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Aadi Bioscience, Inc. is a clinical-stage biopharmaceutical company based in Pacific Palisades, California, focused on developing precision therapies for genetically-defined cancers. The company specializes in mTOR inhibitors, particularly ABI-009, aimed at treating patients with alterations in the TSC1 or TSC2 genes, where existing mTOR inhibitors have limitations in pharmacology, drug delivery, or safety. Aadi's primary focus is on providing transformational therapies for patients with ultra-rare cancers, such as perivascular epithelioid cell tumors (PEComa). Since its founding in 2011, Aadi Bioscience has aimed to address significant unmet medical needs in oncology and related therapeutic areas.

Bambusa Therapeutics operates as biotechnology company, that focuses on developing bispecific antibodies.

Aadi Bioscience, Inc. is a clinical-stage biopharmaceutical company based in Pacific Palisades, California, focused on developing precision therapies for genetically-defined cancers. The company specializes in mTOR inhibitors, particularly ABI-009, aimed at treating patients with alterations in the TSC1 or TSC2 genes, where existing mTOR inhibitors have limitations in pharmacology, drug delivery, or safety. Aadi's primary focus is on providing transformational therapies for patients with ultra-rare cancers, such as perivascular epithelioid cell tumors (PEComa). Since its founding in 2011, Aadi Bioscience has aimed to address significant unmet medical needs in oncology and related therapeutic areas.

Cartesian Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in the United States that focuses on developing innovative cell and gene immunotherapies for the treatment of cancer and autoimmune diseases. The company specializes in chimeric antigen receptor therapies aimed at addressing conditions such as multiple myeloma. Utilizing its ImmTOR immune tolerance platform, Cartesian Therapeutics seeks to foster antigen-specific immunity while reducing adverse immune responses. The company employs proprietary technology and a manufacturing platform to deliver mRNA molecules into cells, enhancing their functionality without the risk of integrating into the cell's genetic material, as mRNA naturally degrades over time.

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 biotechnology company focused on precision oncology, dedicated to developing targeted therapeutics that specifically address validated oncogenic drivers in cancer. The firm aims to discover and create alteration-specific therapies that provide hope and improved treatment options for individuals affected by cancer. By concentrating on high-value oncogenic alterations, Alterome Therapeutics seeks to enhance the effectiveness of cancer treatments and support healthcare providers in their efforts to combat the disease.

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.

Sensorion is a biopharmaceutical company based in Montpellier, France, focused on developing innovative therapies for inner ear disorders, particularly hearing loss and vestibular deficits. The company's lead product, SENS-401, is currently in phase II clinical trials aimed at treating sudden sensorineural hearing loss. Additionally, Sensorion is advancing SENS-111, which is in phase II trials for acute unilateral vestibulopathy, and SENS-300, which is in the pre-clinical stage for addressing inner ear toxicity. The company also collaborates with Cochlear Limited to explore combination therapies for patients with cochlear implants. Founded in 2009, Sensorion is dedicated to conducting research and development to discover drugs that can restore and treat hearing loss, positioning itself as a key player in the field of otology.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Syros Pharmaceuticals is a life sciences company that is focused on treating disease by mapping gene regulatory circuits and modulating the factors that regulate gene expression. Syros has pioneered world-leading gene control research and drug discovery capabilities with a proven ability to integrate disease biology and genomic data--a proficiency that is not well represented in pharmaceutical R&D. Central to the Syros approach is a proprietary platform of carefully integrated assay technologies, bioinformatics, and biologic insights developed by members of Syros' senior leadership. While this scientific approach has applications in many therapeutic areas, Syros has demonstrated success initially in oncology, where it may help address numerous unmet medical needs.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Engine Biosciences is a biotech company focused on revolutionizing drug discovery through the application of machine learning and network biomedicine. Based in San Francisco and Asia, the company has developed a proprietary platform that combines high-throughput wet lab experimentation with advanced artificial intelligence algorithms. This platform is designed to map and analyze complex biological networks, generating unique data on gene interactions and providing insights validated through controlled experiments. By doing so, Engine Biosciences aims to accelerate research and development processes and reduce costs associated with creating new medical therapies. The company collaborates with various partners and actively applies its technology to address a range of disease indications, leveraging the expertise of its scientific leadership team, which includes faculty members from renowned institutions such as MIT, Harvard, and the Mayo Clinic.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Sensorion is a biopharmaceutical company based in Montpellier, France, focused on developing innovative therapies for inner ear disorders, particularly hearing loss and vestibular deficits. The company's lead product, SENS-401, is currently in phase II clinical trials aimed at treating sudden sensorineural hearing loss. Additionally, Sensorion is advancing SENS-111, which is in phase II trials for acute unilateral vestibulopathy, and SENS-300, which is in the pre-clinical stage for addressing inner ear toxicity. The company also collaborates with Cochlear Limited to explore combination therapies for patients with cochlear implants. Founded in 2009, Sensorion is dedicated to conducting research and development to discover drugs that can restore and treat hearing loss, positioning itself as a key player in the field of otology.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Flare Therapeutics is a biotechnology company focused on developing small molecule medicines by exploring the biology of transcription factors. The company aims to identify 'switch sites,' which are druggable regions crucial for regulating transcription factors, to target mutations associated with various diseases. Flare's innovative approach has advanced its drug discovery efforts, resulting in a promising pipeline of programs that primarily address well-established transcription factors. Initially concentrating on precision oncology, the company's research also holds potential applications in neurology, rare genetic disorders, immunology, and inflammation. By leveraging insights from its scientific founders, Flare Therapeutics seeks to transform the treatment landscape for cancer patients and other disease areas.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing therapeutic products and therapies for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts aim to address disabling symptoms in conditions affecting the nervous system, including Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma seeks to improve the quality of life and patient outcomes for individuals suffering from these rare conditions.

