Perceptive Advisors LLC is an investment management firm founded in 1999 and headquartered in New York City, with an additional office in Boston. The firm specializes in the healthcare sector, focusing on biotechnology, pharmaceuticals, life sciences, diagnostics, digital health, medical devices, and healthcare services. It engages in venture capital and incubation, investing at various stages of a company's life cycle, including seed and Series A funding, as well as late-stage venture and public market investments. Perceptive Advisors employs a long/short equity strategy and utilizes hedging techniques, including equity options and index options, while conducting in-house research to inform investment decisions. The firm also manages separate client-focused equity portfolios and provides customized debt financing solutions through its Perceptive Credit Opportunities Fund. Its clientele includes institutional investors, financial institutions, investment funds, and high-net-worth individuals.
MeiraGTx is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company has a diverse pipeline that includes several ongoing clinical programs focused primarily on treating conditions related to the eye, salivary gland, and central nervous system. Key product candidates include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, leveraging its expertise in viral vector design and gene therapy manufacturing. With a portfolio approach to technology development, the company plans to expand its focus to include additional gene therapy treatments for various serious diseases in the future.
Cerebral Therapeutics, Inc. is a pharmaceutical company that develops implanted drug-device combination therapies for the treatment of neurologic diseases. The company uses implanted, refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose for the formulation of anti-epileptic drug valproic acid, called CT-010, to treat refractory epilepsy. Cerebral Therapeutics, Inc. was formerly known as Cerebral Therapeutics LLC. The company was incorporated in 2010 and is based in Aurora, Colorado.
Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.
Neuron23, Inc. develops and manufactures medicines for genetic disorders using advanced artificial intelligence technology and genetics. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company focuses on creating therapeutics for neurological diseases, including Parkinson's and Alzheimer's, as well as immunological disorders. By leveraging recent advancements in human genetics alongside its AI-enabled drug discovery and biomarker platforms, Neuron23 aims to provide innovative treatments for patients suffering from these debilitating conditions, enhancing their quality of life through targeted and effective therapies.
Endeavor BioMedicines is a clinical-stage company focused on creating innovative treatments for pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). The company is developing a small-molecule inhibitor aimed at addressing the underlying causes of this disease. This potential therapy not only seeks to halt the progression of IPF but may also reverse its effects, thereby providing healthcare professionals with precision treatment options for this terminal condition. By targeting the root causes of the disease, Endeavor BioMedicines aims to improve patient outcomes and enhance quality of life for those affected by pulmonary fibrosis.
AvengeBio is focusing on Cell-Generated Immunotherapies to eradicate solid tumors. It is a developer of a drug delivery platform intended to treat cancer. The company was founded in 2019 and based in Cambridge, Massachusetts.
Mythic Therapeutics is a biotechnology company focused on a new generation of groundbreaking cancer therapeutics. The company is pioneering a powerful protein engineering-focused approach to improving the effectiveness of antibody-drug conjugates (ADCs) and other antibody-based therapeutics. Their technology dramatically increases therapeutic potency without compromising safety, thereby unlocking the full potential of targeted therapies against a broad array of tumor targets. Mythic Therapeutics was founded in 2017 and is headquartered in Waltham, MA, USA.
Sermonix Pharmaceuticals, Inc. is a biotechnology company based in Columbus, Ohio, founded in 2014. The company specializes in the development and commercialization of female-specific oncology products, particularly focusing on late-stage women's health issues. Its primary product, lasofoxifene, is a selective estrogen receptor modulator designed for the treatment of vulvovaginal atrophy and for both the treatment and prevention of post-menopausal osteoporosis. Additionally, Sermonix is involved in developing pharmaceutical drugs aimed at treating ESR1-mutated metastatic breast and gynecological cancers, thereby enhancing the quality of care for women facing these health challenges.
Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing oncology medicines tailored to individual patients. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments, thereby enhancing the effectiveness of drug development. Acrivon's lead program is ACR-368, also known as prexasertib, which is currently in Phase 2 trials. Additionally, the pipeline includes preclinical programs that target critical components of the DNA Damage Response and cell cycle regulation pathways, specifically focusing on proteins such as WEE1 and PKMYT1. By leveraging these innovative approaches, Acrivon aims to improve therapeutic outcomes for cancer patients.
CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, a novel aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its promising clinical candidates, CinCor Pharma seeks to gain marketing approval for innovative therapies that can effectively address unmet medical needs in these critical areas of health.
Science 37, Inc. is a technology-driven clinical trial company focused on transforming clinical research through patient-centric models. It has developed NORA (Network Oriented Research Assistant), a cloud-based mobile research platform that facilitates communication between researchers and patients via videos, photographs, and surveys, enabling end-to-end networked clinical trial services. The company also offers the Science 37 Metasite, which enhances access to diverse patient populations for clinical studies, and the Science 37 Platform for decentralized clinical trials. By utilizing its virtual model, Science 37 has demonstrated faster enrollment and higher patient retention, reaching a broader and more representative patient demographic than traditional site-based research. The company serves a wide range of clients, including large pharmaceutical firms, biotechnology companies, universities, and startups. Since its incorporation in 2014, Science 37 has established itself as a leader in virtual clinical trials, leveraging an extensive network of telemedicine investigators and home-health nurses to conduct more decentralized interventional trials than any other organization.
HilleVax is a clinical-stage biopharmaceutical company established in 2021 and based in Boston, Massachusetts. The company specializes in the development and commercialization of innovative vaccines. Its primary focus is on HIL-214, a vaccine candidate that utilizes virus-like particle (VLP) technology aimed at preventing moderate-to-severe acute gastroenteritis caused by norovirus infection.
Zentera Therapeutics
Series B in 2021
Zentera Therapeutics is a biopharmaceutical company.
Amylyx Pharmaceuticals, Inc. develops novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. It offers AMX0035, an investigational therapy and a fixed-dose co-formulation of two active compounds, such as sodium phenylbutyrate and Taurursodiol optimized to treat both the energy crisis in the mitochondria and the toxic, unfolded proteins in the endoplasmic reticulum, disrupting the neurological chain of events that leads to patient suffering. The company’s pipeline includes amyotrophic lateral sclerosis, Wolfram syndrome, and Alzheimer’s diseases. Amylyx Pharmaceuticals, Inc. was founded in 2013 and is based in Cambridge, Massachusetts.
POINT Biopharma Global Inc. is a globally focused radiopharmaceutical company building a platform for the clinical development and commercialization of radioligands that fight cancer. POINT Biopharma Global Inc. is combining a portfolio of best-in-class radiopharmaceutical assets, a seasoned management team, expertise in radioisotopes such as Actinium-225 (Ac-225) and Lutetium-177 (Lu-177), manufacturing technology and novel direct-to-patient targeting to revolutionize theragnostic drug development and radioligand commercialization.
RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing tumor-targeted small molecule medicines that utilize radioisotopes, particularly focusing on alpha-emitting isotopes like Actinium-225 for the treatment of solid tumors. RayzeBio aims to improve cancer treatment outcomes by creating a robust pipeline of radiopharmaceuticals targeting validated oncology drug targets. Its portfolio includes multiple drug candidates at various stages of development, ranging from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.
NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.
Juno Diagnostics, Inc. is a health technology company based in San Diego, California, focused on democratizing access to genetic information. Established in 2017, Juno specializes in cell-free DNA-based prenatal testing and is also engaged in the development of genetic testing tools for various applications, including epigenetics, inherited diseases, and cancer diagnostics. The company aims to create simple and cost-effective devices that facilitate point-of-need genetic testing, thereby enhancing accessibility to vital health information.
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.
