Perceptive Advisors

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.

Axonis Therapeutics is a biotechnology company focused on developing therapies for neurological disorders by targeting KCC2, a key mediator of inhibition in the brain. The company utilizes a proprietary discovery engine centered on the neuron-specific K-Cl cotransporter (KCC2) to address various conditions, including epilepsy and pain, as well as other central nervous system pathologies. By harnessing breakthrough research from institutions such as Boston Children’s Hospital, Harvard, and Laval University, Axonis aims to enhance the lives of individuals affected by these disorders through improved modulation of neuronal inhibition.

scPharmaceuticals Inc. is a pharmaceutical company focused on developing and commercializing innovative therapeutic options for patients with serious medical conditions. The company’s lead product candidate, Furoscix, features a proprietary buffered formulation of furosemide delivered via the SmartDose drug delivery system, targeting congestion in patients with decompensated heart failure. In addition to Furoscix, scPharmaceuticals is advancing its product pipeline with scCeftriaxone, an antibiotic for infections from gram-positive and gram-negative organisms, and its scCarbapenem program, aimed at treating infections caused by gram-negative organisms. Founded in 2013 and headquartered in Burlington, Massachusetts, scPharmaceuticals aims to provide a convenient, safe, and effective alternative to intravenous drug administration through subcutaneous delivery, addressing the associated costs and risks. The company collaborates with West Pharmaceutical Services, Inc. to enhance the SmartDose system for Furoscix.

CereVasc, Inc. is a medical device company dedicated to developing innovative, minimally invasive treatments for neurological diseases. Founded in 2014 and based in Auburndale, Massachusetts, the company focuses on its flagship product, the eShunt System. This system provides an endovascularly deliverable cerebrospinal fluid shunt along with necessary delivery components, designed to eliminate the need for invasive surgical procedures, general anesthesia, and prolonged hospitalization associated with traditional treatments for communicating hydrocephalus. By offering a less invasive option, the eShunt System aims to enhance patient outcomes and reduce the overall costs of surgical interventions for this serious condition.

Adaptilens, LLC is focused on developing an injectable, physiologically adaptive intraocular lens that provides patients with clear vision at various distances without the need for eyeglasses or contact lenses. Incorporated in 2019 and based in Chestnut Hill, Massachusetts, the company's innovative lens mimics the function of the natural human lens, allowing the ciliary muscles within the eye to alter the shape of the lens. This unique design enables natural accommodation, enabling users to focus on both near and distant objects effectively. Adaptilens aims to enhance the quality of life for patients by offering a solution that facilitates excellent near, intermediate, and distance vision.

Ocular Therapeutix, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for eye diseases using its proprietary bioresorbable hydrogel platform technology. The company offers several products, including ReSure Sealant, a hydrogel ophthalmic wound sealant designed to protect corneal incisions after cataract surgery, and DEXTENZA, an ophthalmic insert for the treatment of post-surgical ocular pain and inflammation, as well as allergic conjunctivitis and dry eye diseases. Ocular Therapeutix is also developing OTX-TP, an insert aimed at reducing intraocular pressure in glaucoma patients, and OTX-TIC, an intracameral implant for similar indications, along with OTX-TKI, an intravitreal implant targeting wet age-related macular degeneration. In addition, the company has preclinical programs focused on various ocular conditions. Ocular Therapeutix collaborates with Regeneron Pharmaceuticals to integrate its hydrogel technology with Regeneron's VEGF-targeting compounds for retinal disease treatments. Founded in 2006, the company is headquartered in Bedford, Massachusetts.

Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for patients affected by rare allergic and immunological diseases. The company focuses on creating life-changing treatments, with its lead program, STAR-0215, being a monoclonal antibody designed to inhibit plasma kallikrein, currently in preclinical development for hereditary angioedema. Additionally, Astria is advancing STAR-0310, another monoclonal antibody, which targets the OX40 receptor and is also in preclinical development for atopic dermatitis, a condition characterized by impaired skin barrier function and significant itching. Through its targeted approach, Astria Therapeutics aims to address unmet medical needs in the field of immunology.

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.

