Immunocore Limited is a biotechnology company specializing in the discovery and development of innovative T cell receptor-based therapeutics aimed at treating cancer, viral diseases, and autoimmune disorders. Founded in 1999 and headquartered in Abingdon, United Kingdom, with an additional office in Conshohocken, the company has developed a proprietary technology platform that enables the engineering of T cell receptors (TCRs) linked to antibody fragments. This expertise has led to the advancement of IMCgp100, a bi-specific immunotherapeutic currently undergoing clinical trials for metastatic melanoma. Immunocore's focus on addressing diseases with significant unmet medical needs underscores its commitment to advancing novel treatment options.
Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.
MoMa Therapeutics, Inc. discovers precision medicines by targeting the molecular machines that underlie human disease. MoMa Therapeutics, Inc. was formerly known as ATPases NewCo, Inc. and changed its name to MoMa Therapeutics, Inc. in March 2020. The company was founded in 2019 and is based in Cambridge, Massachusetts.
MiRXES Pte. Ltd. develops life science research tools and molecular diagnostic tests or research and clinical diagnostic use. It offers RT-qPCR assays, assay panels, spike-in RNA kits, and biomarker discovery solutions. The company was founded in 2014 and is based in Singapore.
Simcha Therapeutics is a biopharmaceutical company focused on developing engineered cytokine immunotherapies for cancer treatment. Based in New Haven, Connecticut, the company employs directed evolution to create novel cytokines aimed at enhancing the immune system's ability to combat diseases. Its lead program, ST-067, is a designer IL-18 cytokine that has demonstrated significant antitumor activity in animal studies, both as a standalone treatment and in combination with anti-PD-1 checkpoint inhibitors. Founded in 2018, Simcha Therapeutics is dedicated to advancing innovative therapies that empower patients to effectively fight cancer.
AvengeBio is focusing on Cell-Generated Immunotherapies to eradicate solid tumors. It is a developer of a drug delivery platform intended to treat cancer. The company was founded in 2019 and based in Cambridge, Massachusetts.
CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, a novel aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its promising clinical candidates, CinCor Pharma seeks to gain marketing approval for innovative therapies that can effectively address unmet medical needs in these critical areas of health.
Private Equity Round in 2021
Aadi Bioscience, Inc. is a clinical stage biopharmaceutical company based in Pacific Palisades, California, founded by Dr. Neil Desai in 2011. The company focuses on the development of a potentially best-in-class mTOR inhibitor, specifically ABI-009, aimed at treating patients with oncology, cardiovascular, and metabolic diseases. Aadi Bioscience is dedicated to addressing unmet medical needs through its innovative therapeutic approaches in these areas.
Disc Medicine, Inc. is a biotechnology company focused on developing therapeutics based on human genetics to address ineffective red blood cell production in hematologic diseases. The company specializes in programs targeting the hepcidin metabolism axis for the treatment of disorders related to hepcidin. Founded in 2017 and headquartered in Cambridge, Massachusetts, Disc Medicine is led by a team of experienced scientists and industry experts, supported by a panel of advisors with a proven history in discovering and developing innovative therapies. The company was established with backing from Atlas Venture, along with contributions from Novo Ventures and Access Biotechnology.
Vigil will use the Series A funds to progress its lead pipeline candidate, a monoclonal antibody TREM2 agonist, through Phase 1 studies, advance their small molecule TREM2 agonist to IND, as well as pursue additional assets to grow the pipeline and increase the body of data supporting microglia biology as an important therapeutic pathway.
Vedanta Biosciences is a developer of immunotherapies designed to treat immune-mediated and infectious diseases. The company's immunotherapies include a suite of proprietary assays to select pharmacologically potent strains, vast proprietary datasets from human interventional studies, and facilities for cGMP-compliant manufacturing of rationally-defined bacterial consortia in powder form, enabling physicians to access live bacteria drug to treat autoimmune and inflammatory diseases easily.
It was founded in 2010 and is headquartered in Cambridge, Massachusetts.
Amylyx Pharmaceuticals, Inc. develops novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. It offers AMX0035, an investigational therapy and a fixed-dose co-formulation of two active compounds, such as sodium phenylbutyrate and Taurursodiol optimized to treat both the energy crisis in the mitochondria and the toxic, unfolded proteins in the endoplasmic reticulum, disrupting the neurological chain of events that leads to patient suffering. The company’s pipeline includes amyotrophic lateral sclerosis, Wolfram syndrome, and Alzheimer’s diseases. Amylyx Pharmaceuticals, Inc. was founded in 2013 and is based in Cambridge, Massachusetts.
Imperative Care, Inc. is dedicated to enhancing the speed and effectiveness of acute stroke treatment through innovative medical technologies. Founded in 2015 and based in Campbell, California, the company has developed a range of solutions aimed at addressing critical needs in stroke care. Among its offerings is the Large Distal Platform, a large diameter catheter designed to reach treatment sites for both ischemic and hemorrhagic strokes. Complementing this is the ZOOM Aspiration System, a comprehensive solution that works in conjunction with the Large Distal Platform and includes various trackable reperfusion catheters for effective clot removal. By advancing stroke treatment technologies, Imperative Care aims to improve patient outcomes and optimize recovery processes for those affected by strokes.
dMed Company Limited, also known as dMed Biopharmaceutical Co., Ltd., is a clinical contract research organization based in Shanghai, China. Established in 2016, dMed specializes in offering a comprehensive range of services to support the development of medicines and medical devices. Its offerings include consulting, clinical operations, biostatistics and programming, data management, pharmacovigilance, clinical science and medical affairs, quality assurance, and regulatory affairs and strategy. The company serves a diverse clientele, including biopharmaceutical and medical device companies, and aims to establish itself as a prominent international brand in the field of drug innovation services and solutions.
MiRXES Pte. Ltd. develops life science research tools and molecular diagnostic tests or research and clinical diagnostic use. It offers RT-qPCR assays, assay panels, spike-in RNA kits, and biomarker discovery solutions. The company was founded in 2014 and is based in Singapore.
Clinipace Inc. is a clinical research organization that provides integrated clinical research services to pharmaceutical, biotechnology, and medical device companies in the United States and globally. Founded in 2003 and headquartered in Morrisville, North Carolina, Clinipace specializes in various therapeutic areas, including oncology, nephrology, gastroenterology, cardiology, diabetes, and respiratory diseases. The company offers a comprehensive range of services such as biostatistics, clinical monitoring, patient recruitment, regulatory affairs, and legal representation. With additional offices in Boulder, Colorado, and international locations in Argentina, Brazil, Europe, and the Asia Pacific, Clinipace emphasizes collaboration and flexibility in its approach, distinguishing itself from traditional clinical research organizations.
