F Prime, Inc. is a venture capital firm based in Cambridge, Massachusetts, with additional offices in San Francisco and London. Founded in 1969, it has a strong legacy rooted in Fidelity Investments, which has been a significant player in asset management since 1946. F Prime focuses on early-stage investments in North America and Europe, primarily in the healthcare, life sciences, technology, therapeutics, fashion, medtech, and health information technology sectors. The firm operates several funds, including those dedicated to healthcare, oncology, life sciences, and health technology services. F Prime Capital Partners emphasizes supporting entrepreneurs without the pressure of external fundraising, allowing them to concentrate on identifying and nurturing promising companies.
Comanche is a biopharmaceutical company developing an investigational siRNA medicine for preterm preeclampsia. We envision a world where all women and their babies have access to safe and effective therapies for treating life threatening complications of pregnancy, and those solutions must be evidence-based and affordable.
Immuneel Therapeutics provides pioneering cell and gene therapies for cancer treatment. They develop CAR-T cell therapies from patients' blood cells by genetically modifying healthy immune cells to target malignant cells, giving cancer patients access to revolutionary cell-based immunotherapies.
Proof Diagnostics focuses on developing CRISPR-based molecular tests for the detection of COVID-19. The company aims to empower communities by providing rapid diagnostic kits that enable medical professionals to effectively test patients for coronavirus infections. By leveraging advanced genetic technologies, Proof Diagnostics contributes to improved health outcomes and better management of infectious diseases.
AviadoBio mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute.
Neumora Therapeutics operates as a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience.
Embark offers dog DNA testing that ends preventable diseases in dogs. It was founded by top experts in genomics, computational biology, and software design to sell an industry-leading dog DNA test. They use a clinical-grade CLIA-certified SNP chip to provide the most accurate and comprehensive results (200,000+ genetic markers) available on the market. Their data contributes to proprietary genetics research that will help dog owners optimize nutrition, exercise, allergies, and prevent a variety of heritable conditions. Their technological advantage, intellectual capital, and research-focus enable them to push canine health toward our shared vision of preventative veterinary care.
Castor is an international health-tech company that offers a cloud-based clinical data platform designed to streamline the clinical trial process for researchers around the world. Founded by CEO Derk Arts, MD, Ph.D., the platform is utilized by over 50,000 researchers in 90 countries and has facilitated more than 4,000 studies across diverse therapeutic areas such as diabetes, cardiovascular diseases, rare diseases, infectious diseases, and oncology. Researchers using Castor generate substantial amounts of data from both traditional and remote trials, having reached significant milestones of 180 million data points and 2 million enrolled patients. The company's mission is to enhance the reusability of research data, supporting AI-driven clinical trials and ultimately maximizing the impact of data to improve medical research globally.
Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
Pediatrix Therapeutics is a pharmaceutical company focused on the field of pediatrics by developing and introducing effective and clinically proven children's medicines to provide Chinese children and families with high-quality and affordable treatment options.
Yisheng Biopharma Co., Ltd. is a biopharmaceutical company focused on the research, development, manufacturing, and marketing of immuno-oncology products and vaccines. Founded in 2002 and headquartered in Beijing, the company operates in China, the United States, Cambodia, and Singapore. Yisheng Biopharma utilizes its novel PIKA immunomodulating technology to enhance immune responses against cancers and infectious diseases. Key products under development include YS-ON-001, an investigational therapy aimed at treating advanced solid tumors, as well as YS-HBV-001, a vaccine for hepatitis B, and a PIKA-based rabies vaccine designed for rapid protection against rabies infection. The company's innovative approach targets multiple immune pathways to improve treatment outcomes.
Cohort, Inc. specializes in developing software that enhances chronic care management and clinical trials. The company's clinical AI and natural language processing technology analyzes electronic health records (EHR), extracting insights from diagnostics, medications, and clinical notes to provide tailored context for patients and their conditions. Cohort's platform streamlines the identification and enrollment of eligible patients, generates care plans automatically, and facilitates compliance and billing processes. By offering these solutions, Cohort enables primary care providers to improve the scale and efficiency of their chronic care management programs while also enhancing the identification and enrollment process for clinical trials. The company is headquartered in New York, with an additional office in San Francisco.
Atalanta Therapeutics is a biotechnology company focused on developing innovative treatment options for neurodegenerative diseases through its proprietary RNA interference platform. The company aims to create therapeutics that target various conditions, including Huntington's disease by specifically targeting the HTT gene. By addressing the underlying causes of these diseases, Atalanta Therapeutics seeks to provide clinicians with effective strategies to halt disease progression and improve patient outcomes.
J-Pharma Co., Ltd., founded in 2005 and based in Yokohama, Japan, specializes in the development of therapeutics and diagnostic agents for cancer treatment. The company focuses on a human-genomic approach, creating novel pharmaceuticals that target specific diseases with selective agents. J-Pharma's research emphasizes the role of cell membrane transporters, which are linked to various diseases, particularly in cancer. The company has identified significant drug transporters within the human body that can enhance the evaluation and optimization of pharmacokinetics for new drug candidates. J-Pharma's product pipeline includes anti-cancer drugs and PET diagnostic agents targeting LAT1, a transporter protein associated with cancer cell growth. Through its innovative drug development and diagnostic methods, J-Pharma aims to improve human health and welfare, particularly for the aging population in industrialized nations.