Abivax is a clinical-stage biotechnology company based in France that develops therapeutics aimed at harnessing the body's natural immune response to treat autoimmune diseases, viral infections, and cancer. The company is currently evaluating its drug candidate, obefazimod, in Phase 3 clinical trials for the treatment of adults with moderately to severely active ulcerative colitis. Abivax focuses on addressing significant unmet medical needs in chronic inflammatory diseases, particularly inflammatory bowel diseases, which include ulcerative colitis and Crohn's disease. Through its innovative drug development platforms, Abivax aims to provide novel and effective treatment options for patients suffering from these conditions.

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.

Neumora Therapeutics operates as a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Pretzel was founded by some of the world’s foremost academics in mitochondrial biology, and we benefit from the leadership of accomplished experts in drug discovery, drug development, and company formation. Our name is inspired by the focal point of our science, the mitochondrion, whose highly folded inner membrane is one of many fascinating features that make it unlike any other organelle in the body.

Abivax is a clinical-stage biotechnology company based in France that develops therapeutics aimed at harnessing the body's natural immune response to treat autoimmune diseases, viral infections, and cancer. The company is currently evaluating its drug candidate, obefazimod, in Phase 3 clinical trials for the treatment of adults with moderately to severely active ulcerative colitis. Abivax focuses on addressing significant unmet medical needs in chronic inflammatory diseases, particularly inflammatory bowel diseases, which include ulcerative colitis and Crohn's disease. Through its innovative drug development platforms, Abivax aims to provide novel and effective treatment options for patients suffering from these conditions.

Sironax is a clinical-stage biotechnology company based in Beijing, China, founded in 2017. The company focuses on developing innovative products and treatments for age-related degenerative diseases, concentrating on mechanisms such as regulated cell death, neuroprotective pathways, and neuroinflammation. By researching apoptosis and other cell death pathways, Sironax aims to create advanced pharmaceuticals that can improve the lives of patients suffering from these conditions. Their goal is to transform the treatment landscape for age-related degenerative diseases, benefiting millions of patients and their families globally.

Lexicon Pharmaceuticals is a biopharmaceutical company focused on the discovery and development of innovative pharmaceutical products aimed at treating various human diseases in areas such as immunology, metabolism, cardiology, and ophthalmology. Its pipeline includes drug candidates like LX1031, which has completed Phase II trials for irritable bowel syndrome, and LX4211, also in Phase II for type 2 diabetes. Other candidates include LX2931, currently in Phase II trials for rheumatoid arthritis, and LX1032, which has completed Phase II for carcinoid syndrome symptoms. The company is also advancing LX7101, a preclinical candidate for glaucoma. Lexicon has formed strategic alliances with several major pharmaceutical companies, including Bristol-Myers Squibb and Takeda Pharmaceutical, as well as collaborations for drug development and discovery.