Apollo Therapeutics is a collaborative initiative involving three prominent UK universities—Imperial College London, University College London, and the University of Cambridge—and three major pharmaceutical companies: AstraZeneca, GlaxoSmithKline, and Johnson & Johnson Innovation. The organization focuses on funding and developing novel therapeutics derived from leading British academic research, facilitating their progression toward clinical application. With a model that provides rapid access to resources, Apollo Therapeutics champions innovative research and enhances collaboration between academia and industry. Its team of experienced scientists, who have a strong track record in therapeutic delivery, works closely with academic researchers and industry partners to tailor projects for optimal success. The overarching goal of Apollo Therapeutics is to advance future therapies that significantly improve patient treatments and outcomes.
BioTheryX, Inc. is a biopharmaceutical company based in Chappaqua, New York, that specializes in the development of therapies for hematological malignancies and other diseases. The company focuses on restoring protein homeostasis through innovative approaches such as Protein Homeostatic Modulators, which are small molecular glue cereblon binders with significant therapeutic potential. Additionally, BioTheryX is advancing targeted protein degradation technologies, including PROTACs, which utilize dual ligands to facilitate the degradation of specific proteins. The company also offers BTX-A51, an oral small molecule that acts as a multi-kinase inhibitor to target leukemic stem cells and suppress key oncogenic gene transcription. Founded in 2007, BioTheryX aims to expedite the delivery of effective therapies to patients with unmet medical needs by leveraging enhanced biology-driven models and clinically proven translational approaches.
Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing therapies to target and destroy cancer cells using viral nanoparticle conjugates. The company’s lead product, AU-011, utilizes viral nanoparticles modeled on the human papillomavirus, conjugated with infrared-activated small molecules, specifically for the treatment of ocular melanoma, a primary eye cancer. By employing nanotechnology, Aura aims to safely eliminate cancer locally, particularly in early-stage cases, to prevent progression to more severe forms of the disease, such as metastasis to the liver. Additionally, the company is advancing its bel-sar candidate for treating primary choroidal melanoma and exploring applications in bladder cancer. Founded in 2007, Aura Biosciences is committed to developing precision immunotherapies that preserve the function of organs affected by cancer.
Xilio Therapeutics is a biotechnology company advancing next-generation cancer immunotherapies designed to improve patient outcomes by unleashing the power of the immune system selectively at the site of the tumor. The company’s tumor-selective immunotherapies are based on its proprietary technology, which maximizes the potency of proven immuno-oncology therapies and restricts their activity to the tumor to minimize peripheral side effects. The broad applicability of these therapies across cancer types means that all patients could benefit from these potentially curative medicines. These tumor-selective therapies are designed to overcome the significant toxicities associated with validated IO therapies, such as IL-2 and aCTLA4, which have historically limited the number of patients that can be treated and prevented patients from completing full courses of treatment Xilio Therapeutics proprietary technology enables the explosion of therapeutic activity specifically within the tumor while reducing toxicity throughout the body a nod to the company name, Xilio, derived from the Latin term, Ex Nihilo, meaning creation or big-bang. The company applying its technology to build a broad pipeline of engineered antibodies, cytokines and chemokines as potential new options for people living with cancer. Xilio Therapeutics was founded in 2016 and is headquartered in Waltham, Massachusetts, United States.
Sagimet Biosciences is a biotechnology company that focuses on developing novel therapeutics to treat important diseases such as liver disease NASH and specific cancers, with a focus on targeting dysfunctional metabolic pathways. It was founded in 2007 and headquartered in San Mateo, California.
Personal Genome Diagnostics develops a patient-specific analysis of the cancer genome by using advanced tools and technologies. They were the first to sequence and analyze whole human cancer exomes and subsequently identified many alterations responsible for the development of cancer. They have described the principles underlying the genetic basis of human cancer and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Through PGDx, our founders bring their proprietary methods and expertise to research teams worldwide. The services help oncologists and translational scientists sequence and analyze cancer genomes and identify mutations to characterize aspects of the disease. Their expertise in genome analysis ranges from sample preparation and sequencing to data interpretation and analysis. They specialize in high-throughput next-generation sequencing and proprietary algorithms to identify alterations in complex cancer genomes and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Personal Genome Diagnostics was founded in 2010 and is based in Baltimore, Maryland, United States.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
Biomea Fusion is a preclinical-stage biopharmaceutical company dedicated to advancing precision medicine for cancer treatment. The company's focus is on the discovery and development of irreversible small molecule drugs that target specific genetic alterations associated with tumor growth. Its lead product candidate, BMF-219, is designed as a potent and selective irreversible inhibitor of menin, a key transcriptional regulator involved in oncogenic signaling across various cancers. Biomea Fusion aims to expedite the development of these innovative therapies to provide highly effective treatment options for patients with genetically defined cancers.
Immuneering Corporation is a bioinformatics company based in Cambridge, Massachusetts, with an additional office in New York. Established in 2008, it specializes in genetic, genomic, and proteomic data analysis services aimed at enhancing the clinical and commercial success of medicines for serious diseases, including cancer, autoimmune disorders, and neurodegeneration. Immuneering employs advanced analytical techniques such as RNA sequencing, exome sequencing, and microarray analysis to uncover difficult-to-detect biological signals, generate hypotheses, and design follow-up studies. The company's proprietary Disease Cancelling Technology enables it to develop innovative drug candidates targeting conditions often overlooked by traditional drug discovery methods, including cancer cachexia and metastasis. In addition to its internal drug development efforts, Immuneering offers computational biology services to leading pharmaceutical and biotechnology companies, leveraging its expertise to improve patient outcomes across various debilitating conditions.
Cullinan Pearl, a subsidiary of Cullinan Oncology, focuses on developing innovative cancer therapies, specifically targeting Epidermal Growth Factor Receptor (EGFR) exon 20 mutations through its orally available tyrosine kinase inhibitor. The company is part of a broader strategy to create a diverse portfolio of oncology therapeutics, relying on a combination of internal research and collaborative efforts with academic institutions and pharmaceutical companies. This approach allows Cullinan Pearl to efficiently advance its drug development programs, with a commitment to rapidly discontinue any projects that show limited potential based on early research findings. As part of its ongoing initiatives, Cullinan Pearl collaborates with Cullinan Oncology to support the development and commercialization of its therapies.