Citrine Medicine
Series A in 2020
Citrine Medicine is a rare disease-focused pharmaceutical firm.
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.
Checkmate Pharmaceuticals is a clinical-stage biotechnology company based in Cambridge, Massachusetts, that specializes in developing novel immunotherapies for cancer treatment. Founded in 2015, the company focuses on leveraging CpG oligonucleotides to enhance anti-tumor T-cell responses and overcome the mechanisms that allow tumors to evade immune detection. By combining its proprietary technology with checkpoint inhibition, Checkmate aims to improve the efficacy of existing immunotherapies and provide new treatment options for patients. The company has formed strategic alliances with major pharmaceutical firms, including Merck KGaA and Pfizer, to further its research and development efforts in the field of cancer immunotherapy.
NFlection Therapeutics, Inc. is a biotechnology company dedicated to developing targeted therapies for rare disorders, particularly those associated with neurofibromatosis type 1 and related conditions. The company specializes in creating novel therapies aimed at addressing the needs of patients suffering from immunosuppressant-mediated squamous cell carcinoma and congenital birthmarks, including keratinocytic epidermal nevi and nevus sebaceous. NFlection focuses on first-in-class MEK inhibitors that target the aberrant activation of the Ras/Raf/MEK/ERK pathway, which is central to these disorders. Founded in 2014 and headquartered in Wayne, Pennsylvania, NFlection Therapeutics is committed to advancing treatments that can significantly impact the lives of patients with these challenging conditions.
NodThera Limited is a biotechnology company focused on researching and developing novel inhibitors of the NLRP3 inflammasome, which play a critical role in inflammatory and neuroinflammatory diseases. Established in 2016 and headquartered in Little Chesterford, United Kingdom, NodThera operates a laboratory in Seattle, Washington, and a corporate office in Lexington, Massachusetts. The company aims to create therapies targeting a range of conditions including arthritis, atherosclerosis, Alzheimer’s disease, diabetes, and certain cancers. By developing potent and selective NLRP3 inflammasome inhibitors, NodThera seeks to reduce pro-inflammatory cytokines, enabling more effective treatment options for patients suffering from chronic inflammation-related diseases.
Arvelle Therapeutics GmbH is a biopharmaceutical company based in Zug, Switzerland, founded in 2019. The company focuses on developing and commercializing innovative treatments for patients with central nervous system (CNS) disorders. Its primary product is cenobamate, an investigational antiepileptic drug aimed at providing effective solutions for patients suffering from focal seizures. By targeting debilitating neurological and neuromuscular diseases, Arvelle Therapeutics aims to enhance the quality of care available to clinicians and their patients.
Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing therapies for idiopathic pulmonary fibrosis (IPF) and other severe respiratory diseases. The company is advancing inhaled formulations of pirfenidone, specifically AP01, which aims to enhance drug delivery to the lungs while potentially reducing side effects associated with oral treatments. This innovative approach seeks to improve the care and outcomes for patients suffering from these conditions. Established in 2011, Avalyn Pharma operates from its headquarters in Seattle, Washington, and maintains an additional office in San Diego, California. The company was previously known as Genoa Pharmaceuticals, Inc. before rebranding in July 2017.
Rallybio, LLC is a biopharmaceutical company based in New Haven, Connecticut, that focuses on developing innovative therapies for patients with severe and rare disorders. Founded in 2018, the company is dedicated to addressing life-threatening conditions, particularly through its lead product candidate, RLYB211, which aims to prevent fetal and neonatal alloimmune thrombocytopenia (FNAIT). This rare disease can lead to uncontrolled bleeding in fetuses and newborns. Rallybio's research emphasizes the development of antibody, small molecule, and engineered protein therapies, primarily in the late discovery to early clinical stages. Through its efforts, Rallybio seeks to transform the lives of patients suffering from devastating diseases.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
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.
Adagene (Suzhou) Limited is a clinical-stage biotechnology company based in Suzhou, China, focused on antibody discovery and engineering. The company specializes in developing immuno-oncology antibodies that target novel epitopes, utilizing its proprietary Smart Antibody Technology. This technology enhances the success rates of antibody development, significantly shortens the time required to bring products to market, and lowers associated costs. Adagene's offerings extend beyond therapeutic applications, as its technology is also applicable in diagnostics and research, aiming to advance the field of immunotherapy and improve patient outcomes. Founded in 2011, Adagene continues to innovate in the biopharmaceutical sector.
Immuneel Therapeutics provides pioneering cell and gene therapies for cancer treatment. They develop CAR-T cell therapies from patients' blood cells by genetically modifying healthy immune cells to target malignant cells, giving cancer patients access to revolutionary cell-based immunotherapies.
AviadoBio mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute.
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.
Nocion Therapeutics, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, established in 2017. The company focuses on developing novel therapeutics called "nocions," which are designed to treat conditions associated with neurogenic inflammation. These nocions specifically target activated sensory neurons, entering nociceptors through large-pore channels that open in response to stimulation. This mechanism allows for the selective blocking of sodium channels, providing targeted and sustained relief for serious conditions such as cough, itch, pain, and inflammation. By offering an alternative to traditional small molecule anesthetics, Nocion Therapeutics aims to deliver more effective treatment options for patients suffering from these debilitating symptoms.