Senti Biosciences is a clinical-stage biotechnology company specializing in synthetic biology and gene circuit engineering for therapeutic purposes. The company employs its Gene Circuits platform to develop innovative therapies that enhance precision in targeting cancer cells while avoiding damage to healthy cells. Senti Bio's approach involves the use of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells that incorporate its gene circuit technologies, aimed at addressing various oncology indications. Among its product candidates are SENTI-202, a Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells without harming healthy bone marrow, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences aims to provide effective treatment options for difficult-to-treat diseases through advanced cell and gene therapies.

LifeMine Therapeutics, Inc. is a biotechnology company focused on discovering new drug modalities from eukaryotic microbes, particularly fungi. Founded in 2016 and headquartered in Cambridge, Massachusetts, with an additional office in New York, the company employs an integrated platform that combines genomics, artificial intelligence, and synthetic biology. This innovative Avatar-Rx platform utilizes high-throughput microbiology, data science, machine learning, genome engineering, and automation to explore the fungal biosphere for novel genetically encoded molecules with specific biological functions. By integrating chemoinformatic-assisted drug optimization and advanced chemical synthesis with biotransformation, LifeMine Therapeutics aims to advance a pipeline of new medicines into development.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Neumora Therapeutics operates as a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience.

Lycia Therapeutics is a biotechnology firm focused on discovering and developing first-in-class therapies. The company utilizes its innovative lysosomal targeting chimeras (LYTACs) platform to create therapeutics that target and degrade extracellular and membrane-bound proteins associated with challenging diseases, including cancer and autoimmune disorders. By addressing these difficult-to-treat conditions, Lycia Therapeutics aims to provide medical practitioners with effective treatment options to improve patient outcomes.

Laronde is a biopharmaceutical company that specializes in developing eRNA-based therapeutics designed to express a variety of proteins within the body. Founded in 2017 by Flagship Labs, Laronde's innovative platform, known as Endless RNA, is characterized by its persistence, non-immunogenic properties, and adaptability for repeat dosing and various delivery methods. This flexibility positions Laronde to support the simultaneous advancement of multiple therapeutic programs across different disease areas, aiming to create more predictable and impactful medicines for patients. The company's approach seeks to redefine the possibilities of therapeutic development, ultimately contributing to the creation of essential medicines for the future.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

Engine Biosciences is a biotech company focused on revolutionizing drug discovery through the application of machine learning and network biomedicine. Based in San Francisco and Asia, the company has developed a proprietary platform that combines high-throughput wet lab experimentation with advanced artificial intelligence algorithms. This platform is designed to map and analyze complex biological networks, generating unique data on gene interactions and providing insights validated through controlled experiments. By doing so, Engine Biosciences aims to accelerate research and development processes and reduce costs associated with creating new medical therapies. The company collaborates with various partners and actively applies its technology to address a range of disease indications, leveraging the expertise of its scientific leadership team, which includes faculty members from renowned institutions such as MIT, Harvard, and the Mayo Clinic.

Flare Therapeutics is a biotechnology company focused on developing small molecule medicines by exploring the biology of transcription factors. The company aims to identify 'switch sites,' which are druggable regions crucial for regulating transcription factors, to target mutations associated with various diseases. Flare's innovative approach has advanced its drug discovery efforts, resulting in a promising pipeline of programs that primarily address well-established transcription factors. Initially concentrating on precision oncology, the company's research also holds potential applications in neurology, rare genetic disorders, immunology, and inflammation. By leveraging insights from its scientific founders, Flare Therapeutics seeks to transform the treatment landscape for cancer patients and other disease areas.

ElevateBio, LLC is a holding company based in Cambridge, Massachusetts, focused on the development of cell and gene-based therapies for treating cancer and viral infections. Founded in 2017 by Vikas Sinha, Mitchell Finer, and David Hallal, ElevateBio operates a portfolio of companies dedicated to creating life-changing medicines. The company collaborates with leading scientists and innovators to build this portfolio and has established a centralized facility that streamlines the transition from research and development to the commercialization of these therapies. This integrated approach enhances efficiency and accelerates the availability of medical products aimed at addressing severe diseases.