Reneo Pharmaceuticals develops therapies for patients with rare genetic mitochondrial diseases, which impact cellular metabolism and energy production. Founded in 2014 and based in San Diego, California, the company is focused on creating treatments that enhance mitochondrial function, thereby improving muscle health and overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes essential for energy metabolism and the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. By addressing the underlying metabolic deficiencies in these patients, Reneo aims to prevent muscle injury, weakness, and wasting, ultimately enhancing daily functioning for those with these orphan diseases.
Acumen Pharmaceuticals is a biotechnology company dedicated to discovering and developing therapeutics and diagnostics for Alzheimer’s disease and related neurodegenerative conditions. Founded in 1996 and based in Livermore, California, the company focuses on creating ADDL-Select antibodies that specifically target soluble amyloid beta oligomers, which are considered major contributors to the progression of Alzheimer’s. Acumen employs a sensitivity assay to measure levels of these oligomers in cerebrospinal fluid, aiding in the selection of patients for clinical trials. The company holds a comprehensive portfolio of patents and intellectual property related to amyloid beta soluble oligomers. It has formed a partnership with Merck & Co. for the development of certain monoclonal antibody therapeutics. Additionally, Acumen has attracted investments from firms such as NeuroVentures LLC and Biotechnology Value Fund LP.
Flame Biosciences, Inc. is a clinical-stage biotechnology company based in New York that specializes in the research, development, and commercialization of innovative therapies targeting cancer and inflammatory diseases. Established in 2017, the company is advancing its FL-101 therapy, designed to treat lung cancers associated with inflammation, and FL-201, aimed at alleviating chronic inflammation in patients. By leveraging the emerging field of inflammasome science, Flame Biosciences seeks to provide effective treatments that enhance the quality of life for individuals affected by serious health conditions.
E-Scape Bio, Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapies for neurodegenerative diseases. Established in 2015 and headquartered in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer’s disease, Parkinson’s disease, and sphingolipid storage disorders. E-Scape Bio is advancing a pipeline of therapeutics that target specific genetic drivers of these diseases, including ESB1609, a small molecule S1P5 receptor agonist for CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor designed for Parkinson’s disease patients with the LRRK2 G2019S variant. Additionally, the company is developing a program targeting ApoE4 in Alzheimer’s disease, emphasizing its commitment to precision medicine in treating genetically defined subpopulations.
Chinook Therapeutics is a biotechnology company that specializes in the fields of therapeutics and life science. The company focuses on developing precision medicines for kidney diseases. Its services offers utilization of novel translational platforms and patient stratification tools, and emergence of accelerated regulatory pathways based on surrogate endpoints.
The company was founded in 2019 and headquartered in Alberta, Canada.
Cerevel (www.cerevel.com) is a biopharmaceutical company focused on developing drug candidates to treat disorders of the central nervous system (CNS). The company has a portfolio of pre-commercial neuroscience assets, which include three clinical-stage compounds and several pre-clinical compounds designed to target a broad range of CNS disorders including Parkinson’s, Alzheimer’s, epilepsy, schizophrenia and addiction. Headquartered in the Greater Boston area, Cerevel was formed in 2018 through a partnership between Bain Capital and Pfizer.
Thrive Earlier Detection Corp. is a healthcare company dedicated to improving cancer detection through innovative technology. It focuses on the development of CancerSEEK, a liquid biopsy test that analyzes genomic mutations in tumor DNA and protein markers in plasma to identify multiple types of cancer at their earliest stages, often before symptoms become apparent. Founded in 2018 and headquartered in Cambridge, Massachusetts, with an additional office in Baltimore, Maryland, Thrive aims to integrate early cancer detection into routine medical care. The company was previously known as Thrive Sciences Inc. and has been a subsidiary of Exact Sciences Corporation since early 2021. Thrive was launched with support from various investors, including Third Rock Ventures and Section 32, reflecting a strong commitment to advancing cancer diagnostics.
Bolt Biotherapeutics is a biotechnology company focused on developing innovative cancer treatments through its Boltbody platform, which centers on immune-stimulating antibody conjugates (ISAC). This platform combines tumor-targeting antibodies with potent immune stimulants, such as TLR agonists, to transform cold tumors into immunologically active ones and facilitate tumor elimination. One of the company's key developments is BDC-1001, designed as a monotherapy for patients with HER2-expressing solid tumors. Founded in 2015 and headquartered in Redwood City, California, Bolt Biotherapeutics aims to advance cancer immunotherapy and improve patient outcomes. The company was previously known as Bolt Therapeutics before rebranding in July 2015.
Athira Pharma is a clinical-stage drug development company striving to improve human health by advancing bold and innovative therapies with the potential to restore the lives of people impacted by brain disorders. Athira is derived from the word Athir, the energy that reaches everyone. It captures its mission to develop therapies that can reach and positively impact everyone.
Everest Medicines Limited, a clinical-stage biopharmaceutical company, focuses on licensing, developing, and commercializing therapies for addressing critical unmet medical needs in Greater China and other Asia Pacific markets. It has a portfolio of eight clinical-stage drug candidates for the treatment of oncology, immunology, cardio-renal disease, and infectious diseases. The company was founded in 2017 and is headquartered in Shanghai, China.
MoMa Therapeutics, Inc. discovers precision medicines by targeting the molecular machines that underlie human disease. MoMa Therapeutics, Inc. was formerly known as ATPases NewCo, Inc. and changed its name to MoMa Therapeutics, Inc. in March 2020. The company was founded in 2019 and is based in Cambridge, Massachusetts.
Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, established in 2015. It specializes in targeted protein degradation, developing innovative small molecule therapeutics that selectively degrade proteins associated with various diseases by utilizing the body's natural protein degradation mechanisms. The company is advancing several therapeutic programs, including the IRAK4 program aimed at treating immunology-inflammation diseases such as hidradenitis suppurativa, atopic dermatitis, and rheumatoid arthritis. Additionally, it is developing the IRAKIMiD program for MYD88-mutated diffuse large B cell lymphoma and the STAT3 program for hematologic malignancies and solid tumors, as well as autoimmune diseases. Through its proprietary predictive modeling platform, Kymera Therapeutics seeks to accelerate drug discovery and address previously untreatable conditions.