Pyxis Oncology, Inc. is a biotechnology company focused on developing antibody therapeutics that enhance the immune response against cancer. Founded in 2018 and based in Boston, Massachusetts, the company specializes in analyzing tumor antigen-specific tumor-infiltrating lymphocytes within hot tumors and tumor cell signaling pathways in cold tumors. This research informs the creation of novel antibody-based immunotherapies aimed at targeting difficult-to-treat cancers. Pyxis Oncology's goal is to develop next-generation therapeutics that not only kill tumor cells but also address the underlying issues that facilitate cancer's proliferation and immune evasion, ultimately improving the quality of life for patients.
OnKure, Inc. engages in the discovery and development of epigenetic therapies for the treatment of cancer. The company focuses on developing selective inhibitors of histone deacetylases. Its portfolio includes ONK102, an inhibitor that reduces the growth rate of tumors in hematological and solid tumor cancers. OnKure, Inc. was incorporated in 2011 and is based in Boulder, Colorado.
Pipeline Therapeutics Inc. is a biotechnology company focused on developing and commercializing small molecules aimed at neuroregeneration, which includes promoting synaptogenesis, remyelination, and axonal repair. Based in San Diego, California, the company’s lead product candidate, PIPE-505, is a gamma secretase inhibitor designed to treat mild-to-moderate sensorineural hearing loss associated with cochlear synaptopathy. Additionally, Pipeline Therapeutics is advancing earlier-stage programs, such as PIPE-307, which target remyelination and axonal repair to address various neurological disorders, including multiple sclerosis. Founded in 2017, the company aims to harness the body's natural repair mechanisms to facilitate recovery from neurological damage.
Day One Biopharmaceuticals is a clinical-stage biotechnology company focused on developing targeted therapies for genetically defined cancers in patients of all ages, with an initial emphasis on pediatric patients. Founded in 2018 and headquartered in South San Francisco, California, the company aims to address the critical needs of children and adults diagnosed with cancer by creating innovative treatments derived from the biology of childhood cancer. Day One Biopharmaceuticals prioritizes programs that can be swiftly brought to market, thereby providing timely options for families facing cancer diagnoses. Its lead product candidate, DAY101, is an oral, brain-penetrant pan-RAF kinase inhibitor designed to target specific cancer types. The company seeks to license and acquire promising therapeutic products from research institutions and other biotechnology and pharmaceutical companies, striving to enhance the treatment landscape for underserved populations.
Earli is focused on transforming cancer detection and treatment by developing innovative technologies that enable early identification of tumors. The company specializes in creating synthetic biopsies, which are designed to induce cancer cells to produce non-human synthetic biomarkers. These biomarkers can be detected in biological samples such as blood, urine, or breath, allowing for the visualization of tumors that are typically too small to identify in traditional screenings. By leveraging these programmable genetic constructs, Earli aims to provide real-time data on cancer presence and progression, facilitating personalized treatment approaches and improving the chances of successful intervention. Overall, Earli's mission is to make the experience of cancer more manageable by ensuring timely detection and response.
IsoPlexis Corp is a life sciences company based in Branford, Connecticut, that specializes in developing a diagnostic platform designed to measure cellular immune responses in patients. Founded in 2013, the company's technology focuses on single-cell detection, allowing for the identification of diverse immune responses at the individual cell level. This platform plays a crucial role in assessing the safety and efficacy of therapeutics, as well as monitoring disease progression. By providing detailed insights into immune responses, IsoPlexis aims to enhance therapeutic development and inform clinical decision-making for healthcare professionals.
IsoPlexis Corp is a life sciences company based in Branford, Connecticut, that specializes in developing a diagnostic platform designed to measure cellular immune responses in patients. Founded in 2013, the company's technology focuses on single-cell detection, allowing for the identification of diverse immune responses at the individual cell level. This platform plays a crucial role in assessing the safety and efficacy of therapeutics, as well as monitoring disease progression. By providing detailed insights into immune responses, IsoPlexis aims to enhance therapeutic development and inform clinical decision-making for healthcare professionals.
