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 the KCC2, a key mediator of inhibition in the brain. The company leverages groundbreaking research from institutions such as Boston Children’s Hospital, Harvard, and Laval University to create a proprietary neuron-specific K-Cl cotransporter discovery engine. This innovative platform aims to enhance the treatment of various conditions, including epilepsy, pain, and other central nervous system pathologies, by fine-tuning neuronal inhibition. Through its efforts, Axonis Therapeutics seeks to contribute to the healthcare industry's ability to combat neurodegeneration, restore neuronal excitation, and promote neuron regeneration, ultimately improving the quality of life for individuals affected by these disorders.

scPharmaceuticals Inc. is a pharmaceutical company focused on developing and commercializing innovative therapeutic products, particularly 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, aimed at treating congestion in patients with decompensated heart failure. Additionally, scPharmaceuticals is advancing its product pipeline with scCeftriaxone, an antibiotic targeting infections from both gram-positive and gram-negative organisms, and a carbapenem program for addressing gram-negative infections. Founded in 2013 and headquartered in Burlington, Massachusetts, scPharmaceuticals emphasizes the importance of subcutaneous drug administration, offering a convenient two-component delivery system that allows for patient self-administration, thereby providing a safer and more comfortable alternative to traditional intravenous methods. The company collaborates with West Pharmaceutical Services to enhance its SmartDose system, furthering its mission to improve patient outcomes.

CereVasc, Inc. is a medical device company based in Auburndale, Massachusetts, specializing in the development of innovative, minimally invasive treatments for neurological diseases. The company has created the eShunt System, an endovascularly deliverable cerebrospinal fluid (CSF) shunt designed to replace traditional surgical methods for treating communicating hydrocephalus. This system aims to eliminate the need for invasive surgery, general anesthesia, and hospitalization, which are typically associated with the conventional use of ventriculo-peritoneal shunts. By providing a less invasive alternative, CereVasc seeks to improve patient outcomes and reduce the overall costs related to the surgical management of life-threatening neurological conditions. Established in 2014, CereVasc is focused on advancing healthcare solutions that enhance the treatment of various neurological brain disorders.

Adaptilens, LLC is a Massachusetts-based company focused on the development of an innovative intraocular lens that provides patients with clear vision at varying distances without the need for eyeglasses or contact lenses. Founded in 2019, Adaptilens has created a physiologically adaptive lens that mimics the natural human lens, allowing the eye's ciliary muscles to adjust the shape of the lens for optimal focus on near, intermediate, and distant objects. This technology aims to enhance the quality of life for patients by enabling natural accommodation, thereby facilitating a seamless transition between different visual tasks.

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 markets ReSure Sealant, a hydrogel ophthalmic wound sealant designed to protect corneal incisions after cataract surgery. Its lead product candidate, DEXTENZA, is an ophthalmic insert that has completed Phase III clinical trials for post-surgical ocular pain, inflammation, and allergic conjunctivitis, and is also being investigated for dry eye diseases. Additionally, Ocular Therapeutix is developing several other products, including OTX-TP, an intracanalicular travoprost insert for glaucoma, and OTX-TKI, an intravitreal implant for wet age-related macular degeneration, among others. The company has established a strategic collaboration with Regeneron Pharmaceuticals to leverage its hydrogel technology in combination with Regeneron's VEGF-targeting compounds for retinal diseases. Founded in 2006 and headquartered in Bedford, Massachusetts, Ocular Therapeutix aims to address unmet needs in ophthalmology through its advanced therapeutic solutions.

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.

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 headquartered in Boston, Massachusetts, founded in 2021. The company specializes in the development and commercialization of innovative vaccines. Its primary focus is on HIL-214, a virus-like particle (VLP) based vaccine candidate aimed at preventing moderate-to-severe acute gastroenteritis (AGE) caused by norovirus infection. HilleVax is dedicated to addressing significant public health challenges through its vaccine initiatives.

Emulate, Inc. is a biotechnology company specializing in organs-on-chips technology that emulates human biology to enhance the understanding of diseases, medicines, chemicals, and food impacts on human health. The company develops organ-chips that replicate normal organ functions as well as various disease states, primarily targeting the lung, intestine, liver, and skin. Emulate's innovative human emulation system provides researchers with advanced in vitro models that offer more precise and detailed predictions of human responses compared to traditional experimental methods, such as cell culture or animal testing. The company serves a diverse range of markets, including biotechnology, pharmaceuticals, consumer health, cosmetics, chemicals, food, agrochemicals, government agencies, and academic research. Founded in 2013 and based in Boston, Massachusetts, Emulate was previously known as Emulate Living MicroDevices, Inc. before rebranding in July 2014.