Immunocore Limited is a biotechnology company specializing in the discovery and development of innovative T cell receptor-based therapeutics aimed at treating cancer, viral diseases, and autoimmune disorders. Founded in 1999 and headquartered in Abingdon, United Kingdom, with an additional office in Conshohocken, the company has developed a proprietary technology platform that enables the engineering of T cell receptors (TCRs) linked to antibody fragments. This expertise has led to the advancement of IMCgp100, a bi-specific immunotherapeutic currently undergoing clinical trials for metastatic melanoma. Immunocore's focus on addressing diseases with significant unmet medical needs underscores its commitment to advancing novel treatment options.
Immunocore Limited is a biotechnology company specializing in the discovery and development of innovative T cell receptor-based therapeutics aimed at treating cancer, viral diseases, and autoimmune disorders. Founded in 1999 and headquartered in Abingdon, United Kingdom, with an additional office in Conshohocken, the company has developed a proprietary technology platform that enables the engineering of T cell receptors (TCRs) linked to antibody fragments. This expertise has led to the advancement of IMCgp100, a bi-specific immunotherapeutic currently undergoing clinical trials for metastatic melanoma. Immunocore's focus on addressing diseases with significant unmet medical needs underscores its commitment to advancing novel treatment options.
Spruce Biosciences, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for rare endocrine disorders. Founded in 2014 and based in Daly City, California, the company is primarily focused on its lead candidate, tildacerfont, which is being evaluated in Phase II clinical trials for classic congenital adrenal hyperplasia (CAH) in both adults and children. Tildacerfont aims to be the first non-steroidal treatment that enhances disease control while minimizing the reliance on steroids for managing CAH. Additionally, Spruce Biosciences is exploring tildacerfont's potential applications in treating polycystic ovary syndrome in women. The company has also entered a licensing agreement with Eli Lilly and Company to research and develop compounds for various pharmaceutical applications. Through its efforts, Spruce Biosciences seeks to significantly improve the quality of life for patients with rare endocrine disorders who have historically been underserved by medical advancements.
Imperative Care, Inc. is dedicated to enhancing the speed and effectiveness of acute stroke treatment through innovative medical technologies. Founded in 2015 and based in Campbell, California, the company has developed a range of solutions aimed at addressing critical needs in stroke care. Among its offerings is the Large Distal Platform, a large diameter catheter designed to reach treatment sites for both ischemic and hemorrhagic strokes. Complementing this is the ZOOM Aspiration System, a comprehensive solution that works in conjunction with the Large Distal Platform and includes various trackable reperfusion catheters for effective clot removal. By advancing stroke treatment technologies, Imperative Care aims to improve patient outcomes and optimize recovery processes for those affected by strokes.
Fusion Pharmaceuticals Inc., a clinical-stage oncology company, focuses on developing radiopharmaceuticals as precision medicines. The company has developed Targeted Alpha Therapies platform together with its proprietary Fast-Clear linker technology to enable us to connect alpha particle emitting isotopes to antibodies and other targeting molecules in order to selectively deliver the alpha particle payloads to tumors. Its lead product candidate is FPI-1434 that is in Phase 1 clinical trials as a monotherapy in patients with solid tumors expressing insulin-like growth factor 1 receptor. The company is also conducting additional preclinical studies of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors to further assess the anti-tumor activity, and dosing schedule and pharmacodynamics of the combinations. In addition, it is progressing its earlier-stage product candidate, FPI-1966 into clinical development for the treatment of head and neck, and bladder cancers expressing fibroblast growth factor receptor. The company was founded in 2014 and is headquartered in Hamilton, Canada.
Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.
Vedanta Biosciences is a developer of immunotherapies designed to treat immune-mediated and infectious diseases. The company's immunotherapies include a suite of proprietary assays to select pharmacologically potent strains, vast proprietary datasets from human interventional studies, and facilities for cGMP-compliant manufacturing of rationally-defined bacterial consortia in powder form, enabling physicians to access live bacteria drug to treat autoimmune and inflammatory diseases easily.
It was founded in 2010 and is headquartered in Cambridge, Massachusetts.
Akero Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing treatments for serious metabolic diseases, particularly nonalcoholic steatohepatitis (NASH). Founded in 2017, the company aims to address high unmet medical needs associated with NASH, a condition characterized by liver inflammation and damage that can lead to severe complications. Akero's lead product candidate, AKR-001, is a long-acting Fc fusion modified FGF21 protein currently undergoing Phase IIa clinical trials. This candidate is designed to restore metabolic balance by reducing liver fat and suppressing inflammation and fibrosis, thereby potentially halting the progression of NASH. The development of AKR-001 builds upon extensive research into FGF21 biology, positioning it as a promising treatment option in a field lacking approved therapies.
Aprea Therapeutics, Inc. is a biopharmaceutical company focused on developing and commercializing cancer therapeutics that target the mutant p53 tumor suppressor protein. The lead product candidate, APR-246, is a first-in-class small molecule p53 reactivator currently in late-stage clinical development for hematologic malignancies, specifically myelodysplastic syndromes and acute myeloid leukemia. Founded in 2006 and headquartered in Boston, Massachusetts, Aprea aims to address significant unmet medical needs in cancer treatment through innovative therapies. The company is primarily backed by KDev Investments AB and other stakeholders, including Östersjöstiftelsen and Praktikerinvest, reflecting a strong investment foundation for its research and development efforts.
Mirum Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapies for debilitating liver diseases. It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases.
Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics that aim to enhance the effectiveness of radiotherapy in cancer treatment. The company’s lead product candidate, avasopasem manganese (GC4419), is a small molecule mimetic of superoxide dismutase designed to address radiation-induced severe oral mucositis in patients with head and neck cancer. It is currently undergoing Phase III clinical trials for this indication and Phase IIa trials for treating radiotherapy-induced esophagitis in lung cancer patients. Additionally, Galera is developing a second candidate, GC4711, which is in Phase I/II trials for pancreatic and lung cancers. Founded in 2012 and headquartered in Malvern, Pennsylvania, Galera Therapeutics is dedicated to transforming cancer treatment through advancements in oxygen metabolic pathways.
PhaseBio Pharmaceuticals is a clinical-stage biopharmaceutical company based in Malvern, Pennsylvania, focused on developing innovative therapies for cardiopulmonary diseases. Its lead product candidate, PB2452, is a reversal agent for the antiplatelet drug ticagrelor and is currently undergoing Phase III clinical trials for patients experiencing uncontrolled bleeding or requiring urgent surgery. The company is also advancing PB1046, a fusion protein aimed at treating pulmonary arterial hypertension, which is in Phase IIb clinical trials. Additionally, PhaseBio is developing PB6440, an oral agent intended for the treatment of resistant hypertension. Founded in 2002, the company leverages proprietary recombinant biopolymers to enhance the stability and efficacy of its drug candidates.