Embark offers dog DNA testing that ends preventable diseases in dogs. It was founded by top experts in genomics, computational biology, and software design to sell an industry-leading dog DNA test. They use a clinical-grade CLIA-certified SNP chip to provide the most accurate and comprehensive results (200,000+ genetic markers) available on the market. Their data contributes to proprietary genetics research that will help dog owners optimize nutrition, exercise, allergies, and prevent a variety of heritable conditions. Their technological advantage, intellectual capital, and research-focus enable them to push canine health toward our shared vision of preventative veterinary care.
Caplin Point Laboratories Limited is an India-based pharmaceutical company established in 1990, specializing in the development, manufacture, and marketing of generic and branded pharmaceutical products. The company offers a diverse range of products, including tablets, capsules, liquid and lyophilized injections, ophthalmic solutions, ointments, creams, gels, and various specialized formulations. Caplin Point has a significant presence in emerging markets, particularly in Latin America, the Caribbean, and Francophone Africa, holding over 2,000 product licenses worldwide. The company is expanding into regulated markets for injectables with a state-of-the-art manufacturing facility that is compliant with international standards, including approvals from the US FDA and EU-GMP. Additionally, Caplin Point operates QueTenX, an e-commerce platform. The company is headquartered in Chennai, India.
Checkmate Pharmaceuticals is a clinical-stage biotechnology company based in Cambridge, Massachusetts, that specializes in developing novel immunotherapies for cancer treatment. Founded in 2015, the company focuses on leveraging CpG oligonucleotides to enhance anti-tumor T-cell responses and overcome the mechanisms that allow tumors to evade immune detection. By combining its proprietary technology with checkpoint inhibition, Checkmate aims to improve the efficacy of existing immunotherapies and provide new treatment options for patients. The company has formed strategic alliances with major pharmaceutical firms, including Merck KGaA and Pfizer, to further its research and development efforts in the field of cancer immunotherapy.
Avidity Biosciences is a biopharmaceutical company specializing in the development of oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disorder. The company's development pipeline also includes programs focused on treating various muscle diseases, such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Additionally, Avidity is exploring therapeutic applications for immune and other cell types, leveraging its proprietary AOC platform to reach previously undruggable tissues and effectively target the genetic underpinnings of these conditions. Founded in 2012, Avidity Biosciences is headquartered in La Jolla, California.
Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with significant unmet medical needs. The company, based in Oakland, California, was incorporated in 2016 and is a subsidiary of Zogenix, Inc. Modis Therapeutics is advancing its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapeutic candidate is specifically designed to address thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance, increasing mitochondrial DNA copy number, and enhancing cell function, Modis Therapeutics aims to provide meaningful treatment options that can prolong the lives of affected patients.
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.
TraceLink, Inc. specializes in providing track and trace network solutions that enhance the life sciences supply chain and combat counterfeit prescription drugs. The company offers a range of products, including pharmaceutical serialization, drug tracking, and compliance solutions through its TraceLink Life Sciences Cloud. Key offerings include the Smart Rx Manager, which helps healthcare providers comply with the European Union Falsified Medicines Directive, and Serialized Product Intelligence, a cloud application that utilizes serialization data to identify supply chain issues and optimize operations. TraceLink serves a diverse clientele, including pharmaceutical companies, wholesale distributors, and re-packagers, and has established strategic partnerships with notable firms such as Amazon and KPMG. Founded in 2009 and headquartered in North Reading, Massachusetts, TraceLink also has offices in the United Kingdom, India, Singapore, and Brazil. The company's platform is recognized for its ability to deliver global connectivity and visibility across the pharmaceutical supply chain, ensuring compliance and improving operational efficiency.
Compass Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing and discovering therapeutic antibodies for the treatment of solid tumors and hematological malignancies. The company's lead product candidate, CTX-471, is a fully human monoclonal antibody that acts as an agonist of CD137, currently undergoing Phase I clinical trials for patients who have had inadequate responses to existing PD-1/PD-L1 checkpoint inhibitors. In addition to CTX-471, Compass is advancing several preclinical assets, including CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, and CTX-8573, designed to engage natural killer (NK) cells against B cell maturation antigen, which is prevalent in multiple myeloma. Founded in 2014, Compass Therapeutics is headquartered in Cambridge, Massachusetts, and employs innovative platforms to enhance the efficacy of its antibody therapeutics.
NodThera Limited is a biotechnology company focused on researching and developing novel inhibitors of the NLRP3 inflammasome, which play a critical role in inflammatory and neuroinflammatory diseases. Established in 2016 and headquartered in Little Chesterford, United Kingdom, NodThera operates a laboratory in Seattle, Washington, and a corporate office in Lexington, Massachusetts. The company aims to create therapies targeting a range of conditions including arthritis, atherosclerosis, Alzheimer’s disease, diabetes, and certain cancers. By developing potent and selective NLRP3 inflammasome inhibitors, NodThera seeks to reduce pro-inflammatory cytokines, enabling more effective treatment options for patients suffering from chronic inflammation-related diseases.