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.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing medicines for underserved patient populations affected by rare diseases and cancer. The company employs a precision medicine approach and has a diverse portfolio of small molecule targeted oncology products. Its lead candidate, nirogacestat, an oral gamma secretase inhibitor, is currently in Phase III clinical trials for desmoid tumors. Additionally, SpringWorks is advancing several other product candidates, including mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for multiple myeloma. The company has established collaborations with organizations such as BeiGene, GlaxoSmithKline, and Allogene, aimed at enhancing the development of its therapies. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks is committed to unlocking the potential of its pipeline to provide innovative solutions for patients suffering from cancer and rare diseases.

AvengeBio is a biotechnology company based in Cambridge, Massachusetts, focused on developing cell-generated immunotherapies aimed at eradicating solid tumors. Founded in 2019, the company is advancing a drug delivery platform designed to enable the precision delivery of cytokines and other potent immunomodulatory molecules. This innovative approach allows for controlled release over time, fostering robust local and systemic anti-tumor immune responses while minimizing the toxicities typically associated with conventional immunotherapies. Through its research and development efforts, AvengeBio seeks to address the challenges posed by intractable solid tumors and improve treatment outcomes for cancer patients.

HilleVax is a clinical-stage biopharmaceutical company established in 2021 and based in Boston, Massachusetts. The company specializes in the development and commercialization of innovative vaccines. Its primary focus is on HIL-214, a vaccine candidate that utilizes virus-like particle (VLP) technology aimed at preventing moderate-to-severe acute gastroenteritis caused by norovirus infection.

Emulate, Inc. specializes in organ-on-chip technology that simulates human biology to enhance the understanding of how diseases, medications, chemicals, and food impact human health. The company develops organ-chips for various organs, including the lung, intestine, liver, and skin, which replicate normal functions and disease conditions. Emulate's innovative technology offers researchers a more accurate alternative to traditional experimental methods, such as cell cultures and animal testing, by providing detailed insights into human biological responses. Additionally, the company supplies drug development and instrumentation software to facilitate research and development workflows for its clients. Emulate serves a diverse range of sectors, including biotechnology, pharmaceuticals, consumer health, cosmetics, chemicals, food, agrochemicals, government agencies, and academic institutions. Founded in 2013 and headquartered in Boston, Massachusetts, Emulate was previously known as Emulate Living MicroDevices, Inc. until its name change in 2014.

Amylyx Pharmaceuticals develops innovative therapies for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. The company's primary product, AMX0035, is an investigational therapy that combines two active compounds, sodium phenylbutyrate and Taurursodiol. This fixed-dose formulation aims to address mitochondrial energy crises and the accumulation of toxic proteins in the endoplasmic reticulum, which contribute to neurological decline. In addition to ALS, Amylyx's research pipeline includes potential treatments for Wolfram syndrome and Alzheimer's disease. Founded in 2013 and based in Cambridge, Massachusetts, Amylyx Pharmaceuticals is dedicated to advancing solutions for serious brain disorders.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Renovia Inc. was a Boston-based healthcare company founded in 2016, focused on developing prescription digital therapeutics for pelvic floor disorders. The company aimed to enhance the lives of women suffering from conditions such as stress, mixed, and urgent urinary incontinence, including overactive bladder. Renovia's innovative device provided a clinically proven, non-surgical, drug-free treatment option, allowing for real-time visualization of pelvic movement during muscle training, thereby facilitating effective therapy. However, in late 2022, Renovia ceased operations following a strategic review by its investors, which included the consideration of a potential sale. In January 2023, most of the company's assets, including its intellectual property, were acquired by Axena Health, Inc.

Pyxis Oncology, established in 2019 in Boston, Massachusetts, is a biotechnology company specializing in the development of antibody therapeutics for cancer treatment. The company focuses on promoting the body's immune response to cancer by analyzing tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and tumor cell signaling pathways within cold tumors. Pyxis aims to create novel antibody-based immunotherapies to directly kill tumor cells and address underlying pathologies that enable cancer proliferation and immune evasion, with the goal of improving patient outcomes for difficult-to-treat cancers.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, focused on developing innovative vaccines using its proprietary Multiple Antigen Presenting System (MAPS) technology. This approach allows for the combination of protective polysaccharides and proteins into a single vaccine, thereby enhancing immune responses against a variety of infectious diseases affecting both children and adults. Affinivax targets several significant pathogens, including Streptococcus pneumoniae, bacterial nosocomial infections, Salmonella typhi, and Staphylococcus aureus. The company aims to create vaccines that not only provide broader disease coverage compared to existing options but also address infectious diseases lacking effective immunization strategies. Founded in 2014, Affinivax benefits from the expertise of leading professionals in infectious diseases and vaccines, and it has received backing from the Bill & Melinda Gates Foundation, along with an exclusive license from Boston Children’s Hospital for its MAPS technology.

