Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.
Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.
Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.
NAPIGEN is a biotechnology company focused on innovative genome engineering across a variety of organisms, including plants, microbes, and animals. The company specializes in targeting organelles such as mitochondria and chloroplasts, which contain DNA essential for cellular energy production. By harnessing the capabilities of these organelles, NAPIGEN aims to enhance biological functions and contribute to advancements in agriculture, environmental sustainability, and other sectors, ultimately improving the quality of life in society.
Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing implanted drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company utilizes a refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of its formulation of the anti-epileptic drug valproic acid, known as CT-010. This innovative approach aims to bypass the blood-brain barrier, enhancing treatment outcomes for patients with chronic neurological conditions. Founded in 2010, Cerebral Therapeutics is dedicated to improving the lives of individuals affected by uncontrolled neurological diseases.
Mineralys Therapeutics is a biopharmaceutical company that provides hypertension treatment. They also bring a targeted approach to the management of hypertension.
Concert Pharmaceuticals is a clinical-stage biopharmaceutical company focused on discovering and developing novel small molecule drugs for autoimmune and central nervous system disorders. The company employs an innovative precision deuterium chemistry platform to enhance the properties of established drug molecules, which enables the creation of new chemical entities with potential efficacy and safety advantages. Notable product candidates include CTP-543, currently in Phase III trials for alopecia areata, CTP-692 for schizophrenia in Phase II trials, and AVP-786, which targets neurological and psychiatric disorders in Phase III trials. Concert Pharmaceuticals also engages in strategic collaborations with various pharmaceutical companies to support its development efforts. Founded in 2006 and based in Lexington, Massachusetts, the company aims to streamline research and development processes, reducing associated risks and costs while advancing its therapeutic pipeline.
Celcuity Inc. is a clinical-stage biotechnology company based in Minneapolis, Minnesota, focused on advancing cancer treatment through innovative diagnostics. The company's CELsignia diagnostic platform utilizes living tumor cells to uncover specific cellular processes that drive cancer in individual patients. This approach enables the identification of new cancer sub-types that traditional molecular diagnostics may overlook, ultimately expanding therapeutic options. Celcuity is developing the CELsignia HSF test to diagnose two new sub-types of HER2-negative breast cancer and the CELsignia MP test for diagnosing 11 sub-types across various cancers, including breast, lung, colon, ovarian, kidney, and bladder. The company is also advancing its lead therapeutic candidate, gedatolisib, a pan-PI3K and mTOR inhibitor, currently being evaluated in a Phase 3 clinical trial for patients with advanced HR+/HER2- breast cancer.
Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.
Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.
Ansa Biotechnologies is manufactured via a chemical method that has remained essentially unchanged for 35 years and has approached a plateau. Ansa Biotechnologies is developing a novel DNA synthesis technology based on enzymes that will be more fast, accurate, and clean than existing methods. The technique promises to dramatically accelerate innovation in biological research and engineering.
Triana Biomedicines is focused on discovering and developing molecular glues, a novel therapeutic approach aimed at regulating challenging disease targets. The company employs a sophisticated drug discovery engine that combines high-resolution structural insights, advanced in silico tools, and custom chemical libraries. This platform is designed to stabilize existing protein interactions or create new ones, thereby influencing the functionality of disease targets. Triana has successfully validated its technology and is currently advancing multiple research programs across various disease areas, positioning itself to contribute significantly to therapeutic innovation.
Incyclix Bio develops small molecule inhibitors of CDKs for the treatment of advanced and resistant cancers. Founded by Patrick Roberts, Jay Strum, John Bisi, and Fred Eshelman in 2020 and is headquartered in Durham, North Carolina.
Ocelot Bio brings new treatment options to patients with severe liver disease. It researches and discovers medicines that help in the treatment of hepatorenal syndrome, a complication of severe liver disease.
Plexium, Inc. is a biotechnology company based in San Diego, California, that specializes in developing targeted protein degradation therapies aimed at treating cancer and neurodegenerative diseases. Founded in 2017, Plexium utilizes a platform that identifies small molecules capable of modulating E3 ligases, which play a crucial role in protein degradation. This platform includes the DELPhe screening technology, designed for high-content readouts from cell-based assays. By harnessing E3 ligases, Plexium's approach enables selective targeting of various cancers, including small cell lung and breast tumors. The company's innovative research extends beyond existing therapies, exploring new drug-like protein degraders and molecular glues to enhance treatment options for cancer and inflammatory diseases.