Amylyx Pharmaceuticals, Inc. is a biotechnology company focused on developing innovative therapies for neurodegenerative diseases, particularly Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease, and Wolfram syndrome. Founded in 2013 and based in Cambridge, Massachusetts, the company is best known for its investigational therapy AMX0035, a fixed-dose co-formulation of sodium phenylbutyrate and Taurursodiol. This treatment aims to address the energy crisis within mitochondria and the accumulation of toxic, unfolded proteins in the endoplasmic reticulum, both of which contribute to the progression of neurodegenerative conditions. Through its research and development efforts, Amylyx seeks to provide effective solutions to mitigate the suffering caused by these debilitating diseases.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

Renovia Inc., founded in 2016 and based in Boston, Massachusetts, was a healthcare company focused on developing prescription digital therapeutics for pelvic floor disorders. The company's innovative device provided a clinically validated treatment for conditions such as stress, mixed, and urgent urinary incontinence, including overactive bladder. This technology allowed for real-time visualization of pelvic movement during muscle training sessions, facilitating a non-surgical and drug-free approach to treatment. However, Renovia ceased operations in late 2022 following a strategic review by its investors, and in January 2023, the majority of its assets, including 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, specializing in the development of vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. Founded in 2014, the company aims to enhance the protection against a range of infectious diseases that affect both children and adults globally. MAPS technology allows for the effective binding of protective polysaccharides and proteins within a single vaccine, inducing robust immune responses. Affinivax is advancing vaccine programs targeting significant pathogens, including Streptococcus pneumoniae, which contributes to considerable morbidity and mortality worldwide. The company leverages insights from leading experts in infectious diseases and has secured exclusive intellectual property rights related to its MAPS platform from Boston Children’s Hospital, with notable backing from the Bill & Melinda Gates Foundation.

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 focused on revolutionizing cancer diagnosis through its innovative blood testing platform. The company specializes in CancerSEEK, a liquid biopsy test that analyzes genomic mutations in tumor DNA and protein markers in plasma to detect various types of cancer at their earliest stages, even before symptoms arise. Founded in 2018 and headquartered in Cambridge, Massachusetts, with an additional office in Baltimore, Maryland, Thrive aims to integrate earlier cancer detection into routine medical care. The company's approach combines targeted DNA and protein analysis with real-world data and machine learning to provide comprehensive result interpretation and facilitate follow-up testing and oncology care. Thrive Earlier Detection Corp. operates as a subsidiary of Exact Sciences Corporation, reflecting its commitment to enhancing cancer detection and improving patient outcomes.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Pear Therapeutics, Inc. is a Boston-based commercial-stage healthcare company specializing in software-based digital therapeutics, also known as Prescription Digital Therapeutics (PDTs). Founded in 2013, the company develops clinically validated software solutions designed to treat a range of behavioral health disorders, including addiction, anxiety, depression, and chronic insomnia. Its suite of products includes mobile digital therapies that reward patients for engagement, and specific tools like reSET and reSET-O, aimed at treating substance use disorders. The company also offers Somryst, which provides neurobehavioral interventions for insomnia, and operates PearConnect, a patient services center that connects patients with advocates and resources, ensuring support throughout their treatment journey. Pear Therapeutics aims to enhance treatment outcomes by integrating digital therapies with pharmaceuticals and providing comprehensive support for patients, clinicians, and payors.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates aimed at targeting and eliminating disease-causing proteins for the treatment of cancer, neurodegenerative diseases, and other conditions. The company's lead product candidate, CFT7455, is an orally bioavailable degrader targeting IKZF1/3, designed to treat multiple myeloma and various lymphomas. Additionally, C4 Therapeutics is advancing CFT8634, which targets BRD9 for synovial sarcoma and SMARCB1-deleted solid tumors, as well as programs targeting BRAF V600E and RET for genetically defined resistant solid tumors. The company employs its proprietary Degronimid platform, which utilizes small molecule binders to flag harmful proteins for degradation by the cellular proteasome system, allowing for the potential treatment of previously undruggable targets. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics engages in strategic collaborations to enhance its research and development efforts.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates aimed at targeting and eliminating disease-causing proteins for the treatment of cancer, neurodegenerative diseases, and other conditions. The company's lead product candidate, CFT7455, is an orally bioavailable degrader targeting IKZF1/3, designed to treat multiple myeloma and various lymphomas. Additionally, C4 Therapeutics is advancing CFT8634, which targets BRD9 for synovial sarcoma and SMARCB1-deleted solid tumors, as well as programs targeting BRAF V600E and RET for genetically defined resistant solid tumors. The company employs its proprietary Degronimid platform, which utilizes small molecule binders to flag harmful proteins for degradation by the cellular proteasome system, allowing for the potential treatment of previously undruggable targets. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics engages in strategic collaborations to enhance its research and development efforts.

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 based in Auburndale, Massachusetts, specializing in the development of innovative, minimally invasive treatments for neurological diseases. The company has created the eShunt System, an endovascularly deliverable cerebrospinal fluid (CSF) shunt designed to replace traditional surgical methods for treating communicating hydrocephalus. This system aims to eliminate the need for invasive surgery, general anesthesia, and hospitalization, which are typically associated with the conventional use of ventriculo-peritoneal shunts. By providing a less invasive alternative, CereVasc seeks to improve patient outcomes and reduce the overall costs related to the surgical management of life-threatening neurological conditions. Established in 2014, CereVasc is focused on advancing healthcare solutions that enhance the treatment of various neurological brain disorders.