IsoPlexis is a life science technology company based in Branford, Connecticut, founded in 2013. It focuses on developing a single-cell detection system that identifies patient immune responses at the single-cell level. This innovative platform enables the assessment of therapeutic safety and efficacy, as well as the monitoring of disease progression. IsoPlexis specializes in cell-based proteomic systems and biomarkers, providing insights into functional protein biology and cellular signaling networks with high resolution. By offering these capabilities, the company aids in predicting patient responses to novel immunotherapies and cellular therapies, ultimately enhancing therapeutic development and informing clinical decisions.

IsoPlexis is a life science technology company based in Branford, Connecticut, founded in 2013. It focuses on developing a single-cell detection system that identifies patient immune responses at the single-cell level. This innovative platform enables the assessment of therapeutic safety and efficacy, as well as the monitoring of disease progression. IsoPlexis specializes in cell-based proteomic systems and biomarkers, providing insights into functional protein biology and cellular signaling networks with high resolution. By offering these capabilities, the company aids in predicting patient responses to novel immunotherapies and cellular therapies, ultimately enhancing therapeutic development and informing clinical decisions.

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.

Zymergen, Inc. is a biotechnology company that focuses on researching, developing, and manufacturing microbes for various industries, including agriculture, chemicals, materials, pharmaceuticals, electronics, and personal care. Founded in 2013 and headquartered in Emeryville, California, Zymergen employs a platform that integrates automation, machine learning, and genomics to enhance the efficiency of microbial strain optimization and production processes. This technology enables the company to improve existing manufacturing strains and facilitates the development of new products by engineering novel molecules from microbes. With additional offices in Boise, Idaho; Medford, Massachusetts; Seattle, Washington; and Tokyo, Japan, Zymergen aims to partner with nature to create innovative materials and products that deliver significant value across multiple sectors.

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.

Thrive Earlier Detection Corp. is a healthcare company that specializes in developing advanced blood testing technologies for early cancer detection. Its flagship product, CancerSEEK, is a liquid biopsy test that analyzes genomic mutations in tumor DNA and protein markers in plasma, allowing for the identification of various cancers at their nascent stages, often before symptoms manifest. Founded in 2018 and headquartered in Cambridge, Massachusetts, with an additional office in Baltimore, Maryland, Thrive aims to integrate cancer screening into standard medical practice. The company leverages real-world data and machine learning to enhance the accuracy of cancer detection, facilitate follow-up testing, and streamline the transition to oncological care. Thrive operates as a subsidiary of Exact Sciences Corporation and has received investments from notable firms including Third Rock Ventures and Section 32.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

Pear Therapeutics, Inc. is a healthcare company specializing in the discovery, development, and delivery of software-based therapeutics aimed at treating behavioral health disorders, including addiction, schizophrenia, anxiety, depression, and chronic insomnia. Founded in 2013 and headquartered in Boston, with an additional office in San Francisco, Pear Therapeutics offers a range of digital therapies designed to enhance treatment outcomes. Notable products include reSET, a tool for substance abuse treatment used alongside traditional therapy; reSET-O, which addresses opioid use disorder; and Somryst, aimed at chronic insomnia. The company also provides PearConnect, a patient services center that connects users with advocates, clinicians, and insurance providers to facilitate support and resource access throughout treatment. By integrating digital interventions with pharmaceuticals, Pear Therapeutics aims to improve patient engagement, clinical outcomes, and cost-effectiveness in healthcare.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

CereVasc, Inc. is a medical device company dedicated to developing innovative, minimally invasive treatments for neurological diseases. Founded in 2014 and based in Auburndale, Massachusetts, the company focuses on its flagship product, the eShunt System. This system provides an endovascularly deliverable cerebrospinal fluid shunt along with necessary delivery components, designed to eliminate the need for invasive surgical procedures, general anesthesia, and prolonged hospitalization associated with traditional treatments for communicating hydrocephalus. By offering a less invasive option, the eShunt System aims to enhance patient outcomes and reduce the overall costs of surgical interventions for this serious condition.

