering techniques, enabling effective drug delivery to specific organs, including the brain and central nervous system. By leveraging these natural delivery capabilities, Evox aims to overcome the limitations associated with conventional protein, antibody, and nucleic acid therapies, thereby creating novel therapeutic solutions that could significantly impact human health. The company is supported by a robust intellectual property portfolio and is positioned as a leader in this emerging therapeutic space.

Sagimet Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapeutics to treat diseases like nonalcoholic steatohepatitis (NASH) and certain cancers. The company specializes in targeting dysfunctional metabolic pathways, with a particular expertise in fatty acid synthase (FASN) biology.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

FogPharma is a biotechnology company focused on developing innovative therapies for cancer treatment through its proprietary cell-penetrating mini proteins (CPMPs). Founded by Greg Verdine, a leader in drug discovery, the company aims to target cancer-causing proteins within cells that are typically inaccessible to traditional drugs. This novel approach allows for the neutralization of these proteins, potentially transforming the treatment landscape for cancer-related diseases. FogPharma collaborates with leading experts in cancer biology and therapy, fostering a network of researchers, investors, and cancer patients dedicated to advancing its mission. The company is committed to delivering this new class of medicines to improve patient outcomes and enhance quality of life for individuals affected by cancer.

Yumanity Therapeutics is a biotechnology company dedicated to transforming drug discovery for neurodegenerative diseases linked to protein misfolding. Founded in December 2014 by protein folding expert Susan Lindquist and biotech leader Tony Coles, Yumanity aims to develop innovative, disease-modifying therapies to address critical unmet medical needs in conditions such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). The company leverages proprietary platforms to identify potential therapeutic targets and has made progress in advancing a new chemical lead series specifically for Parkinson’s disease, while also exploring additional compounds for Alzheimer's disease and ALS.

Locanabio is a biotechnology company developing RNA-targeted gene therapies for underserved diseases, with a focus on neuromuscular, neurodegenerative, and retinal conditions. It employs an RNA-targeting platform to modify disease-causing RNA and correct aberrant transcripts, offering therapies distinct from DNA-based approaches. The company identifies target RNA sequences within transcripts and programs therapies to advance candidates for rare genetic neuromuscular, neurodegenerative, and retinal diseases. Located in San Diego, Locanabio aims to translate RNA-directed interventions into treatments for patients with severe conditions.

Faze Medicines is a biotechnology company focused on developing small molecule therapies that target biomolecular condensates to address the underlying biology of disease. It uses screening and proteomics to define condensate interaction networks and identify therapeutic candidates aimed at cellular mechanisms implicated in conditions such as amyotrophic lateral sclerosis and myotonic dystrophy type 1, with research extending to other diseases including frontotemporal dementia. Founded in 2020 and based in Cambridge, Massachusetts.

Founded in 2019, Noema Pharma is a biotechnology company based in Basel, Switzerland. It specializes in developing innovative therapies for orphan neurological disorders characterized by imbalanced neuronal networks, aiming to improve patient outcomes and quality of life.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.

Ambrx, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, specializing in the development of engineered precision biologics through an expanded genetic code technology platform. This innovative approach enables the incorporation of synthetic amino acids into proteins, facilitating the creation of next-generation therapeutics. Ambrx focuses on various product candidates, including antibody drug conjugates (ADCs), bispecific antibodies, targeted immuno-oncology treatments, and long-acting therapeutic peptides aimed at addressing metabolic and cardiovascular diseases. The company has a robust development pipeline featuring multiple clinical and preclinical programs, with its lead candidate ARX788 among them. Ambrx also collaborates with other organizations, such as MabSpace Biosciences and BeiGene, to enhance its research and development efforts in oncology and other therapeutic areas. Founded in 2003, Ambrx is committed to advancing the efficacy and safety of new biologic therapies for patients.

GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

Talaris Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies aimed at eliminating chronic immunosuppression for organ transplant recipients. The company's leading product, FCR001, is designed to enable living donor kidney transplant recipients to discontinue all chronic immunosuppression therapy within twelve months post-transplant. In addition to its applications in organ transplantation, Talaris’s technology facilitates the safe use of hematopoietic stem cells from unmatched and unrelated donors for bone marrow transplants, reducing the associated toxicity and risks of traditional methods. Founded in 1988 and originally named Regenerex, Inc., Talaris Therapeutics rebranded in March 2019. The company is headquartered in Louisville, Kentucky, with an additional office in Wellesley, Massachusetts.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Founded in 2018 and headquartered in Cambridge, Massachusetts, the company is advancing a diverse pipeline that includes five clinical-stage investigational therapies and several preclinical compounds targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Key products under development include CVL-231, a positive allosteric modulator for schizophrenia; CVL-865, which is being tested for drug-resistant focal onset epilepsy and acute anxiety; and Tavapadon, a selective dopamine D1/D5 partial agonist for Parkinson's disease. Cerevel Therapeutics employs a unique approach that leverages expertise in neurocircuitry and receptor selectivity to address unmet medical needs in the field of neuroscience.

Imvax, Inc. is a clinical-stage biotechnology company based in Philadelphia, Pennsylvania, focused on developing innovative, patient-specific vaccines and immunotherapy strategies for the treatment of malignant gliomas and other cancers with significant unmet medical needs. Incorporated in 2015, Imvax's primary product, IGV-001, is an autologous tumor cell vaccine designed to harness the patient's immune system to generate a robust response against tumor cells. The company employs advanced methodologies, including patient cell harvest and treatment, immunogenic cell death, and activation of both the innate and adaptive immune systems, to enhance therapeutic outcomes. Through its novel approach, Imvax aims to provide effective treatments for patients facing challenging cancer diagnoses.

Autobahn Therapeutics develops brain-targeting, small molecule therapies for central nervous system disorders. Its lead program ABX-002 is a thyroid hormone receptor beta agonist being developed as an adjunctive treatment for mood disorders, including major depressive disorder and bipolar depression. The company emphasizes precision tuning of CNS exposure, validated clinical and biologic targets, and biomarker-guided development across neuropsychiatry, neurodegeneration, and neuroinflammation to address high unmet medical needs and improve patient outcomes.

SQZ Biotech is a clinical-stage biotechnology company focused on developing innovative cell therapies for cancer, infectious diseases, and other serious health conditions. Utilizing its proprietary CellSqueeze technology, the company can deliver a variety of materials into diverse patient cell types, enabling the engineering of therapies that address a wide range of clinical challenges. SQZ Biotech aims to create well-tolerated and effective cell therapies that enhance patient experiences compared to traditional approaches. With its commitment to internal research and strategic partnerships, SQZ is working to advance a new generation of cell therapies through both immune activation and immune suppression methods, showcasing its potential to transform treatment options for patients.

Erasca is a clinical-stage precision oncology company dedicated to discovering and developing therapies for patients with cancers driven by the RAS/MAPK pathway. Founded in 2018, the company's pipeline includes Naporafenib, ERAS-007, and ERAS-601, targeting various nodes of this pathway.

iTeos Therapeutics is a biotechnology company focused on immuno-oncology, developing cancer therapies that target the tumor microenvironment to overcome immune suppression and enhance efficacy of existing treatments. The company pursues small molecule and antibody approaches, including EOS-850, a small molecule antagonist of the adenosine A2A receptor, and EOS-448, an antagonist of TIGIT, with early-stage clinical programs designed to restore immune activity against tumors. By modulating metabolic and immune pathways within the tumor microenvironment, iTeos aims to improve responses across cancer types and complement immune checkpoint therapies.