Atea Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on discovering and developing next-generation antiviral therapeutics for severe viral diseases. 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 the SARS-CoV-2 virus. Additionally, Atea is advancing several candidates in clinical trials, including AT-787 for hepatitis C and AT-752 for dengue, both in phase 2 trials. The company aims to provide innovative treatments for infections caused by RNA and DNA viruses, improving outcomes for patients suffering from serious viral infections. Atea Pharmaceuticals was incorporated in 2012.

Quellis Biosciences, Inc. is a biopharmaceutical company based in Waltham, Massachusetts, established in 2017. The company is committed to discovering and developing novel monoclonal antibody therapies aimed at treating rare diseases, particularly for patients who are underserved by existing treatment options. Quellis focuses on optimizing multiple therapeutic leads to identify effective antibodies that can enhance patient outcomes, thus striving to improve the quality of life for individuals facing serious health challenges. Through its innovative approach, Quellis aims to deliver best-in-class therapies that address significant unmet medical needs in the realm of 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.

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, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

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 focused on developing novel antisense oligonucleotide medicines aimed at addressing the underlying causes of severe genetic diseases. Founded in 2014 and headquartered in Bedford, Massachusetts, the company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to enhance gene expression through RNA splicing. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics has also partnered with Invitae Corporation to offer complementary genetic testing services.

Renovia Inc., founded in 2016 and based in Boston, Massachusetts, was a healthcare company focused on developing prescription digital therapeutics for pelvic floor disorders. The company's innovative device provided a clinically validated treatment for conditions such as stress, mixed, and urgent urinary incontinence, including overactive bladder. This technology allowed for real-time visualization of pelvic movement during muscle training sessions, facilitating a non-surgical and drug-free approach to treatment. However, Renovia ceased operations in late 2022 following a strategic review by its investors, and in January 2023, the majority of its assets, including 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.

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, Inc. is a clinical-stage pharmaceutical company based in Norwood, Massachusetts, focused on developing and commercializing therapeutics for the treatment of rare and serious inflammatory fibrotic diseases. Founded in 2009, the company’s lead product candidate, lenabasum, is a synthetic, orally administered cannabinoid receptor type 2 (CB2) agonist currently undergoing Phase III clinical trials for systemic sclerosis and dermatomyositis, as well as Phase IIb trials for systemic lupus erythematosus and cystic fibrosis. Additionally, Corbus is developing CRB-4001, a peripherally-restricted cannabinoid receptor type 1 (CB1) inverse agonist, aimed at treating nonalcoholic steatohepatitis and other fibrotic conditions. The company has a strategic collaboration with Kaken Pharmaceutical Co., Ltd. for the commercialization of lenabasum in Japan and has a licensing agreement with Jenrin Discovery, LLC to utilize a library of novel compounds targeting the endocannabinoid system. With a commitment to addressing chronic inflammation and fibrotic diseases, Corbus Pharmaceuticals is advancing its pipeline of innovative drug candidates.

AVEO Oncology is a biopharmaceutical company that specializes in developing and commercializing targeted therapies for oncology and other unmet medical needs. The company is known for its lead product, FOTIVDA (tivozanib), an oral vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor used in the treatment of renal cell carcinoma (RCC). AVEO has completed a Phase III trial for tivozanib and is exploring its use in combination with the immune checkpoint inhibitor Opdivo. Additionally, the company is developing other therapeutic candidates, including Ficlatuzumab, an antibody for various cancers, and AV-203, a monoclonal antibody for esophageal cancer, which has completed Phase I trials. AVEO's preclinical pipeline includes promising products aimed at treating conditions such as cachexia and pulmonary arterial hypertension. Headquartered in Boston, Massachusetts, the company was founded in 2001 and has established collaborations with various pharmaceutical firms to enhance 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.

Minerva Neurosciences, Inc. is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative treatments for central nervous system diseases. The company is advancing a diverse portfolio of product candidates, with its lead compound, roluperidone, currently in Phase III clinical trials for schizophrenia. Additionally, Minerva is developing seltorexant, which has completed Phase IIb trials for insomnia and major depressive disorders, and MIN-301, a soluble recombinant form of the neuregulin-1b1 protein in pre-clinical trials aimed at treating Parkinson’s disease. Minerva has established strategic partnerships, including a licensing agreement with Mitsubishi Tanabe Pharma Corporation for roluperidone outside Asia, and a co-development agreement with Janssen Pharmaceutica for seltorexant. Originally founded in 2007 as Cyrenaic Pharmaceuticals, the company rebranded to Minerva Neurosciences in 2013 and is headquartered in Waltham, Massachusetts.

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.