Kaleido Biosciences is a clinical-stage healthcare company based in Lexington, Massachusetts, dedicated to developing microbiome metabolic therapies (MMTs) aimed at treating various diseases and improving human health. Founded in 2015, the company is advancing a diverse pipeline of MMT candidates targeting conditions such as urea cycle disorders, hepatic encephalopathy, multi-drug resistant infections, cardiometabolic and liver diseases, and immune oncology. Kaleido employs a unique, chemistry-driven approach to harness the microbiome's potential, focusing on modulating its metabolic output and profile by influencing the existing microbial functions. The company has established collaborations with notable institutions, including Gustave Roussy Cancer Center and Washington University School of Medicine, to further explore the applications of MMTs in immuno-oncology and other health conditions.
Innovent Biologics, Inc. is a prominent biopharmaceutical company based in Suzhou, China, specializing in the discovery, development, manufacture, and commercialization of monoclonal antibody drug candidates. The company focuses on various therapeutic areas, including oncology, ophthalmology, immunology, and metabolic diseases. Its flagship product, Tyvyt (sintilimab), is an anti-PD-1 monoclonal antibody used in treating multiple cancers and is featured on the National Reimbursement Drug List. Innovent has developed several other promising candidates, including biosimilars and bispecific antibodies for conditions such as non-Hodgkin’s lymphoma, autoimmune diseases, and advanced solid tumors. The company has established strategic collaborations with major global pharmaceutical firms, enhancing its research and development capabilities. Founded in 2011, Innovent has a strong management team with extensive experience in biologics, and it continues to expand its portfolio of therapeutic options while maintaining a commitment to innovation in the biopharmaceutical industry.
Aptinyx Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative therapies for brain and nervous system disorders. Founded in 2015 and based in Evanston, Illinois, the company focuses on novel, synthetic small molecules that modulate the NMDA receptor, enhancing communication pathways between nerve cells. Its primary products in development include NYX-2925, which is undergoing Phase II trials for painful diabetic peripheral neuropathy and fibromyalgia, and NYX-783, currently in Phase I trials for post-traumatic stress disorder. Additionally, Aptinyx is developing NYX-458, another NMDA receptor modulator, which is in Phase I clinical studies aimed at treating cognitive impairment associated with Parkinson’s disease. The company also collaborates with Allergan to develop compounds for major depressive disorder, underscoring its commitment to addressing significant unmet medical needs in the central nervous system arena.
Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company specializes in developing antibody-based therapeutics aimed at treating various diseases linked to dysregulation of the T-helper type 2 immune response, including allergic diseases, inflammation, and conditions characterized by excessive inflammatory cell production. Its lead therapeutic candidate, antolimab (AK002), targets conditions such as eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is advancing another program, AK006, which targets the Siglec-6 receptor on mast cells to modulate their activity and reduce excessive activation. Through these initiatives, Allakos aims to address significant unmet medical needs in immunological health.
resTORbio is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapeutics for the treatment of aging-related diseases.The Company develops medicines and therapies to treat aging-related diseases and conditions such as cancer, heart disease, neurodegenerative diseases, and diabetes mellitus. resTORbio serves clients in the State of Massachusetts.
resTORbio is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapeutics for the treatment of aging-related diseases.The Company develops medicines and therapies to treat aging-related diseases and conditions such as cancer, heart disease, neurodegenerative diseases, and diabetes mellitus. resTORbio serves clients in the State of Massachusetts.
Homology Medicines, Inc. is a genetic medicines company based in Bedford, Massachusetts, focused on improving the lives of patients with rare genetic diseases. The company employs a proprietary platform that utilizes human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies in vivo, either through gene therapy or nuclease-free gene editing. This innovative approach allows for targeted treatment of various disease-relevant tissues, including the liver and central nervous system, through a single injection. Homology's lead product candidate, HMI-102, is currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, while HMI-103 targets pediatric PKU patients, and HMI-202 is being developed for metachromatic leukodystrophy. Founded in 2015, Homology Medicines aims to address significant unmet medical needs by targeting the underlying causes of genetic disorders.
VYNE Therapeutics is a late-stage biopharmaceutical company that specializes in the development and commercialization of serlopitant, a treatment for pruritus associated with various dermatologic conditions, including atopic dermatitis, psoriasis, and prurigo nodularis. The company aims to address a significant unmet medical need, as there are currently no approved therapies in the United States that specifically target pruritus in these conditions. In addition to its focus on dermatologic applications, VYNE is also investigating serlopitant for refractory chronic cough, a persistent cough that lasts more than eight weeks despite treatment. Serlopitant acts as a selective small molecule inhibitor of the neurokinin 1 receptor and is administered as an oral tablet once daily. The company has completed Phase II clinical trials for pruritus related to various conditions and is planning to advance into Phase III trials for prurigo nodularis, anticipating further data to support its clinical development. VYNE Therapeutics is headquartered in Redwood City, California, and was established in 2011.
Zai Lab Limited is a Shanghai-based biopharmaceutical company founded in 2013, dedicated to discovering, developing, and commercializing innovative therapeutics for oncology, autoimmune, and infectious diseases. The company offers several proprietary products, including Niraparib for treating solid tumors, Optune for glioblastoma multiforme, and Ripretinib for KIT and PDGFRa-driven cancers. Additionally, Zai Lab develops other investigational therapies such as REGN1979, Margetuximab, INCMGA0012, Bemarituzumab, Omadacycline, and Durlobactam. With a focus on the Chinese market, Zai Lab has established partnerships with leading global biopharmaceutical firms to enhance its drug pipeline and improve access to transformative medicines. The company leverages its expertise in drug development and favorable regulatory conditions in China, along with in-house manufacturing capabilities, to support both clinical and commercial production. Zai Lab aims to expand its operations and build a specialized commercial team to effectively market its products in China and beyond.
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.
Spero Therapeutics, a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializes in developing novel therapies for multi-drug resistant (MDR) bacterial infections and rare diseases. Its key product candidates include tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic intended for adults with MDR Gram-negative infections, and SPR206, an intravenous agent targeting MDR Gram-negative infections in hospital settings. Additionally, the company is advancing SPR720, an oral antibiotic designed for treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics has established various collaborations and license agreements to support the development and commercialization of its products, including partnerships with Meiji Seika Pharma, Everest Medicines, and the Bill & Melinda Gates Medical Research Institute. Founded in 2013, Spero aims to address the critical need for effective treatments against serious bacterial infections using innovative approaches and mechanisms.