Somatus, Inc. is a healthcare company based in McLean, Virginia, specializing in kidney care services. Founded in 2016, it partners with health plans, health systems, nephrology, and primary care groups to offer integrated care for patients with or at risk of developing kidney disease. The company provides a range of services, including home hemodialysis, peritoneal dialysis, in-center hemodialysis, kidney transplantation, and conservative care. Somatus focuses on delaying or preventing the progression of chronic kidney disease and aims to enhance the quality of care through improved coordination and increased utilization of home dialysis and transplantation options. Its approach combines vertically integrated clinical services and technology to optimize patient outcomes.
Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.
Electra Therapeutics is a clinical stage biotechnology company developing therapies that target signal regulatory proteins (SIRP) for the treatment of immunological diseases and cancer. Electra currently has one program in clinical development and two additional preclinical programs. The company’s lead product candidate, ELA026, is a monoclonal antibody that targets SIRP on the cell surface of myeloid and T cells, and depletes pathological immune cells. ELA026 is in clinical development for secondary hemophagocytic lymphohistiocytosis (sHLH), a life-threatening hyperinflammatory condition for which there is no approved treatment.
Indapta Therapeutics is a privately held biotechnology company developing an NK (natural killer) cell therapy platform for the treatment of blood and solid tumor cancers.
Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.
Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.
Third Harmonic Bio is a clinical-stage company developing a first-in-class, highly selective, oral KIT inhibitor for treatment of severe allergy and inflammation. KIT is a cell surface receptor that acts as the master survival and functional regulator of mast cells, which are immune cells concentrated at the body’s interfaces with the external environment and that act as the key mediator of the inflammation associated with allergic diseases.
AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.
AmbAgon Therapeutics is a biotechnology company focused on developing small-molecule therapeutics for cancer treatment. The company specializes in research aimed at stabilizing the interactions of oncogenic proteins, thereby enhancing the activity of tumor-suppressing proteins. By targeting these proteins, AmbAgon Therapeutics seeks to address significant unmet clinical needs in oncology that conventional treatment methods have struggled to meet. Founded by Christian Ottmann, Lucas Brunsveld, and Michelle Arkin, the company aims to provide innovative solutions in cancer care through its unique approach to drug development.
Pardes Biosciences is a clinical-stage biopharmaceutical company focused on addressing significant health challenges, particularly those posed by viral infections. It utilizes modern reversible-covalent chemistry to discover and develop novel oral drug candidates. The company's lead product candidate, PBI-0451, is an oral antiviral designed to treat and prevent infections from SARS-CoV-2, the virus responsible for COVID-19. By reimagining the patient journey, Pardes aims to provide accessible and convenient treatment options for both adult and pediatric patients, ultimately striving to improve health outcomes and respond effectively to global health crises.
Nodexus is a biotech startup focused on providing innovative solutions for live single-cell isolation through its NX One platform. This platform features a low-cost, low-infrastructure hardware system paired with single-use disposable microfluidic cartridges, facilitating various applications in the biopharma, clinical research, and academic fields. Nodexus's technology supports critical workflows such as gene editing, cell line and antibody development, and studies of tumor heterogeneity. By offering affordable automated cell isolation, Nodexus aims to enhance biological research and development, making advanced methodologies more accessible to a wider range of clients.
Freenome, Inc. is a biotechnology company based in South San Francisco, California, founded in 2014. It specializes in developing artificial intelligence-driven genomic solutions for the early detection of cancer. The company focuses on creating simple, non-invasive blood tests that can identify early-stage cancer and assist in treatment selection, thus enabling proactive disease management. Freenome’s multi-omics platform employs proprietary algorithms to enhance disease screening processes, aiming to facilitate timely interventions and improve treatment effectiveness. In addition to its diagnostic products, Freenome also offers clinical research services to support its mission of transforming cancer management through systematic early detection.