Sagimet Biosciences is a clinical-stage biopharmaceutical company based in San Mateo, California, that specializes in developing novel therapeutics for oncology and infectious diseases. The company focuses on targeting dysfunctional metabolic pathways, particularly through its expertise in fatty acid synthase (FASN) biology. Its lead drug candidate, denifanstat, is a selective FASN inhibitor designed for the treatment of nonalcoholic steatohepatitis (NASH) and is administered as an oral, once-daily pill. Additionally, Sagimet is working on antiviral therapeutics aimed at addressing respiratory syncytial virus, cytomegalovirus, and other human pathogens. Originally known as 3-V Biosciences, Inc., the company rebranded to Sagimet Biosciences in August 2019 and has been active since its incorporation in 2006.

Scorpion Therapeutics, Inc. is a biotechnology company based in Boston, Massachusetts, focused on developing next-generation precision oncology technologies for cancer treatment. Founded in 2020, the company specializes in creating targeted small-molecule drugs that interact with validated cancer targets. Its research encompasses therapies aimed at known oncogenes, previously undruggable cancer targets, and novel targets. By integrating technologies across target discovery, medicinal chemistry, and translational medicine, Scorpion Therapeutics aims to advance a comprehensive pipeline of optimized compounds. The company's goal is to expand the reach of precision medicine, enabling healthcare professionals to offer innovative treatments to a broader range of cancer patients.

Ikena Oncology is a biotechnology company based in Boston, Massachusetts, focused on developing innovative therapies for cancer treatment. The company specializes in biomarker-driven and precision therapies that target specific mechanisms involved in cancer growth, spread, and resistance. Its product pipeline includes several candidates such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-930, an oral small-molecule inhibitor of the TEAD transcription factor in the Hippo signaling pathway. Additionally, Ikena is exploring a kynurenine-degrading enzyme with IK-412 and other discovery-stage programs targeting various oncogenic pathways. Established in 2016, Ikena Oncology aims to address unmet medical needs through a combination of chemistry, translational science, and patient-centered drug development.

Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.

Faze Medicines is a biotechnology company founded in 2020 and located in Cambridge, Massachusetts. The company focuses on developing small molecule drugs that target the underlying mechanisms of disease pathology, with initial therapeutic efforts directed towards conditions such as amyotrophic lateral sclerosis (ALS) and myotonic dystrophy type 1 (DM1). Faze Medicines employs advanced screening and proteomics techniques to explore biomolecular condensate interaction networks, which aids in the identification and development of novel therapeutics. Through its innovative approach, Faze aims to provide effective treatments for patients suffering from these challenging diseases.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing therapeutic products and therapies for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts aim to address disabling symptoms in conditions affecting the nervous system, including Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma seeks to improve the quality of life and patient outcomes for individuals suffering from these rare conditions.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Established in 2018 and based in Cambridge, Massachusetts, the company is advancing a diversified pipeline that includes several clinical-stage investigational therapies targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Notable products in development include CVL-231, a positive allosteric modulator currently in Phase Ib trials for schizophrenia, and CVL-865, also a positive allosteric modulator, which is in Phase II trials for drug-resistant focal onset seizures. Additionally, Tavapadon, a selective dopamine D1/D5 partial agonist, is in a Phase III program for Parkinson's disease, while CVL-936, a selective dopamine D3-preferring antagonist, is undergoing Phase I trials for substance use disorder. The company is also engaged in preclinical research aiming to address various neuroscience indications, leveraging expertise in neurocircuitry and receptor selectivity.

Seer is a developer of a life sciences and health data platform intended to facilitate early, pre-symptomatic detection of chronic life-threatening diseases. The company's platform captures and translates molecular insights from the proteome with improved accuracy and speed, enabling healthcare professionals to detect cancer and neurological diseases, drive earlier treatment and improve patient outcomes.

ElevateBio, LLC is a holding company based in Cambridge, Massachusetts, focused on the development of cell and gene-based therapies for treating cancer and viral infections. Founded in 2017 by Vikas Sinha, Mitchell Finer, and David Hallal, ElevateBio operates a portfolio of companies dedicated to creating life-changing medicines. The company collaborates with leading scientists and innovators to build this portfolio and has established a centralized facility that streamlines the transition from research and development to the commercialization of these therapies. This integrated approach enhances efficiency and accelerates the availability of medical products aimed at addressing severe diseases.