Atea Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that focuses on discovering and developing antiviral therapies for severe viral infections. The company's lead candidate, AT-527, is an antiviral agent currently undergoing phase 3 clinical trials for the treatment of COVID-19, caused by SARS-CoV-2. Additionally, Atea is developing several other product candidates, including AT-787, which is in phase 2 trials for hepatitis C, and AT-752 for dengue. Other candidates, such as AT-889 and AT-934, are also in phase 2 clinical trials targeting respiratory syncytial virus. Established in 2014, Atea Pharmaceuticals aims to improve treatment options for patients suffering from serious viral infections.

Quellis Biosciences, Inc. is a biopharmaceutical company headquartered in Waltham, Massachusetts, established in 2017. The company is dedicated to developing new monoclonal antibody therapies aimed at treating rare diseases, particularly for patients who lack effective treatment options. By focusing on next-generation antibody therapies, Quellis seeks to enhance patient outcomes and deliver innovative solutions that address unmet medical needs. Through its research and development efforts, the company aims to optimize therapeutic leads and identify promising antibodies, ultimately improving the quality of life for individuals affected by serious rare conditions.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

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.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing medicines for underserved patient populations affected by rare diseases and cancer. The company employs a precision medicine approach and has a diverse portfolio of small molecule targeted oncology products. Its lead candidate, nirogacestat, an oral gamma secretase inhibitor, is currently in Phase III clinical trials for desmoid tumors. Additionally, SpringWorks is advancing several other product candidates, including mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for multiple myeloma. The company has established collaborations with organizations such as BeiGene, GlaxoSmithKline, and Allogene, aimed at enhancing the development of its therapies. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks is committed to unlocking the potential of its pipeline to provide innovative solutions for patients suffering from cancer and rare diseases.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

Relay Therapeutics, Inc. is a clinical-stage precision medicine company based in Cambridge, Massachusetts, focused on transforming the drug discovery process. The company employs a novel allosteric drug-discovery platform, known as Dynamo, which integrates advanced computational techniques with experimental methods to study protein motion and interactions. This innovative approach enables the identification and development of therapies targeting complex diseases, particularly in oncology. Relay Therapeutics is advancing a pipeline of product candidates, including RLY-1971, a small molecule inhibitor targeting SHP2, RLY-4008, a selective inhibitor of FGFR2 for advanced solid tumors, and RLY-PI3K1047, aimed at mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, the company aims to deliver life-changing therapies to patients by addressing previously intractable targets in precision oncology and genetic diseases.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

Vapotherm, Inc. is a medical technology company specializing in the development and commercialization of proprietary Hi-VNI technology products designed to treat patients of all ages experiencing respiratory distress. Headquartered in Exeter, New Hampshire, the company offers a range of precision flow systems, including Precision Flow Hi-VNI, Precision Flow Plus, Precision Flow Classic, and Precision Flow Heliox, which deliver heated, humidified, and oxygenated air at high velocity through a small-bore nasal interface. Vapotherm also provides various companion products, such as the Vapotherm Transfer Unit for patient mobility, the Q50 compressor for compressed air, and aerosol solutions for medication delivery. Additionally, the company offers disposable products, including single-use nasal interfaces and Oxygen Assist Modules to help clinicians maintain target oxygen levels. Vapotherm generates revenue primarily through direct sales to hospitals in the United States and through distributors in select international markets. Founded in 1993, the company is committed to enhancing clinical outcomes and improving the quality of life for patients.

Renovia Inc. was a Boston-based healthcare company founded in 2016, focused on developing prescription digital therapeutics for pelvic floor disorders. The company aimed to enhance the lives of women suffering from conditions such as stress, mixed, and urgent urinary incontinence, including overactive bladder. Renovia's innovative device provided a clinically proven, non-surgical, drug-free treatment option, allowing for real-time visualization of pelvic movement during muscle training, thereby facilitating effective therapy. However, in late 2022, Renovia ceased operations following a strategic review by its investors, which included the consideration of a potential sale. In January 2023, most of the company's assets, including its intellectual property, were acquired by Axena Health, Inc.