NanoMosaic specializes in developing innovative solutions for early illness detection, prognostic monitoring, and biomarker identification. The company utilizes a unique nanoneedle proteomic platform that facilitates protein detection and quantification on a single chip. This technology enhances the capabilities of the medical and healthcare industry by accelerating the discovery and validation of biomarkers, thereby improving early disease detection and monitoring. Through its advanced proteomic system, NanoMosaic aims to contribute significantly to advancements in medical diagnostics and patient care.
Curie Therapeutics is a fully integrated, next-generation radiopharmaceuticals company focused on pioneering a new class of targeted radiopharmaceuticals that are both safe and effective. Curie's radiopharmaceuticals are composed of rationally designed conjugates against carefully selected biological targets, to deliver alpha and beta-emitting radionuclide payloads.
Avilar Therapeutics is a biopharmaceutical company dedicated to the discovery and development of extracellular protein degraders, which represent an innovative approach to targeted protein degradation. The company has created an integrated discovery platform that extends the concept of protein degradation beyond intracellular proteins. Avilar’s novel degraders are designed to bind to disease-causing proteins in circulation and shuttle them to the hepatocyte endolysosome for degradation. This mechanism aims to facilitate the treatment of serious diseases by removing unwanted proteins from the body, thereby offering potential therapeutic benefits in various medical conditions.
Antios Therapeutics is a biopharmaceutical company devoted to developing innovative therapies for viral diseases. With an experienced and proven leadership team, the company is focused on the development of its oral drug candidate for potentially curative treatment of HBV infections. Antios Therapeutics was founded by Abel De La Rosa, Douglas Mayers, and Idean Marvasty.
Cedilla is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation. The company was founded in 2018 and based in Cambridge, Massachusetts.
Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that have the potential to treat a wide range of severe health conditions. By leveraging its innovative technology, Expansion Therapeutics aims to provide effective treatment options for patients suffering from these challenging diseases.
Delix Therapeutics is a neuroscience company focused on harnessing the power of psychoplastogens—novel neuroplasticity-promoting therapeutics—to better treat mental health disorders at scale. The company was founded in 2019 and is headquartered in Boston, Massachusetts.
Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.
HilleVax is a biopharmaceutical company that focuses on the development and commercialization of novel vaccines. HilleVax was founded in 2021 and was headquartered in Boston, Massachusetts.
Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.
ARS Pharmaceuticals is a stealth pharmaceutical company that developed a novel intranasal epinephrine spray with a unique absorption technology that could be easy-to-use, convenient, and more reliable for patients and loved ones at risk of severe allergic reactions to food, medications and insect bites that could lead to life-threatening anaphylaxis.
DICE Therapeutics is a biopharmaceutical company that utilizes its proprietary technology platform to develop a pipeline of novel oral therapeutic candidates aimed at treating chronic diseases, particularly in the field of immunology. The company focuses on creating oral therapeutics that target well-established proteins, intending to achieve efficacy similar to existing systemic biologics. DICE's DELSCAPE platform is specifically designed to discover selective oral small molecules capable of modulating protein-protein interactions effectively. Its lead candidate, S011806, is an oral antagonist targeting interleukin-17, a key player in various immunological conditions. In addition to S011806, DICE is developing other oral therapeutic candidates that target α4ß7 integrin and αVß1/αVß6 integrin, aimed at treating inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.
Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that focuses on drug discovery through its innovative platform targeting solute carrier (SLC) transporters. Established in 2016, the company aims to improve patient outcomes by developing small molecule therapies for various diseases, including those related to immunometabolism, lysosomal function, and mucosal defense. By utilizing advanced chemoproteomics techniques, Jnana accelerates the identification of therapeutic targets, addressing significant unmet medical needs in areas such as immuno-oncology, inflammatory disorders, and neurological diseases. The company's approach seeks to enhance drug discovery efficiency by comprehensively targeting the vast family of membrane proteins associated with SLC transporters.
GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.
Tango Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cancer therapies through the principle of synthetic lethality. Established in 2014, the company aims to discover and target specific driver genes involved in cancer progression. Its research concentrates on three main areas: counteracting the loss of tumor suppressor genes, reversing cancer cells' ability to evade the immune system, and identifying effective drug combinations that outperform single-agent therapies. Tango's pipeline includes TNG908, a selective inhibitor of PRMT5 designed for cancer cells with an MTAP deletion, alongside other discovery programs like TNG462, TNG348, and TNG260, which target various cancer types with limited treatment options.