TraceLink, Inc. specializes in providing track and trace network solutions that enhance the life sciences supply chain and combat counterfeit prescription drugs. The company offers a range of products, including pharmaceutical serialization, drug tracking, and compliance solutions through its TraceLink Life Sciences Cloud. Key offerings include the Smart Rx Manager, which helps healthcare providers comply with the European Union Falsified Medicines Directive, and Serialized Product Intelligence, a cloud application that utilizes serialization data to identify supply chain issues and optimize operations. TraceLink serves a diverse clientele, including pharmaceutical companies, wholesale distributors, and re-packagers, and has established strategic partnerships with notable firms such as Amazon and KPMG. Founded in 2009 and headquartered in North Reading, Massachusetts, TraceLink also has offices in the United Kingdom, India, Singapore, and Brazil. The company's platform is recognized for its ability to deliver global connectivity and visibility across the pharmaceutical supply chain, ensuring compliance and improving operational efficiency.
Tempest Therapeutics is a biotechnology company that focuses on small molecule therapeutics that modulate anti-tumor immunity pathways. The company has a balanced and deep pipeline consisting of first-in-class and best-in-class small molecule therapeutics, which modulate distinct pathways relevant to mounting an effective anti-tumor response.
Tempest Therapeutics was established in 2011 and is headquartered in San Francisco, California.
Hua Medicine is a clinical-stage, innovative drug development company in China, focused on novel therapies for the treatment of diabetes and CNS disorders. Founded by pharma industry veterans and funded by a premier group of international VC investment firms such as Fidelity Asia, Fidelity Biosciences, ARCH Ventures, Venrock, SAIL and WuXi Ventures, the company has in-licensed from major pharma, world-wide rights to a potential best-in-class, oral drug for the treatment of Type 2 Diabetes which utilizes a novel mechanism of action.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who traditionally rely on insulin injections. Founded based on groundbreaking research by Professor Douglas Melton, the company has licensed a method to generate functional, insulin-producing beta cells in the laboratory. Semma Therapeutics aims to combine these proprietary cells with advanced devices to create a comprehensive therapy that can replace the missing beta cells without the need for immunosuppression. By striving to bring this transformative treatment to clinical application, Semma Therapeutics seeks to significantly enhance the lives of diabetes patients. Headquartered in Cambridge, Massachusetts, the company is a subsidiary of Vertex Pharmaceuticals and collaborates strategically with Defymed.
Apellis Pharmaceuticals is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, dedicated to developing therapeutic compounds that inhibit the complement system to address autoimmune and inflammatory diseases. The company's lead candidate, pegcetacoplan, is undergoing Phase III clinical trials for geographic atrophy associated with age-related macular degeneration and paroxysmal nocturnal hemoglobinuria, while also being assessed in Phase II trials for several other conditions, including cold agglutinin disease and lupus nephritis. Additionally, Apellis is developing APL-9, which is currently in Phase I clinical trials aimed at preventing immune activation related to adeno-associated virus. The company collaborates with Swedish Orphan Biovitrum AB to advance pegcetacoplan for various rare diseases. Founded in 2009, Apellis focuses on creating innovative therapies for conditions characterized by excessive activation of the complement cascade across multiple medical fields, including hematology and ophthalmology.
Indalo Therapeutics is a preclinical-stage biotechnology company focused on developing innovative therapies for patients afflicted by fibrotic diseases. Founded on significant scientific advancements, the company leverages deep insights into the molecular mechanisms that drive abnormal scarring. By assembling a skilled team of pharmaceutical scientists and industry executives, Indalo aims to address critical unmet medical needs in the field of fibrosis. Its therapeutic approach seeks to not only halt but also potentially reverse the progression of fibrotic conditions, ultimately improving the quality of life for affected individuals.
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.
Compass Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing and discovering therapeutic antibodies for the treatment of solid tumors and hematological malignancies. The company's lead product candidate, CTX-471, is a fully human monoclonal antibody that acts as an agonist of CD137, currently undergoing Phase I clinical trials for patients who have had inadequate responses to existing PD-1/PD-L1 checkpoint inhibitors. In addition to CTX-471, Compass is advancing several preclinical assets, including CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, and CTX-8573, designed to engage natural killer (NK) cells against B cell maturation antigen, which is prevalent in multiple myeloma. Founded in 2014, Compass Therapeutics is headquartered in Cambridge, Massachusetts, and employs innovative platforms to enhance the efficacy of its antibody therapeutics.
Checkmate Pharmaceuticals is a clinical-stage biotechnology company based in Cambridge, Massachusetts, that specializes in developing novel immunotherapies for cancer treatment. Founded in 2015, the company focuses on leveraging CpG oligonucleotides to enhance anti-tumor T-cell responses and overcome the mechanisms that allow tumors to evade immune detection. By combining its proprietary technology with checkpoint inhibition, Checkmate aims to improve the efficacy of existing immunotherapies and provide new treatment options for patients. The company has formed strategic alliances with major pharmaceutical firms, including Merck KGaA and Pfizer, to further its research and development efforts in the field of cancer immunotherapy.
Genoa Pharmaceuticals, Inc., a biopharmaceutical company, develops therapies for the treatment of idiopathic pulmonary fibrosis (IPF). It develops GP-101, an aerosol pirfenidone formulation for direct inhalation delivery to the lungs for the treatment of IPF. The company was founded in 2011 and is based in San Diego, California.