Partner Therapeutics, Inc. is an oncology company based in Lexington, Massachusetts, dedicated to the development and commercialization of approved and late-stage cancer therapies. The company focuses on improving treatment outcomes and reducing overall healthcare costs for cancer patients through a comprehensive range of therapies, from primary treatments to supportive care. One of its key products is Leukine, which promotes the growth of white blood cells to aid in cancer treatment. Founded in 2017, Partner Therapeutics aims to connect investigators and healthcare professionals in the field of oncology, ensuring effective treatments are accessible to patients at all stages of their cancer journey.

Vapotherm, Inc. is a medical technology company specializing in the development and commercialization of proprietary Hi-VNI technology products designed to treat patients of all ages experiencing respiratory distress. Headquartered in Exeter, New Hampshire, the company offers a range of precision flow systems, including Precision Flow Hi-VNI, Precision Flow Plus, Precision Flow Classic, and Precision Flow Heliox, which deliver heated, humidified, and oxygenated air at high velocity through a small-bore nasal interface. Vapotherm also provides various companion products, such as the Vapotherm Transfer Unit for patient mobility, the Q50 compressor for compressed air, and aerosol solutions for medication delivery. Additionally, the company offers disposable products, including single-use nasal interfaces and Oxygen Assist Modules to help clinicians maintain target oxygen levels. Vapotherm generates revenue primarily through direct sales to hospitals in the United States and through distributors in select international markets. Founded in 1993, the company is committed to enhancing clinical outcomes and improving the quality of life for patients.

Xontogeny LLC is a life sciences accelerator based in Boston, Massachusetts, founded in 2016. The company specializes in the development of pharmaceuticals and related technologies, aiming to enhance the success rate of early-stage drug and technology ventures. Xontogeny provides entrepreneurs with essential leadership, strategic guidance, and operational support, fostering collaboration with founding scientists to realize their visions. By adopting a differentiated approach, Xontogeny supports efficient development models that benefit both company founders and early equity holders, thereby advancing innovation in the healthcare and life science sectors.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

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.

VBI Vaccines Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, with research facilities in Ottawa, Canada. It develops and sells vaccines for infectious diseases and immuno-oncology, with a focus on expanding protection in large, underserved markets. The company's enveloped virus-like particle (eVLP) platform enables the creation of vaccines that closely mimic target viruses, eliciting potent immune responses. VBI's commercial product, Sci-B-Vac, is a hepatitis B vaccine approved in Israel and 14 other countries, offering rapid onset of protection and high antibody levels. The company is also developing VBI-2601 for a functional cure of chronic hepatitis B, VBI-1901 for glioblastoma, and VBI-1501 for cytomegalovirus, among other candidates. VBI collaborates with pharmaceutical companies and research institutions, and its products are primarily distributed through direct sales to physicians and pharmacists.

VBI Vaccines Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, with research facilities in Ottawa, Canada. It develops and sells vaccines for infectious diseases and immuno-oncology, with a focus on expanding protection in large, underserved markets. The company's enveloped virus-like particle (eVLP) platform enables the creation of vaccines that closely mimic target viruses, eliciting potent immune responses. VBI's commercial product, Sci-B-Vac, is a hepatitis B vaccine approved in Israel and 14 other countries, offering rapid onset of protection and high antibody levels. The company is also developing VBI-2601 for a functional cure of chronic hepatitis B, VBI-1901 for glioblastoma, and VBI-1501 for cytomegalovirus, among other candidates. VBI collaborates with pharmaceutical companies and research institutions, and its products are primarily distributed through direct sales to physicians and pharmacists.

VBI Vaccines Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, with research facilities in Ottawa, Canada. It develops and sells vaccines for infectious diseases and immuno-oncology, with a focus on expanding protection in large, underserved markets. The company's enveloped virus-like particle (eVLP) platform enables the creation of vaccines that closely mimic target viruses, eliciting potent immune responses. VBI's commercial product, Sci-B-Vac, is a hepatitis B vaccine approved in Israel and 14 other countries, offering rapid onset of protection and high antibody levels. The company is also developing VBI-2601 for a functional cure of chronic hepatitis B, VBI-1901 for glioblastoma, and VBI-1501 for cytomegalovirus, among other candidates. VBI collaborates with pharmaceutical companies and research institutions, and its products are primarily distributed through direct sales to physicians and pharmacists.