Element Science is a medical device and digital health company that develops solutions at the intersection of clinical-grade wearable devices, machine learning algorithms, and lifesaving therapies to address the needs of high-risk cardiovascular patients, primarily as they transition from the hospital to home. The company developing a proprietary next-generation wearable digital platform that unites patient-centric human factor engineering, sophisticated machine learning algorithm development principles, and rigorous electromechanical medical device development standards. The company was founded in 2011 and is headquartered in San Francisco, California.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Seer is a developer of a life sciences and health data platform intended to facilitate early, pre-symptomatic detection of chronic life-threatening diseases. The company's platform captures and translates molecular insights from the proteome with improved accuracy and speed, enabling healthcare professionals to detect cancer and neurological diseases, drive earlier treatment and improve patient outcomes.

ORIC Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing therapies to overcome resistance in cancer treatments. Founded in 2014 and based in South San Francisco, the company’s lead product candidate, ORIC-101, is a small molecule antagonist of the glucocorticoid receptor, which is associated with resistance to various cancer therapies in solid tumors. Its second candidate, ORIC-533, is an orally bioavailable small molecule inhibitor of CD73, targeting a critical component of the adenosine pathway linked to resistance against chemotherapy and immunotherapy. In addition to these products, ORIC is advancing multiple precision medicines aimed at addressing other mechanisms of cancer resistance. The company’s founders, Charles Sawyers and Scott Lowe, have extensive experience in identifying novel cancer targets that have led to innovative therapies.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Seer is a developer of a life sciences and health data platform intended to facilitate early, pre-symptomatic detection of chronic life-threatening diseases. The company's platform captures and translates molecular insights from the proteome with improved accuracy and speed, enabling healthcare professionals to detect cancer and neurological diseases, drive earlier treatment and improve patient outcomes.

Schrödinger develops a computational platform that facilitates the research efforts of biopharmaceutical and industrial companies, academic institutions, and government laboratories worldwide. Schrödinger also has wholly-owned and collaborative drug discovery programs in a broad range of therapeutic areas. Schrödinger is deeply committed to investing in the science and talent that drive its computational platform.

Oyster Point Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for ocular surface diseases. The company's primary focus is on its lead product candidate, OC-01, a nasal spray that acts as a selective nicotinic acetylcholine receptor agonist. OC-01 aims to address the signs and symptoms of dry eye disease, a chronic condition that affects over 30 million Americans. By activating the trigeminal parasympathetic pathway, OC-01 seeks to restore tear film homeostasis and stimulate the natural production of tears through the Lacrimal Functional Unit.

Hookipa Pharma Inc. is a clinical-stage biopharmaceutical company focused on developing immunotherapeutics for infectious diseases and cancers using its proprietary arenavirus platform. The company’s lead product candidate for infectious diseases, HB-101, is currently undergoing a randomized double-blinded Phase II clinical trial for patients awaiting kidney transplants from cytomegalovirus-positive donors. In oncology, Hookipa is advancing its candidates HB-201 and HB-202, which target human papillomavirus-positive cancers and are in preclinical studies. The company employs its innovative VaxWave and TheraT platforms to stimulate robust immune responses, generating high levels of antigen-specific killer T cells and antibodies, which are essential for effective treatment. Additionally, Hookipa has partnered with Gilead Sciences to develop therapies aimed at achieving functional cures for chronic hepatitis B and HIV infections. Founded in 2011 and based in New York, Hookipa Pharma is dedicated to transforming the landscape of immune therapies.

KSQ Therapeutics, based in Cambridge, Massachusetts, specializes in precision functional genomics to discover innovative drug therapies. Founded in 2015, the company employs its proprietary CRISPRomics™ drug discovery engine to gain insights into the role of human genes across various diseases. This comprehensive understanding of disease biology allows KSQ to identify and validate high-confidence, patient-specific drug targets, enhancing the drug development process by focusing on candidates with the greatest therapeutic potential. The company has initiated several preclinical discovery programs aimed at developing novel medications that could significantly improve patient outcomes.