Promentis Pharmaceuticals, Inc. is a biopharmaceutical company based in Milwaukee, Wisconsin, established in 2006. The company specializes in developing therapies for neuropsychiatric disorders, with a focus on addressing glutamatergic imbalance and oxidative stress. Promentis is known for its lead compound, SXC-2023, which targets System xc- to treat multiple conditions, including impulse control disorders and obsessive-compulsive disorder. In addition to SXC-2023, Promentis is also working on other compounds aimed at restoring glutamatergic neurotransmission, which is crucial for improving cortical function and managing various psychiatric disorders, including schizophrenia.
Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who traditionally rely on insulin injections. Founded based on groundbreaking research by Professor Douglas Melton, the company has licensed a method to generate functional, insulin-producing beta cells in the laboratory. Semma Therapeutics aims to combine these proprietary cells with advanced devices to create a comprehensive therapy that can replace the missing beta cells without the need for immunosuppression. By striving to bring this transformative treatment to clinical application, Semma Therapeutics seeks to significantly enhance the lives of diabetes patients. Headquartered in Cambridge, Massachusetts, the company is a subsidiary of Vertex Pharmaceuticals and collaborates strategically with Defymed.
Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.
Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.
Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing therapies for idiopathic pulmonary fibrosis (IPF) and other severe respiratory diseases. The company is advancing inhaled formulations of pirfenidone, specifically AP01, which aims to enhance drug delivery to the lungs while potentially reducing side effects associated with oral treatments. This innovative approach seeks to improve the care and outcomes for patients suffering from these conditions. Established in 2011, Avalyn Pharma operates from its headquarters in Seattle, Washington, and maintains an additional office in San Diego, California. The company was previously known as Genoa Pharmaceuticals, Inc. before rebranding in July 2017.
Core Diagnostics Private Limited is a clinical laboratory based in Gurugram, India, established in 2012. The company specializes in next-generation diagnostics aimed at disease stratification and therapy selection. By employing advanced testing techniques, Core Diagnostics seeks to provide comprehensive diagnostic solutions and become a leading provider of high-end diagnostic services in the region.
TraceLink, Inc. specializes in providing track and trace network solutions that enhance the life sciences supply chain and combat counterfeit prescription drugs. The company offers a range of products, including pharmaceutical serialization, drug tracking, and compliance solutions through its TraceLink Life Sciences Cloud. Key offerings include the Smart Rx Manager, which helps healthcare providers comply with the European Union Falsified Medicines Directive, and Serialized Product Intelligence, a cloud application that utilizes serialization data to identify supply chain issues and optimize operations. TraceLink serves a diverse clientele, including pharmaceutical companies, wholesale distributors, and re-packagers, and has established strategic partnerships with notable firms such as Amazon and KPMG. Founded in 2009 and headquartered in North Reading, Massachusetts, TraceLink also has offices in the United Kingdom, India, Singapore, and Brazil. The company's platform is recognized for its ability to deliver global connectivity and visibility across the pharmaceutical supply chain, ensuring compliance and improving operational efficiency.
Turnstone Biologics Inc. is a biotechnology company dedicated to developing viral immunotherapies aimed at enhancing cancer survival rates. Founded in 2015 and headquartered in Ottawa, Canada, with an additional office in New York, the company’s lead candidate, RIVAL-01, is based on a vaccinia virus backbone that incorporates three effective immunomodulators: Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine. These components are designed to synergistically stimulate immune responses and modify the tumor microenvironment to facilitate tumor eradication. Turnstone's platform leverages discoveries from prominent researchers in the field of oncolytic viral immunotherapy and has progressed RIVAL-01 into a Phase I/II clinical development study in collaboration with four Canadian academic institutions and the Fight Against Cancer Innovation Trust (FACIT).
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.
Promentis Pharmaceuticals, Inc. is a biopharmaceutical company based in Milwaukee, Wisconsin, established in 2006. The company specializes in developing therapies for neuropsychiatric disorders, with a focus on addressing glutamatergic imbalance and oxidative stress. Promentis is known for its lead compound, SXC-2023, which targets System xc- to treat multiple conditions, including impulse control disorders and obsessive-compulsive disorder. In addition to SXC-2023, Promentis is also working on other compounds aimed at restoring glutamatergic neurotransmission, which is crucial for improving cortical function and managing various psychiatric disorders, including schizophrenia.
Denali Therapeutics is a biopharmaceutical company based in South San Francisco, California, dedicated to discovering and developing therapies for neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS). The company is advancing several therapeutic candidates, including LRRK2 inhibitors like DNL201 and DNL151, which are in various phases of clinical trials for Parkinson's disease. Additionally, Denali is developing DNL747, a selective RIPK1 inhibitor currently in Phase 1b trials for Alzheimer's and ALS. It also has programs focused on enzyme replacement therapy for MPS II and antibody transport vehicles targeting key proteins involved in neurodegeneration. Denali collaborates with various organizations, including Takeda Pharmaceutical Company and Genentech, to enhance its research efforts. Founded in 2013, Denali Therapeutics emphasizes a science-driven approach to address the complex challenges posed by neurodegenerative diseases, leveraging recent scientific insights and translational medicine tools.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
Hua Medicine is a clinical-stage, innovative drug development company in China, focused on novel therapies for the treatment of diabetes and CNS disorders. Founded by pharma industry veterans and funded by a premier group of international VC investment firms such as Fidelity Asia, Fidelity Biosciences, ARCH Ventures, Venrock, SAIL and WuXi Ventures, the company has in-licensed from major pharma, world-wide rights to a potential best-in-class, oral drug for the treatment of Type 2 Diabetes which utilizes a novel mechanism of action.