Biohaven is a clinical-stage biopharmaceutical company. It has a portfolio of late-stage product candidates targeting neurological diseases, including rare disorders. The company product candidates are based on multiple mechanisms-calcitonin gene-related peptide receptor antagonists, glutamate modulators and myeloperoxidase inhibitor. Its pipeline products include BHV3000-301, BHV3000-302, BHV3000-303, and others.
Ardelyx, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for cardiorenal diseases. Headquartered in Fremont, California, Ardelyx's lead product candidate, tenapanor, has successfully completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and is also in Phase 3 trials for managing hyperphosphatemia in end-stage renal disease patients undergoing dialysis. The company is advancing RDX013, a small molecule potassium secretagogue, aimed at treating hyperkalemia. Ardelyx is also developing RDX5791, an oral NHE3 sodium transport inhibitor targeting constipation-predominant irritable bowel syndrome and excess dietary sodium absorption, currently in Phase 2 trials. Additionally, RDX002 and RDX009, which inhibit phosphate absorption and target type 2 diabetes, respectively, are in preclinical development. Ardelyx emphasizes non-systemic oral therapeutics that minimize side effects while effectively addressing metabolic disorders. Since its founding in 2007, the company has secured significant funding to support its research and development efforts.
Vedanta Biosciences is a developer of immunotherapies designed to treat immune-mediated and infectious diseases. The company's immunotherapies include a suite of proprietary assays to select pharmacologically potent strains, vast proprietary datasets from human interventional studies, and facilities for cGMP-compliant manufacturing of rationally-defined bacterial consortia in powder form, enabling physicians to access live bacteria drug to treat autoimmune and inflammatory diseases easily.
It was founded in 2010 and is headquartered in Cambridge, Massachusetts.
G1 Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of small molecule therapeutics for cancer treatment. Founded in 2008, the company is headquartered in Research Triangle Park, North Carolina. G1 Therapeutics is advancing several investigational therapies, including trilaciclib, an intravenous cyclin-dependent kinase (CDK) 4/6 inhibitor, currently undergoing Phase 1b/2 trials for extensive-stage small cell lung cancer and Phase 2 trials for first-line small cell lung cancer and metastatic triple-negative breast cancer. The company is also developing lerociclib, an oral CDK4/6 inhibitor, which is in Phase 1b/2a trials for breast cancer and Phase 1b trials for non-small cell lung cancer. Additionally, G1 Therapeutics is progressing rintodestrant, an oral selective estrogen receptor degrader, currently in Phase I/2 trials. The company has partnered with Quantum Leap Healthcare Collaborative to evaluate trilaciclib for neoadjuvant treatment in locally advanced breast cancer. G1 Therapeutics aims to improve treatment outcomes for cancer patients through its innovative therapeutic approaches.
Clearside Biomedical, Inc. is a biopharmaceutical company focused on developing and commercializing innovative treatments for serious eye diseases. Founded in 2011 and based in Alpharetta, Georgia, the company utilizes a proprietary ocular microinjection platform to deliver therapies directly to specific compartments of the eye, including the retina and choroid. Its lead product, XIPERE, is a suprachoroidal injectable suspension of triamcinolone acetonide, targeting macular edema associated with conditions such as uveitis and diabetic macular edema. Additionally, Clearside is advancing CLS-AX, an axitinib formulation for suprachoroidal injection. The company's novel SCS Microinjector allows for a non-surgical, repeatable procedure aimed at preserving and restoring vision in patients with sight-threatening eye diseases.
ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions such as endometriosis, heavy menstrual bleeding, and preterm labor. Its key products include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and uterine fibroids, and OBE022, a selective prostaglandin F2α receptor antagonist designed for once-daily treatment of preterm labor. Additionally, ObsEva is advancing Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily in Switzerland and is committed to improving outcomes for women facing reproductive health challenges.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
Corvus Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Burlingame, California, that specializes in developing innovative immuno-oncology therapies. The company's lead product candidate, Ciforadenant (CPI-444), is an oral small molecule antagonist of the A2A receptor and is currently undergoing Phase Ib/2 clinical trials targeting adenosine, an important immune checkpoint. Additionally, Corvus is advancing CPI-006, an anti-CD73 monoclonal antibody, which is in Phase I/Ib trials to inhibit adenosine production and stimulate immune responses, and is also being evaluated for its potential use in COVID-19. Other candidates in development include an antagonist of the A2B receptor and CPI-818, a small molecule inhibitor targeting interleukin-2-inducible T-cell kinase, which is also in Phase I/Ib trials. The company has established a strategic collaboration with Angel Pharmaceuticals to further develop its pipeline of targeted investigational medicines.
Aclaris Therapeutics is a clinical-stage biopharmaceutical company based in the United States, specializing in the development of innovative dermatologic and immunologic therapies. Founded by the team behind Vicept Therapeutics, Aclaris is dedicated to addressing significant unmet medical needs in dermatology and immunology, particularly in areas lacking FDA-approved treatments. The company operates in two main segments: therapeutics and contract research. The therapeutics segment focuses on creating novel drug candidates for immuno-inflammatory diseases, while the contract research segment generates revenue by providing laboratory services. Aclaris's pipeline includes drug candidates such as Zunsemetinib, an oral MK2 inhibitor, aiming to fill critical treatment gaps in the healthcare landscape.
Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.
Translate Bio is a clinical-stage biotechnology company based in Lexington, Massachusetts, that focuses on developing messenger RNA (mRNA) therapeutics aimed at treating diseases linked to protein or gene dysfunction. Since its founding in 2011, the company has been working on innovative treatments, including MRT5005, which is currently undergoing Phase I/II clinical trials for cystic fibrosis, and MRT5201, intended for the treatment of ornithine transcarbamylase deficiency. Translate Bio aims to create transformative medicines that address unmet medical needs in rare diseases.
Ra Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics for diseases linked to the complement system's excessive activation. Utilizing a proprietary peptide chemistry platform, the company creates synthetic macrocyclic peptides that possess the specificity of antibodies and the advantageous properties of small molecules. Its primary product candidate, Zilucoplan, is an injectable treatment that has completed Phase II clinical trials for generalized myasthenia gravis and paroxysmal nocturnal hemoglobinuria, as well as a Phase Ib trial for patients with renal impairment. Ra Pharmaceuticals is also exploring pre-clinical programs targeting various complement factors for conditions such as C3 glomerulonephritis and autoimmune diseases. Additionally, the company has a collaboration with Merck & Co. to identify orally available cyclic peptides for cardiovascular targets. Founded in 2008, Ra Pharmaceuticals operates as a subsidiary of UCB SA.