Sydnexis is focused on developing a proprietary treatment for pediatric progressive myopia, addressing a significant global medical need. The company manufactures a topical eyedrop formulation specifically designed to treat the progression of myopia in children. This innovative approach enables healthcare professionals to effectively manage and reduce the incidence of eye disorders in pediatric patients.
Exo is a medical device startup focused on developing a handheld ultrasound platform for imaging and therapeutic applications. The company aims to provide affordable and user-friendly medical imaging solutions for healthcare professionals globally. By integrating advanced nano-materials, innovative sensor technologies, and sophisticated signal processing with the cost efficiencies of semiconductor manufacturing, Exo significantly lowers the costs associated with medical imaging. This technology allows healthcare providers to make vital, real-time decisions that enhance patient outcomes. Exo's commitment to high-quality, accessible imaging represents a significant advancement in medical technology, making effective diagnostic tools available at an unprecedented price point.
Thriveworks is a provider of mental health services that focuses on counseling and psychiatric support for individuals facing various life challenges. The company offers a range of services, including therapy for depression and anxiety, marriage counseling, life coaching, and support for relationship issues, low self-esteem, and trauma. Thriveworks emphasizes the importance of personalized care, ensuring that clients receive tailored plans that address their unique mental health needs. Through its diverse offerings, Thriveworks aims to promote overall well-being and assist individuals in navigating their mental health concerns.
Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.
Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.
POINT Biopharma Global Inc. is a clinical-stage pharmaceutical company dedicated to the development and commercialization of radioligand therapies for cancer treatment. The company is focused on creating a robust platform for the clinical development of radiopharmaceuticals, utilizing advanced radioisotopes such as Actinium-225 and Lutetium-177. POINT Biopharma's product pipeline includes several candidates, such as PNT2002, PNT2004, PNT2003, and PNT2001, among others. By leveraging a combination of innovative manufacturing technology and direct-to-patient targeting, POINT Biopharma aims to transform theragnostic drug development and enhance the commercialization of radioligands in the fight against cancer.
Element Biosciences, Inc. is a life science company based in San Diego, California, that specializes in developing innovative genetic analysis tools for research and diagnostics. Established in 2017, the company focuses on creating a modular and high-performing DNA sequencing platform designed to provide high-quality data and workflow flexibility. Their proprietary technology enhances the signal-to-noise ratio and incorporates advancements in surface chemistry, instrumentation, and biochemistry. This approach significantly reduces both operational and capital costs while ensuring superior sequencing data quality, thereby making next-generation sequencing technology more accessible to medical researchers and improving their research capabilities.
LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies aimed at improving vision. The company's primary focus is on addressing presbyopia, a common condition that leads to the gradual loss of near vision, affecting nearly all individuals over the age of 45. LENZ Therapeutics is currently advancing an aceclidine-based eye drop specifically designed to restore near vision, addressing a significant unmet need in the market. Through its efforts, the company aims to enhance the quality of life for individuals experiencing vision changes related to aging.
Synthekine Inc. is a biotechnology company focused on developing innovative cytokine therapies and immunotherapies for cancer and autoimmune disorders. Established in 2018 and located in Menlo Park, California, the company employs advanced platforms such as engineered partial agonists and orthogonal cell therapies. Its Synthekine Platform utilizes surrogate agonists instead of traditional mutant cytokines, allowing for the creation of therapeutics that enhance efficacy while minimizing side effects. The product pipeline includes STK-009, an orthogonal ligand, and SYNCAR-001, a CAR-T therapy targeting CD-19, along with STK-012, which consists of partial agonists of IL-2. Synthekine aims to revolutionize treatment paradigms by harnessing the principles of cytokine partial agonism and immunological specificity, leading to novel immunotherapies that improve patient outcomes.
Born from the founders’ recognition that their diverse but complementary skills and experiences would enable them to successfully address challenges that others had not, the company set about solving a vexing problem: how to bring true proteomics to the world in a way that accelerates therapeutic development, dramatically improves medical diagnostics, and makes personalized and predictive medicine a reality. The extraordinary team at Nautilus represents a wide spectrum of disciplines and expertise, including protein chemists, chip designers, molecular biologists, data scientists, material scientists, biophysicists, optical engineers, microfluidics engineers, bioinformaticists, software engineers, and more. Nautilus is positioned to revolutionize proteomics, transform the way drugs are developed, and significantly improve the way human health is managed.