Adagene (Suzhou) Limited is a clinical-stage biotechnology company based in Suzhou, China, focused on antibody discovery and engineering. The company specializes in developing immuno-oncology antibodies that target novel epitopes, utilizing its proprietary Smart Antibody Technology. This technology enhances the success rates of antibody development, significantly shortens the time required to bring products to market, and lowers associated costs. Adagene's offerings extend beyond therapeutic applications, as its technology is also applicable in diagnostics and research, aiming to advance the field of immunotherapy and improve patient outcomes. Founded in 2011, Adagene continues to innovate in the biopharmaceutical sector.
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.
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.
Unum Therapeutics is a biopharmaceutical company focused on developing immunotherapy products for cancer treatment, utilizing proprietary T-cell engineering technology. The company’s lead programs, based on its Antibody-Coupled T-cell Receptor (ACTR) technology, are in Phase I clinical trials. These include ACTR707 and ACTR087, both being tested in combination with rituximab for relapsed or refractory CD20+ non-Hodgkin lymphoma, as well as ACTR087 combined with SEA-BCMA for relapsed multiple myeloma. Additionally, ACTR707 is being evaluated with trastuzumab for HER2+ solid tumors. Unum Therapeutics is also exploring BOXR1030, a pre-clinical candidate targeting glypican-3 to enhance T cell functionality in the solid tumor microenvironment. Founded in 2014 and headquartered in Cambridge, Massachusetts, Unum aims to activate the body’s immune system to effectively combat cancer through its innovative therapies.
Cogent Biosciences Inc a biotechnology company focused on developing precision therapies for genetically defined diseases. It designs rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Its program CGT9486, is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. In the vast majority of cases, KIT D816V is responsible for driving Systemic Mastocytosis (SM), a serious disease caused by the unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancermore
REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing gene therapy product candidates that utilize its proprietary NAV Technology Platform, which is based on adeno-associated virus (AAV) gene delivery. This platform aims to address genetic defects and enable cells to produce therapeutic proteins or antibodies to combat various diseases. The company's lead candidate, RGX-314, is currently undergoing Phase I/IIa clinical trials for wet age-related macular degeneration. Other notable product candidates include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia, all in various stages of clinical trials. Additionally, REGENXBIO licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has partnered with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.
Denali Therapeutics is a biopharmaceutical company based in South San Francisco, California, dedicated to discovering and developing therapies for neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS). The company is advancing several therapeutic candidates, including LRRK2 inhibitors like DNL201 and DNL151, which are in various phases of clinical trials for Parkinson's disease. Additionally, Denali is developing DNL747, a selective RIPK1 inhibitor currently in Phase 1b trials for Alzheimer's and ALS. It also has programs focused on enzyme replacement therapy for MPS II and antibody transport vehicles targeting key proteins involved in neurodegeneration. Denali collaborates with various organizations, including Takeda Pharmaceutical Company and Genentech, to enhance its research efforts. Founded in 2013, Denali Therapeutics emphasizes a science-driven approach to address the complex challenges posed by neurodegenerative diseases, leveraging recent scientific insights and translational medicine tools.
Symbiomix Therapeutics, LLC is a biopharmaceutical company focused on developing innovative treatments for serious women's health infections, particularly bacterial vaginosis (BV). Founded in 2012, the company is based in Newark, New Jersey, with additional offices in Baltimore, Maryland, and Hamden, Connecticut. Its lead product, Solosec (secnidazole), is a novel 5-nitroimidazole antibiotic designed for a one-time oral treatment of bacterial vaginosis, addressing a significant unmet need in women's healthcare. Symbiomix operates as a subsidiary of Lupin Inc. and is supported by prominent healthcare venture capital firms. The company aims to provide effective therapeutic options for gynecologic infections that have been historically overlooked.
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.
Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who traditionally rely on insulin injections. Founded based on groundbreaking research by Professor Douglas Melton, the company has licensed a method to generate functional, insulin-producing beta cells in the laboratory. Semma Therapeutics aims to combine these proprietary cells with advanced devices to create a comprehensive therapy that can replace the missing beta cells without the need for immunosuppression. By striving to bring this transformative treatment to clinical application, Semma Therapeutics seeks to significantly enhance the lives of diabetes patients. Headquartered in Cambridge, Massachusetts, the company is a subsidiary of Vertex Pharmaceuticals and collaborates strategically with Defymed.
TraceLink, Inc. specializes in providing track and trace network solutions that enhance the life sciences supply chain and combat counterfeit prescription drugs. The company offers a range of products, including pharmaceutical serialization, drug tracking, and compliance solutions through its TraceLink Life Sciences Cloud. Key offerings include the Smart Rx Manager, which helps healthcare providers comply with the European Union Falsified Medicines Directive, and Serialized Product Intelligence, a cloud application that utilizes serialization data to identify supply chain issues and optimize operations. TraceLink serves a diverse clientele, including pharmaceutical companies, wholesale distributors, and re-packagers, and has established strategic partnerships with notable firms such as Amazon and KPMG. Founded in 2009 and headquartered in North Reading, Massachusetts, TraceLink also has offices in the United Kingdom, India, Singapore, and Brazil. The company's platform is recognized for its ability to deliver global connectivity and visibility across the pharmaceutical supply chain, ensuring compliance and improving operational efficiency.