Kaleido Biosciences is a clinical-stage healthcare company that focuses on harnessing the potential of the microbiome to treat diseases and enhance human health. The company has developed a proprietary product platform that enables the efficient discovery and development of Microbiome Metabolic Therapies (MMTs). These therapies aim to modify the metabolic output and profile of the microbiome by influencing the function and distribution of its existing microbes. Kaleido is advancing a diverse pipeline of MMT candidates targeting various diseases and conditions that address significant unmet patient needs.

Unity Biotechnology designs therapeutics that prevent, halt, and reverse various diseases of aging. The company focuses on clearing senescent cells; and designs senolytic medicines. Its medicines target vulnerabilities unique to senescent cells to clear those cells from the human body while leaving normal cells unaffected.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Unity Biotechnology designs therapeutics that prevent, halt, and reverse various diseases of aging. The company focuses on clearing senescent cells; and designs senolytic medicines. Its medicines target vulnerabilities unique to senescent cells to clear those cells from the human body while leaving normal cells unaffected.

Moderna, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing therapeutics and vaccines using messenger RNA technology. Founded in 2010, the company focuses on various therapeutic areas, including infectious diseases, oncology, rare genetic disorders, and cardiovascular diseases. As of September 2024, Moderna has 40 mRNA development candidates in clinical development, demonstrating its commitment to advancing medical science. The company's mRNA platform has gained significant recognition, particularly with the authorization of its COVID-19 vaccine in December 2020. Moderna has established strategic collaborations with notable organizations such as AstraZeneca, Merck & Co., and the Bill & Melinda Gates Foundation. Additionally, it collaborates with Lonza Ltd. and Catalent for the manufacturing of its COVID-19 vaccine. Through its innovative approach, Moderna aims to provide effective solutions for a range of health challenges.

Lexicon Pharmaceuticals is a biopharmaceutical company focused on the discovery and development of innovative pharmaceutical products aimed at treating various human diseases in areas such as immunology, metabolism, cardiology, and ophthalmology. Its pipeline includes drug candidates like LX1031, which has completed Phase II trials for irritable bowel syndrome, and LX4211, also in Phase II for type 2 diabetes. Other candidates include LX2931, currently in Phase II trials for rheumatoid arthritis, and LX1032, which has completed Phase II for carcinoid syndrome symptoms. The company is also advancing LX7101, a preclinical candidate for glaucoma. Lexicon has formed strategic alliances with several major pharmaceutical companies, including Bristol-Myers Squibb and Takeda Pharmaceutical, as well as collaborations for drug development and discovery.

PrimeraDx, Inc. offers multiplexed infectious disease assays. It provides Scalable Target Amplification Routine technology, which is a method for simultaneous quantitative measurement of multiple target nucleic acids. The company also offers ViraQuant, a multiplexed assay providing quantitative and simultaneous measurement of viruses; and FungiQuant Yeast and FungiQuant Mold, which are fungal panels for mycology testing. Its products are used in miRNA, DNA methylation, quantitative genotyping, gene expression, and viral load measurement applications. PrimeraDx, Inc. was founded in 2004 and is based in Mansfield, Massachusetts.

PrimeraDx, Inc. offers multiplexed infectious disease assays. It provides Scalable Target Amplification Routine technology, which is a method for simultaneous quantitative measurement of multiple target nucleic acids. The company also offers ViraQuant, a multiplexed assay providing quantitative and simultaneous measurement of viruses; and FungiQuant Yeast and FungiQuant Mold, which are fungal panels for mycology testing. Its products are used in miRNA, DNA methylation, quantitative genotyping, gene expression, and viral load measurement applications. PrimeraDx, Inc. was founded in 2004 and is based in Mansfield, Massachusetts.

Standard BioTools, previously known as Fluidigm, is driven by a bold vision – unleashing tools to accelerate breakthroughs in human health. Standard BioTools has an established portfolio of essential, standardized next-generation technologies that help biomedical researchers develop medicines faster and better. As a leading solutions provider, the Company provides reliable and repeatable insights in health and disease using its proprietary mass cytometry and microfluidics technologies that help transform scientific discoveries into better patient outcomes. Standard BioTools works with leading academic, government, pharmaceutical, biotechnology, plant and animal research, and clinical laboratories worldwide, focusing on the most pressing needs in translational and clinical research, including oncology, immunology, and immunotherapy.