Corbus Pharmaceuticals is a clinical-stage pharmaceutical company based in Norwood, Massachusetts, that focuses on developing and commercializing therapeutics for inflammatory and fibrotic diseases. Founded in 2009, the company’s lead product candidate, lenabasum, is a synthetic oral CB2 agonist currently undergoing Phase III clinical trials for systemic sclerosis and dermatomyositis, and Phase IIb trials for systemic lupus erythematosus and cystic fibrosis. In addition to lenabasum, Corbus is developing CRB-4001, a peripherally-restricted CB1 inverse agonist aimed at treating nonalcoholic steatohepatitis and other fibrotic conditions. The company is leveraging a diverse pipeline of drug candidates sourced from a library of over 600 novel compounds targeting the endocannabinoid system. Furthermore, Corbus has established collaborations for the development and commercialization of its products, including a partnership with Kaken Pharmaceutical Co., Ltd. for lenabasum in Japan.

AVEO Oncology is a commercial-stage biopharmaceutical company headquartered in Boston, Massachusetts, focused on developing and marketing targeted therapies for cancer. Its lead product, tivozanib, marketed as FOTIVDA, is an oral, once-daily vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor used for treating renal cell carcinoma (RCC). The company has completed a Phase III trial of tivozanib and is investigating its use in combination with the immune checkpoint inhibitor Opdivo. Additionally, AVEO is developing Ficlatuzumab, an antibody targeting hepatocyte growth factor, currently in Phase II trials for several cancers, and AV-203, an anti-ErbB3 monoclonal antibody that has completed Phase I trials for esophageal cancer. The company also has preclinical products aimed at cachexia and pulmonary arterial hypertension. AVEO collaborates with various pharmaceutical companies to enhance its research and development efforts. Established in 2001, AVEO Oncology aims to address unmet medical needs in oncology through its innovative therapies.

Vapotherm, Inc. is a medical technology company specializing in the development and commercialization of proprietary Hi-VNI technology products designed to treat patients of all ages experiencing respiratory distress. Headquartered in Exeter, New Hampshire, the company offers a range of precision flow systems, including Precision Flow Hi-VNI, Precision Flow Plus, Precision Flow Classic, and Precision Flow Heliox, which deliver heated, humidified, and oxygenated air at high velocity through a small-bore nasal interface. Vapotherm also provides various companion products, such as the Vapotherm Transfer Unit for patient mobility, the Q50 compressor for compressed air, and aerosol solutions for medication delivery. Additionally, the company offers disposable products, including single-use nasal interfaces and Oxygen Assist Modules to help clinicians maintain target oxygen levels. Vapotherm generates revenue primarily through direct sales to hospitals in the United States and through distributors in select international markets. Founded in 1993, the company is committed to enhancing clinical outcomes and improving the quality of life for patients.

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.

Minerva Neurosciences, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, dedicated to developing and commercializing therapies for central nervous system diseases. The company's lead product candidate, roluperidone, is in Phase III clinical trials aimed at treating schizophrenia. Additionally, Minerva is advancing seltorexant, which has completed Phase IIb trials for insomnia and major depressive disorders, and MIN-301, currently in pre-clinical trials for Parkinson's disease. Minerva has entered into a license agreement with Mitsubishi Tanabe Pharma Corporation for the global development of roluperidone, excluding Asia, and has a collaboration with Janssen Pharmaceutica for seltorexant. Founded in 2007 and formerly known as Cyrenaic Pharmaceuticals, Minerva Neurosciences focuses on providing innovative therapeutic options for patients with significant unmet medical needs in the CNS space.

Blueprint Medicines Corporation is a biopharmaceutical company that specializes in developing small molecule kinase inhibitors aimed at treating genomically defined cancers and rare diseases. Founded in 2008 and headquartered in Cambridge, Massachusetts, the company focuses on personalized cancer therapies by leveraging its proprietary chemical library and advanced research capabilities. Its key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, and pralsetinib for RET-altered non-small cell lung cancer and medullary thyroid carcinoma. Additionally, Blueprint Medicines is advancing BLU-263, a KIT inhibitor for mast cell disorders, and fisogatinib for advanced hepatocellular carcinoma, both currently in clinical trials. The company’s innovative approach seeks to address the molecular drivers of cancer and resistance mechanisms, aiming to enhance treatment effectiveness and patient outcomes. Blueprint Medicines has established collaborations with several pharmaceutical companies to further its research and development efforts.