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.
REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing gene therapy product candidates that utilize its proprietary NAV Technology Platform, which is based on adeno-associated virus (AAV) gene delivery. This platform aims to address genetic defects and enable cells to produce therapeutic proteins or antibodies to combat various diseases. The company's lead candidate, RGX-314, is currently undergoing Phase I/IIa clinical trials for wet age-related macular degeneration. Other notable product candidates include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia, all in various stages of clinical trials. Additionally, REGENXBIO licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has partnered with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.
Adagene (Suzhou) Limited is a clinical-stage biotechnology company based in Suzhou, China, focused on antibody discovery and engineering. The company specializes in developing immuno-oncology antibodies that target novel epitopes, utilizing its proprietary Smart Antibody Technology. This technology enhances the success rates of antibody development, significantly shortens the time required to bring products to market, and lowers associated costs. Adagene's offerings extend beyond therapeutic applications, as its technology is also applicable in diagnostics and research, aiming to advance the field of immunotherapy and improve patient outcomes. Founded in 2011, Adagene continues to innovate in the biopharmaceutical sector.
Cogent Biosciences Inc a biotechnology company focused on developing precision therapies for genetically defined diseases. It designs rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Its program CGT9486, is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. In the vast majority of cases, KIT D816V is responsible for driving Systemic Mastocytosis (SM), a serious disease caused by the unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancermore
Unum Therapeutics is a biopharmaceutical company focused on developing immunotherapy products for cancer treatment, utilizing proprietary T-cell engineering technology. The company’s lead programs, based on its Antibody-Coupled T-cell Receptor (ACTR) technology, are in Phase I clinical trials. These include ACTR707 and ACTR087, both being tested in combination with rituximab for relapsed or refractory CD20+ non-Hodgkin lymphoma, as well as ACTR087 combined with SEA-BCMA for relapsed multiple myeloma. Additionally, ACTR707 is being evaluated with trastuzumab for HER2+ solid tumors. Unum Therapeutics is also exploring BOXR1030, a pre-clinical candidate targeting glypican-3 to enhance T cell functionality in the solid tumor microenvironment. Founded in 2014 and headquartered in Cambridge, Massachusetts, Unum aims to activate the body’s immune system to effectively combat cancer through its innovative therapies.
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.
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.
Blueprint Medicines Corporation is a biopharmaceutical company that specializes in developing small molecule kinase inhibitors aimed at treating cancers and rare diseases driven by genomic abnormalities. The company’s pipeline includes avapritinib, which targets systemic mastocytosis and gastrointestinal stromal tumors, and BLU-263, an oral KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders. Additionally, fisogatinib is in Phase I trials for advanced hepatocellular carcinoma, while pralsetinib targets RET-altered non-small cell lung cancer and medullary thyroid carcinoma. Other investigational compounds include BLU-782, aimed at treating fibrodysplasia ossificans progressive. Established in 2008 and based in Cambridge, Massachusetts, Blueprint Medicines utilizes its proprietary chemical library and Insights-to-Validation Platform to develop personalized therapies that address specific molecular drivers of cancer and the challenges posed by resistance mechanisms. The company has formed collaboration agreements with several pharmaceutical entities to enhance its research and development efforts.
Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who traditionally rely on insulin injections. Founded based on groundbreaking research by Professor Douglas Melton, the company has licensed a method to generate functional, insulin-producing beta cells in the laboratory. Semma Therapeutics aims to combine these proprietary cells with advanced devices to create a comprehensive therapy that can replace the missing beta cells without the need for immunosuppression. By striving to bring this transformative treatment to clinical application, Semma Therapeutics seeks to significantly enhance the lives of diabetes patients. Headquartered in Cambridge, Massachusetts, the company is a subsidiary of Vertex Pharmaceuticals and collaborates strategically with Defymed.
Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.
Acacia Pharma is a hospital pharmaceutical company based in Cambridge, United Kingdom, that specializes in the discovery, development, and commercialization of supportive care drugs for patients undergoing surgery, invasive procedures, or cancer treatments. The company’s lead product, BAREMSIS, is an intravenous formulation of amisulpride designed to prevent and treat post-operative nausea and vomiting. Other notable products include APD403, which has completed Phase II trials for chemotherapy-induced nausea and vomiting, and BYFAVO, an ultra-short-acting intravenous benzodiazepine anesthetic that has completed Phase III trials for use in procedures like colonoscopy and bronchoscopy. Acacia Pharma primarily serves anesthesiologists and oncologists, with a focus on addressing issues related to nausea, vomiting, cancer-related fatigue, and cachexia. Established in 2006, the company continues to innovate in the field of cancer supportive care.