NiKang Therapeutics is an early-stage biotech company focused on discovering and developing innovative small molecule oncology medicines to help patients with unmet medical needs. Its discovery approach is informed by target structure biology and capitalizes on structure-based drug design enabling a rapid and efficient discovery and development of proprietary drug candidates with the most desirable pharmacological features into clinical studies.
Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact the quality of life for millions. Eliem employs its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, aiming to address unmet medical needs in these areas. Notably, its leading drug candidate, ETX-155, targets major depressive disorder and focal onset seizures. Through its efforts, Eliem Therapeutics seeks to improve the lives of patients suffering from these debilitating disorders.
Larimar Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing treatments for rare diseases. Headquartered in Bala Cynwyd, Pennsylvania, the company is primarily focused on its lead compound, CTI-1601, which is currently undergoing a Phase 1 clinical program aimed at treating Friedreich’s ataxia, a rare and progressive genetic disorder. CTI-1601 is designed as a subcutaneously administered recombinant fusion protein that delivers human frataxin, an essential protein, to the mitochondria of patients affected by this condition. Additionally, Larimar aims to leverage its intracellular delivery platform to create other fusion proteins targeting additional rare diseases associated with deficiencies in intracellular bioactive compounds.
Interius BioTherapeutics is a biopharmaceutical company that develops in vivo cell-specific gene medicines to treat B cell lymphomas. Intravenous in vivo CAR treatment, which treats B cell lymphomas, is the company's flagship program. Its unique engineering provides exceptional target tissue specificity. The business is working on a second initiative to address autoimmune conditions. With the help of Interius, a novel and distinct therapeutic modality for the precise administration of gene therapies may soon be made available to patients in expanded care settings, without the need for preconditioning chemotherapy.
Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.
Ceribell, Inc. is a medical technology company that designs and manufactures devices for electroencephalography (EEG) data acquisition and interpretation, aimed at improving the diagnosis and management of neurological conditions. Its flagship product, the Ceribell EEG System, includes a flexible EEG Headband that accommodates various hair types and head sizes, alongside a portable EEG Recorder that ensures clinical-quality data and on-device display. The system is designed to be set up by any healthcare provider within six minutes, making EEG testing more accessible and efficient. Notably, it features a unique function that converts brainwaves into sound, allowing for quicker detection of seizures without the need for specialized technicians. This innovation facilitates earlier diagnosis and targeted treatment for patients, particularly for non-convulsive seizures, thereby reducing the risk of complications and improving overall patient outcomes. Established in 2014 and based in Mountain View, California, Ceribell is committed to enhancing care in hospital ICUs, inpatient units, and emergency departments.
Forge Biologics is a gene therapy development engine, focused on enabling access to life-changing gene therapies and helping bring them from an idea into reality. The company partners with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups. It was founded in 2020 and headquartered in Grove City, Ohio.
Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.
ViaCyte is a preclinical therapeutic company specializing in regenerative medicine therapies for diabetes. Their therapy is based on the differentiation of stem cells into pancreatic beta-cell precursors, with subcutaneous implantation in an encapsulation device. Data in their publications demonstrate that these cells can produce therapeutically relevant levels of insulin in response to blood glucose, and sustain diabetic animals. Their goal is a product which can free both Type 1 and Type 2 patients with diabetes from insulin dependence on a long-term basis, while reducing or eliminating hypoglycemic, microvasculature, and weight-related cardiovascular complications.
Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at generating immune responses to combat tumors while preserving healthy tissue. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux targets all three stages of the anti-tumor immune response. The company's approach is designed to enhance the efficacy of cancer treatments by specifically engaging the immune system to identify and eliminate cancer cells.
Invivyd is a biopharmaceutical company committed to developing antibody-based therapies aimed at providing protection against serious viral infectious diseases. The company focuses on creating solutions that go beyond naturally occurring immunity, particularly in response to viral threats with pandemic potential, starting with SARS-CoV-2. Its product pipeline includes ADG20, which is designed for the treatment and prevention of COVID-19, and VYD222, a monoclonal antibody engineered for enhanced potency and broad coverage. By prioritizing rapid and enduring therapeutic options, Invivyd aims to safeguard vulnerable populations from the severe impacts of circulating viral infections.