Clementia Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Montreal, Canada, focused on developing innovative treatments for patients with ultra-rare bone disorders and other diseases characterized by high unmet medical needs. Founded in 2010, the company specializes in disease-modifying therapies, with its lead product candidate being palovarotene, an oral small molecule. Palovarotene has demonstrated significant potential in preventing abnormal bone formation and fibrosis across various tissues. The candidate is currently undergoing evaluation in the Phase III MOVE trial, targeting conditions such as fibrodysplasia ossificans progressive and multiple osteochondromas, as well as dry eye disease. Clementia's commitment to addressing debilitating health issues highlights its role in advancing treatment options for affected patients.
Ardelyx, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for cardiorenal diseases. Headquartered in Fremont, California, Ardelyx's lead product candidate, tenapanor, has successfully completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and is also in Phase 3 trials for managing hyperphosphatemia in end-stage renal disease patients undergoing dialysis. The company is advancing RDX013, a small molecule potassium secretagogue, aimed at treating hyperkalemia. Ardelyx is also developing RDX5791, an oral NHE3 sodium transport inhibitor targeting constipation-predominant irritable bowel syndrome and excess dietary sodium absorption, currently in Phase 2 trials. Additionally, RDX002 and RDX009, which inhibit phosphate absorption and target type 2 diabetes, respectively, are in preclinical development. Ardelyx emphasizes non-systemic oral therapeutics that minimize side effects while effectively addressing metabolic disorders. Since its founding in 2007, the company has secured significant funding to support its research and development efforts.
BeiGene, Ltd. is a biotechnology company engaged in the development and commercialization of targeted and immuno-oncology therapies for cancer. Founded in 2010 and based in Beijing, the company operates globally, focusing on addressing significant unmet medical needs across various cancer types. Its commercial portfolio includes treatments such as BRUKINSA for mantle cell lymphoma and Tislelizumab for Hodgkin's lymphoma, alongside other therapies for conditions like breast cancer and multiple myeloma. BeiGene's pipeline features several clinical-stage drug candidates, including Zanubrutinib, an inhibitor targeting Bruton's tyrosine kinase, and Pamiparib, a PARP inhibitor. The company is dedicated to making innovative cancer treatments more affordable and accessible, and it collaborates with various pharmaceutical firms to enhance its research and development efforts.
Adaptive Biotechnologies is a commercial-stage company focused on advancing immune-driven medicine by leveraging the biology of the adaptive immune system for disease diagnosis and treatment. Founded in 2009 and headquartered in Seattle, Washington, the company offers a range of products and services, including the immunoSEQ research service and kit, which aids in research and the discovery of diagnostic signals. Its FDA-authorized clonoSEQ diagnostic test is designed for the detection and monitoring of minimal residual disease in patients with specific blood cancers. Additionally, Adaptive Biotechnologies is developing a pipeline of clinical products aimed at diagnosing and treating cancer, autoimmune conditions, and infectious diseases. The company has formed strategic collaborations with notable partners, including Genentech for neoantigen-directed T cell therapies, Microsoft for early disease detection tests from blood samples, and Amgen for therapeutic development related to COVID-19.
Mirna Therapeutics, Inc. is a discovery-stage biopharmaceutical company that specializes in developing miRNA-directed therapies for human oncology. With a strong focus on research and a deep understanding of microRNA and cancer biology, Mirna is positioned to advance the emerging field of miRNA-based therapeutics. The company boasts a robust intellectual property portfolio that supports its innovative approach to cancer treatment.
Merus N.V. is a clinical-stage immuno-oncology company based in the Netherlands, focused on the discovery and development of bispecific antibody therapeutics. The company's pipeline includes several promising candidates, notably MCLA-128, currently in phase 2 clinical trials for metastatic breast cancer; MCLA-117, in phase I trials for acute myeloid leukemia; and MCLA-158, also in phase I trials for solid tumors. Additionally, Merus is advancing other bispecific antibody candidates, including MCLA-129 and MCLA-145, in collaboration with various pharmaceutical partners. The company employs a unique technology called Oligoclonics, which enables the production of a mixture of therapeutic antibodies from a single cell clone, aimed at targeting common antigens. Merus has established partnerships to enhance its research capabilities, such as its collaboration with Caris Life Sciences for detecting NRG1 fusions in cancer patients. Founded in 2003, Merus is headquartered in Utrecht and is dedicated to developing innovative treatments for cancer.
Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.
Aeglea BioTherapeutics is a clinical-stage biotechnology company focused on developing human enzyme therapeutics for rare genetic and cancer diseases. Its lead product candidate, pegzilarginase, is a recombinant human Arginase 1 enzyme currently undergoing Phase III trials to assess its safety and efficacy in treating Arginase 1 deficiency. The company's preclinical pipeline includes several innovative candidates: ACN00177 for homocystinuria, AEB5100 which degrades plasma cystine and cysteine, AEB2109 targeting the degradation of methionine, and AEB3103. Aeglea BioTherapeutics aims to exploit the amino acid dependencies of tumors by creating novel, human-derived enzymes that selectively target these vulnerabilities, differentiating the metabolic needs of tumor cells from those of normal tissues. Founded in 2013 and headquartered in Austin, Texas, the company was formerly known as Aeglea BioTherapeutics Holdings, LLC before changing its name in 2015.
Collegium Pharmaceutical is a specialty pharmaceutical company that develops and commercializes products aimed at treating chronic pain while addressing the concerns of prescription drug abuse. The company utilizes its patented DETERx platform technology to create abuse-deterrent formulations that maintain the extended-release profiles and safety of medications despite potential misuse methods such as chewing or crushing. Collegium's product lineup includes Xtampza ER, an extended-release oral formulation of oxycodone designed to deter abuse, as well as Nucynta ER and Nucynta IR, which are extended-release and immediate-release formulations of tapentadol respectively, for managing severe pain and acute pain in adults. Founded in 2002 and headquartered in Stoughton, Massachusetts, Collegium focuses on delivering innovative solutions for patients with chronic pain, utilizing formulation improvements backed by strong intellectual property protections.