Symbiomix Therapeutics, LLC is a biopharmaceutical company focused on developing innovative treatments for serious women's health infections, particularly bacterial vaginosis (BV). Founded in 2012, the company is based in Newark, New Jersey, with additional offices in Baltimore, Maryland, and Hamden, Connecticut. Its lead product, Solosec (secnidazole), is a novel 5-nitroimidazole antibiotic designed for a one-time oral treatment of bacterial vaginosis, addressing a significant unmet need in women's healthcare. Symbiomix operates as a subsidiary of Lupin Inc. and is supported by prominent healthcare venture capital firms. The company aims to provide effective therapeutic options for gynecologic infections that have been historically overlooked.
Ultragenyx Pharmaceutical is a biopharmaceutical company based in the United States that focuses on developing therapeutics for rare and ultra-rare diseases, particularly serious genetic conditions. The company identifies, acquires, and commercializes novel treatments, leveraging existing scientific knowledge to address unmet medical needs in this challenging area. Its portfolio includes several medicines such as Crysvita, which treats X-linked hypophosphatemia in both adults and pediatric patients, Dojolvi, and Mepsevii, indicated for Mucopolysaccharidosis VII. Ultragenyx aims to create impactful therapeutics by employing effective development strategies tailored to the complexities of rare diseases.
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.
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.
Yisheng Biopharma Co., Ltd. is a biopharmaceutical company focused on the research, development, manufacturing, and marketing of immuno-oncology products and vaccines. Founded in 2002 and headquartered in Beijing, the company operates in China, the United States, Cambodia, and Singapore. Yisheng Biopharma utilizes its novel PIKA immunomodulating technology to enhance immune responses against cancers and infectious diseases. Key products under development include YS-ON-001, an investigational therapy aimed at treating advanced solid tumors, as well as YS-HBV-001, a vaccine for hepatitis B, and a PIKA-based rabies vaccine designed for rapid protection against rabies infection. The company's innovative approach targets multiple immune pathways to improve treatment outcomes.
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.
Hua Medicine is a clinical-stage, innovative drug development company in China, focused on novel therapies for the treatment of diabetes and CNS disorders. Founded by pharma industry veterans and funded by a premier group of international VC investment firms such as Fidelity Asia, Fidelity Biosciences, ARCH Ventures, Venrock, SAIL and WuXi Ventures, the company has in-licensed from major pharma, world-wide rights to a potential best-in-class, oral drug for the treatment of Type 2 Diabetes which utilizes a novel mechanism of action.
Ultragenyx Pharmaceutical is a biopharmaceutical company based in the United States that focuses on developing therapeutics for rare and ultra-rare diseases, particularly serious genetic conditions. The company identifies, acquires, and commercializes novel treatments, leveraging existing scientific knowledge to address unmet medical needs in this challenging area. Its portfolio includes several medicines such as Crysvita, which treats X-linked hypophosphatemia in both adults and pediatric patients, Dojolvi, and Mepsevii, indicated for Mucopolysaccharidosis VII. Ultragenyx aims to create impactful therapeutics by employing effective development strategies tailored to the complexities of rare diseases.
Blueprint Medicines Corporation is a biopharmaceutical company that specializes in developing small molecule kinase inhibitors aimed at treating cancers and rare diseases driven by genomic abnormalities. The company’s pipeline includes avapritinib, which targets systemic mastocytosis and gastrointestinal stromal tumors, and BLU-263, an oral KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders. Additionally, fisogatinib is in Phase I trials for advanced hepatocellular carcinoma, while pralsetinib targets RET-altered non-small cell lung cancer and medullary thyroid carcinoma. Other investigational compounds include BLU-782, aimed at treating fibrodysplasia ossificans progressive. Established in 2008 and based in Cambridge, Massachusetts, Blueprint Medicines utilizes its proprietary chemical library and Insights-to-Validation Platform to develop personalized therapies that address specific molecular drivers of cancer and the challenges posed by resistance mechanisms. The company has formed collaboration agreements with several pharmaceutical entities to enhance its research and development efforts.
NextWave Pharmaceuticals is a Cupertino, California-based pharmaceutical company that specializes in the development and commercialization of innovative drug delivery technologies. The company primarily focuses on creating once-daily liquid medications for the treatment of attention deficit/hyperactivity disorder (ADHD). By leveraging its proprietary technology, NextWave aims to provide effective treatment options that enhance patient compliance and address disorders related to the central nervous system.
Vicept (Vicept Therapeutics, Inc.), based in Malvern, Pennsylvania, is a privately held specialty biopharmaceutical company founded in August, 2009 with a mission to develop the first effective topically applied-therapy directed toward the erythema (skin redness) of rosacea and other dermatologic disorders characterized by redness and or 'flushing' of the skin.
FoldRx Pharmaceuticals, Inc. is a biotechnology company focused on discovering and developing disease-modifying drug therapies for conditions related to protein misfolding and amyloidosis. The company’s pipeline includes advanced clinical programs targeting genetic neurological and cardiovascular disorders, specifically transthyretin-associated amyloidoses that affect both polyneuropathy and cardiomyopathy. Additionally, FoldRx is engaged in early-stage discovery efforts aimed at addressing diseases such as Parkinson's and cystic fibrosis. Established in 2003 and headquartered in Cambridge, Massachusetts, the company aims to provide effective treatments for the accumulation of misfolded proteins caused by genetic mutations and deficiencies in cellular quality control mechanisms.
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