Antios Therapeutics is a biopharmaceutical company devoted to developing innovative therapies for viral diseases. With an experienced and proven leadership team, the company is focused on the development of its oral drug candidate for potentially curative treatment of HBV infections. Antios Therapeutics was founded by Abel De La Rosa, Douglas Mayers, and Idean Marvasty.
Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.
Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.
New Equilibrium Biosciences is focused on developing therapeutic drugs aimed at treating cancer and neurodegenerative diseases. The company employs a unique approach that integrates artificial intelligence with quantum chemistry to enhance biophysical experiments. This innovative platform specializes in the research and development of therapeutics that target intrinsically disordered proteins (IDPs), which are often implicated in various neurodegenerative disorders. By utilizing IDP-specific compound libraries, New Equilibrium Biosciences seeks to identify and exploit transient binding pockets on these proteins, facilitating the discovery of lead drug candidates. This approach aims to improve medical procedures and ultimately contribute to advancements in healthcare.
Tyra Biosciences is a clinical-stage biotechnology company based in Carlsbad, California, that specializes in developing small molecule therapies for cancer treatment. Founded in 2018, the company aims to address drug resistance in cancer therapies through its proprietary SNÅP platform, which facilitates rapid and precise drug design by generating iterative molecular snapshots. Tyra is particularly focused on creating selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. The company's lead product candidate, TYRA-300, specifically targets FGFR3 and is initially aimed at patients with metastatic urothelial carcinoma of the bladder and urinary tract. Through its innovative approach, Tyra Biosciences seeks to improve treatment outcomes for cancer patients by providing alternatives to existing therapies.
Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.
Pyxis Oncology is a developer of antibody therapeutics intended to promote the body's immune response to cancer. The company has engaged in deep analyses of tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and of tumor cell signaling pathways within cold tumors, providing laboratories with development of novel antibody-based immunotherapies. It was founded in 2019 and is based in Boston, Massachusetts.
Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact the quality of life for millions. Eliem employs its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, aiming to address unmet medical needs in these areas. Notably, its leading drug candidate, ETX-155, targets major depressive disorder and focal onset seizures. Through its efforts, Eliem Therapeutics seeks to improve the lives of patients suffering from these debilitating disorders.
Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.
Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at generating immune responses to combat tumors while preserving healthy tissue. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux targets all three stages of the anti-tumor immune response. The company's approach is designed to enhance the efficacy of cancer treatments by specifically engaging the immune system to identify and eliminate cancer cells.
Century Therapeutics is a biotechnology company focused on developing advanced cell therapies using induced pluripotent stem cells (iPSCs) to treat various cancers, including hematologic and solid tumors. Founded in 2018 and based in Philadelphia, Pennsylvania, the company utilizes a unique allogeneic iPSC-derived cell therapy platform that integrates gene editing, protein engineering, and robust manufacturing capabilities. This innovative approach allows for the generation of modified immune effector cells, such as natural killer (NK) and T cells, designed to meet significant unmet medical needs. By employing techniques like CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to enhance cell product performance and minimize rejection by the host immune system, thereby improving the efficacy of cancer treatments.
Tenaya Therapeutics is a developer of novel therapies designed to offer treatment for heart disease. The company's therapies address heart failure through multipronged efforts that target the fundamental cellular pathologies present in diseased cardiac muscle and that leverage cutting-edge research in cardiac development and regeneration, enabling physicians to regenerate heart tissue, and additional programs focused on cardiomyopathies.
DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.
Satsuma Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.
Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.
Xilio Therapeutics is a clinical-stage biotechnology company focused on developing innovative immunotherapies to enhance the immune system's ability to combat cancer. The company specializes in tumor-selective immunotherapies that leverage its proprietary technology to maximize the effectiveness of existing immuno-oncology treatments while minimizing side effects outside the tumor. Its lead product, XTX201, is designed to induce immune activity specifically within tumors, demonstrating improved antitumor efficacy. Xilio Therapeutics aims to deliver significant improvements in patient outcomes by utilizing its platform to create a pipeline of novel therapies, including tumor-activated cytokines, antibodies, and immune cell engagers, all targeted to optimize therapeutic effects within the tumor microenvironment. Founded in 2015 and based in Waltham, Massachusetts, the company was previously known as Akrevia Therapeutics before rebranding in March 2020.