Chiasma (Israel) Ltd. is a biopharmaceutical company specializing in the development of oral drugs utilizing its proprietary Transient Permeability Enhancer technology. Founded in 2001 and based in Ness Ziona, Israel, the company focuses on transforming injectable drugs into oral formulations, thereby enhancing their absorption and potentially enabling new therapeutic indications. Its primary product is oral octreotide acetate, designed for the treatment of acromegaly, a rare condition. Chiasma's pipeline includes novel drugs aimed at addressing unmet medical needs in well-defined markets, with a particular emphasis on orphan indications. The company operates as a subsidiary of Chiasma, Inc., and is dedicated to improving the treatment options for patients with rare and debilitating diseases.
Cidara Therapeutics is a biotechnology company based in San Diego, California, dedicated to the discovery, development, and commercialization of innovative anti-infective therapies. The company's lead product candidate, rezafungin acetate, is an antifungal agent from the echinocandin class designed to treat and prevent serious invasive fungal infections, such as candidemia and invasive candidiasis, which carry high mortality rates. In addition to its antifungal efforts, Cidara is advancing its Cloudbreak platform, which focuses on developing antiviral Fc-conjugates aimed at preventing and treating viral infections, including influenza, RSV, HIV, and coronaviruses. Founded in 2012, Cidara Therapeutics has positioned itself as a key player in addressing life-threatening illnesses, particularly those that pose challenges due to immune system deficiencies.
Sagimet Biosciences is a biotechnology company that focuses on developing novel therapeutics to treat important diseases such as liver disease NASH and specific cancers, with a focus on targeting dysfunctional metabolic pathways. It was founded in 2007 and headquartered in San Mateo, California.
Nivalis Therapeutics is focused on developing innovative therapies aimed at treating cystic fibrosis (CF) by preserving intracellular S-nitrosoglutathione (GSNO), a molecule involved in cell signaling and implicated in the disease's pathophysiology. The company's lead candidate, N91115, specifically targets patients with the F508del mutation, the most prevalent mutation associated with CF. Founded in 2007 and headquartered in Boulder, Colorado, Nivalis Therapeutics is committed to improving the lives of individuals affected by cystic fibrosis and supporting their families through its research and development efforts.
Invitae is a genetic information company focused on integrating genetic testing into routine medical practice to enhance healthcare quality worldwide. It specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates a wide array of genetic tests into one platform, ensuring faster turnaround times and lower costs compared to traditional single-gene tests. Invitae's test offerings cover various hereditary conditions, including cancers, neurological disorders, cardiovascular diseases, pediatric and metabolic disorders. The company aims to make genetic testing more affordable and accessible, develop a secure infrastructure for genome management, and foster a global community for sharing genetic information to advance scientific research and medical practices. By lowering the barriers to obtaining diagnostic genetic information, Invitae seeks to improve healthcare outcomes for millions of individuals.
Adaptimmune Therapeutics plc is a clinical-stage biopharmaceutical company based in Abingdon, United Kingdom, specializing in innovative cell therapies for cancer treatment, particularly targeting solid tumors. The company utilizes its specific peptide enhanced affinity receptor (SPEAR) T-cell platform to identify and genetically engineer T-cell receptors that enhance the immune response against cancerous cells. Current clinical trials are exploring various therapeutic candidates, including ADP-A2M4 for multiple solid tumor types and ADP-A2AFP for hepatocellular carcinoma. Adaptimmune has formed strategic collaborations with several organizations, including GSK for the development of NY-ESO T-cell therapy and Noile-Immune Biotech Inc. for combining SPEAR T-cells with PRIME technology. Founded in 2008, Adaptimmune is committed to advancing T-cell therapies to improve patient outcomes in oncology and beyond.
Civitas Therapeutics is a biopharmaceutical company dedicated to developing therapies for pulmonary delivery. The company focuses on creating innovative treatments for central nervous system and respiratory disorders, with a particular emphasis on conditions such as multiple sclerosis and spinal cord injuries. Civitas Therapeutics aims to improve the health and quality of life for patients across the United States through its specialized drug delivery systems.
Spark Therapeutics, Inc. is dedicated to developing gene therapy products aimed at treating debilitating genetic diseases. The company is known for LUXTURNA, a treatment for patients with biallelic RPE65 mutation-associated retinal dystrophy, and has an extensive pipeline that includes candidates like SPK-8011 and SPK-8016 for hemophilia, SPK-7001 for choroideremia, and SPK-9001 for hemophilia B. Additionally, Spark is exploring liver-directed gene therapies such as SPK-3006 for Pompe disease and has programs targeting neurodegenerative diseases, including Huntington's disease and a form of Batten disease. Spark Therapeutics also engages in collaborations, notably with Pfizer for the SPK-FIX program for hemophilia B, and has a licensing agreement with Novartis for the commercialization of LUXTURNA outside the United States. Established in 2013 and based in Philadelphia, Spark Therapeutics operates as a subsidiary of Roche Holding AG and leverages a proprietary manufacturing platform to support its clinical studies across various therapeutic areas.
Coherus BioSciences, Inc. is a commercial-stage biopharmaceutical company based in Redwood City, California, that specializes in the development, manufacture, and marketing of biosimilar therapeutics. It primarily targets the oncology and inflammatory disease markets with its product offerings. The company's flagship product, UDENYCA, is a biosimilar to Neulasta, designed to stimulate the production of granulocytes to enhance the body’s infection-fighting capabilities. Coherus is also advancing several other biosimilar candidates, including those for Humira and Enbrel, which have completed Phase III clinical studies. Additionally, the company is working on biosimilars for other biologics, including ranibizumab and aflibercept, as well as a small-molecule drug for metabolic conditions. Coherus BioSciences was founded in 2010 and has established various license agreements to support its product development initiatives.
Adverum Biotechnologies is a clinical-stage gene therapy company based in Redwood City, California, focused on developing innovative therapies for ocular and rare diseases. Its primary product candidate, ADVM-022, is designed for the treatment of wet age-related macular degeneration through a single intravitreal injection that aims to provide long-lasting therapeutic effects. The company's pipeline also includes ADVM-043, targeting alpha-1 antitrypsin deficiency, and ADVM-053, which is in preclinical development for hereditary angioedema. Adverum collaborates with Editas Medicine to utilize its proprietary AAV vectors for genome editing in inherited retinal diseases and has a partnership with Regeneron Pharmaceuticals to explore multiple ocular therapeutic targets. Originally known as Avalanche Biotechnologies, the company rebranded in May 2016 following a merger with Annapurna Therapeutics, further advancing its mission to deliver effective treatments for chronic and debilitating conditions.