Vividion is a biotechnology company focused on developing innovative therapeutics that treat major unmet clinical needs using the first platform for proteome-wide drug and target discovery. Making accessible the broad set of proteins expressed in human cells, the company’s platform was spun out of the labs of Vividion’s scientific founders, a team of experts in chemical biology and synthetic chemistry from The Scripps Research Institute in La Jolla, CA. Vividion is committed to advancing and applying its pioneering synthetic and proteomic chemistry platforms to create therapeutics that will make a transformative difference in patients.
Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing innovative therapies that leverage the biology of DNA repair for cancer treatment and synthetic lethality in autoimmune diseases. The company utilizes its expertise in DNA damage response biology to create a pipeline of small molecule therapeutics aimed at selectively targeting vulnerabilities in cancer cells and inducing their self-destruction through DNA damage overload. Cyteir's lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, which is crucial for repairing double-strand DNA breaks. The company is dedicated to addressing a variety of hematological malignancies, solid tumors, and chronic autoimmune disorders through its integrated drug development platform. Founded in 2012, Cyteir is committed to advancing next-generation therapies that can improve patient outcomes in challenging disease states.
Day One Biopharmaceuticals is a clinical-stage biotechnology company that specializes in developing targeted cancer therapies for patients of all ages, with a particular focus on pediatric patients. Recognizing the advancements in cancer research, the company aims to create treatments that can benefit both children and adults diagnosed with genetically defined cancers. Day One Biopharmaceuticals prioritizes the rapid development of therapies to meet the urgent needs of families facing cancer diagnoses. Their lead product candidate, DAY101, is an oral, highly-selective pan-RAF kinase inhibitor designed to penetrate the brain, addressing a significant treatment gap in pediatric oncology. By licensing and acquiring promising products from research institutions and other companies, Day One Biopharmaceuticals strives to bring effective medicines to market for those impacted by cancer.
Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.
Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.
TScan Therapeutics is a biopharmaceutical company dedicated to discovering and developing innovative T cell receptor (TCR) engineered therapies to combat various cancers. Based in Waltham, Massachusetts, the company focuses on TCR-T therapies for both liquid cancers and solid tumors. Its lead candidates, TSC-100 and TSC-101, are designed to address hematologic malignancies, specifically targeting residual leukemia and preventing relapse after hematopoietic stem cell transplantation. In addition, TScan is advancing a multiplexed TCR-T therapy program aimed at treating a range of solid tumors. The company's proprietary platform identifies clinically relevant T cell antigens and minimizes off-target effects, enhancing the precision of its therapies. Founded in 2018, TScan Therapeutics is committed to improving outcomes for patients with serious diseases.
Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.
DICE Therapeutics is a biopharmaceutical company that utilizes its proprietary technology platform to develop a pipeline of novel oral therapeutic candidates aimed at treating chronic diseases, particularly in the field of immunology. The company focuses on creating oral therapeutics that target well-established proteins, intending to achieve efficacy similar to existing systemic biologics. DICE's DELSCAPE platform is specifically designed to discover selective oral small molecules capable of modulating protein-protein interactions effectively. Its lead candidate, S011806, is an oral antagonist targeting interleukin-17, a key player in various immunological conditions. In addition to S011806, DICE is developing other oral therapeutic candidates that target α4ß7 integrin and αVß1/αVß6 integrin, aimed at treating inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.
Werewolf Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapeutics that enhance the body’s immune response to cancer. Founded in 2017, the company utilizes its proprietary PREDATOR platform to engineer conditionally activated molecules, known as INDUKINE molecules. These therapeutics are designed to remain inactive in peripheral tissues but activate selectively within the tumor microenvironment, addressing the limitations of traditional proinflammatory immune therapies. By stimulating both adaptive and innate immunity, Werewolf Therapeutics aims to improve cancer treatment outcomes.
Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Based in Newton, Massachusetts, Abcuro creates therapeutic antibodies targeting novel mechanisms related to these diseases. The company utilizes advanced bioinformatics to analyze transcription data from human diseases, which aids in the identification of new therapeutic targets. Through ex-vivo validation in diseased tissues, Abcuro seeks to uncover innovative methods to modulate key components of the immune system. Established in 2015, Abcuro aims to enhance treatment strategies in the healthcare industry by offering effective solutions for complex immunological conditions.
Vivace Therapeutics, Inc. is a drug discovery and development company focused on creating cancer therapies that target the Hippo pathway, which plays a crucial role in tissue regeneration and organ development. Established in 2014 and located in the San Francisco Bay Area, Vivace Therapeutics employs an innovative approach to develop small molecule treatments aimed at improving patient outcomes in oncology. The company collaborates with leading academic institutions to advance research and develop therapies that can be used alone or in combination with existing treatments. With a commitment to enhancing cancer care, Vivace Therapeutics is dedicated to delivering novel solutions that can help patients combat cancer more effectively.
Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.
Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, that specializes in developing small molecule therapies for severe, rare musculoskeletal diseases. Founded in 2017, the company employs a precision medicine approach to create innovative treatments targeting various muscle disorders, including Duchenne and Becker muscular dystrophies, as well as limb girdle muscular dystrophies. Utilizing its proprietary muscle-focused drug discovery platform, Edgewise integrates expertise in muscle biology and small molecule engineering to identify and develop therapies that address genetically defined muscle disorders. The company emphasizes a holistic drug discovery method that assesses integrated muscle function, enabling it to create orally bioavailable therapies aimed at improving outcomes for patients with significant unmet medical needs in neuromuscular and cardiac diseases.
Acumen Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing therapies for Alzheimer's disease and related neurodegenerative conditions. The company is advancing a targeted immunotherapy drug candidate, ACU193, which is designed to selectively target amyloid-beta oligomers, believed to be a key factor in the progression of Alzheimer's disease. Acumen employs a sensitivity assay to measure soluble amyloid-beta oligomer levels in cerebrospinal fluid, which aids in the enrollment of patients for clinical trials. The Phase 1 clinical trial for ACU193 began in 2021, aiming to assess its safety, tolerability, and pharmacokinetics in patients with mild cognitive impairment and mild dementia resulting from Alzheimer's disease. Founded in 1996 and based in Livermore, California, Acumen Pharmaceuticals is committed to addressing the challenges posed by Alzheimer’s disease through innovative therapeutic approaches.
Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.
Adagio Medical is engaged in the research and development of treatments for cardiovascular diseases. Adagio Medical is committed to providing electrophysiologists innovative cryoablation catheters that create safe, continuous, transmural lesions to treat cardiac arrhythmias anywhere in the heart, including paroxysmal and persistent atrial fibrillation, atrial flutter, and ventricular tachycardia. The company was founded in 2011 and is based in Laguna Hills, California.
Cytek Biosciences, Inc. is a manufacturer and supplier of flow cytometry tools and equipment, primarily serving cancer and cell biology research. The company offers a range of products including the Cytek Aurora and Cytek Northern Lights flow cytometers, the DxP Athena flow cytometry system that utilizes advanced technology to resolve dim populations in a multicolor format, and the QbSure quality control program for assessing cytometer performance. Additionally, Cytek provides proprietary cFluor reagents designed to optimize instrument performance in multicolor applications. Established in 1990 and headquartered in Fremont, California, Cytek has expanded its presence with locations in Bethesda, Maryland; Amsterdam, the Netherlands; Tokyo, Japan; and Shanghai, China. The company, formerly known as Cytoville, Inc., rebranded to Cytek Biosciences, Inc. in August 2015.
Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.
AavantiBio, Inc. is a biopharmaceutical company based in Gainesville, Florida, focused on developing gene transfer and gene editing therapies for rare genetic diseases. Established in 2019, the company primarily targets Friedreich’s Ataxia, a severe inherited condition that leads to significant cardiac and central nervous system dysfunction. In addition to addressing this specific disorder, AavantiBio is advancing a pipeline of innovative gene therapies aimed at treating various autoimmune diseases and other conditions with unmet medical needs. Through its commitment to research and development, the company seeks to improve the lives of patients affected by these debilitating genetic disorders.
Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.