Neuron23, Inc. develops and manufactures medicines for genetic disorders using advanced artificial intelligence technology and genetics. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company focuses on creating therapeutics for neurological diseases, including Parkinson's and Alzheimer's, as well as immunological disorders. By leveraging recent advancements in human genetics alongside its AI-enabled drug discovery and biomarker platforms, Neuron23 aims to provide innovative treatments for patients suffering from these debilitating conditions, enhancing their quality of life through targeted and effective therapies.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, focused on developing patient-specific immunotherapies to treat cancer. Founded in 2016, the company creates personalized vaccines and T cell therapies that harness the immune system to target and eliminate cancer cells. By identifying truncal tumor neo-antigens and other cancer-specific markers, Achilles Therapeutics aims to develop next-generation therapies that selectively attack tumors while sparing healthy tissues. Currently, the company is conducting two clinical trials: the CHIRON trial for patients with non-small-cell lung cancer and the THETIS trial for those with recurrent or metastatic melanoma.
Prometheus Biosciences, formerly known as Prometheus Laboratories, is a biotechnology company focused on developing and commercializing innovative pharmaceutical and diagnostic products, primarily for gastrointestinal diseases such as inflammatory bowel disease (IBD). Based in San Diego, California, the company offers a range of diagnostic tests, including Anser IFX, Anser ADA, and IBD sgi Diagnostic, which enable healthcare providers to monitor treatment and optimize patient care. Prometheus also markets therapeutic agents, such as Entocort EC and Imuran, for managing conditions like Crohn's disease and rheumatoid arthritis. Additionally, it provides genetic and serologic testing for celiac disease and lactose intolerance, among other disorders. With a commitment to precision medicine, Prometheus aims to enhance individualized patient care through its advanced diagnostic capabilities and therapeutic solutions. The company was founded in 1995 and operates as a subsidiary of Precision IBD, Inc.
Nuvation Bio Inc., a biopharmaceutical company, focuses on the development of therapies for oncology. Its portfolio includes various oncology programs with multiple drug development candidates. Nuvation Bio Inc.was formerly known as RePharmation Inc. and changed its name to Nuvation Bio Inc. in April 2019. The company was founded in 2018 and is based in New York, New York with an additional office in San Francisco, California.
Escient Pharmaceuticals, Inc., a biotechnology company, develops and manufactures G protein-coupled receptor (GPCR)-targeted drugs for treating neuro-immuno-inflammatory and autoreactive diseases. The company focuses on unleashing the therapeutic potential of specific orphan GPCRs, including the novel family of Mas-Related G-Protein Receptors (Mrgprs) for neuro-immuno-inflammatory and autoreactive diseases. Escient Pharmaceuticals, Inc. was formerly known as Mas Therapeutics, Inc. The company was founded in 2017 and is based in San Diego, California.
I-Mab HK is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative biologics aimed at addressing significant unmet medical needs, particularly in the areas of cancer and autoimmune disorders. The company is engaged in developing a diverse pipeline of product candidates, including Felzartamab, a CD38 antibody in Phase III trials for multiple myeloma, and Eftansomatropin, a long-acting human growth hormone for pediatric growth hormone deficiency. Other notable candidates include Olamkicept, an IL-6 blocker for ulcerative colitis, and Enoblituzumab, a B7-H3 antibody for head and neck cancer. I-Mab HK also has preclinical compounds targeting various conditions, including cancers and autoimmune diseases. The company has established strategic collaborations, such as with AbbVie, to enhance its development efforts. Founded in 2014 and headquartered in Shanghai, I-Mab HK is dedicated to advancing therapies that improve patient outcomes across various therapeutic areas.
Rain Therapeutics Inc. is a biotechnology company focused on developing targeted therapies for cancer patients, particularly those with unmet medical needs. Established in 2017 and based in Newark, California, the company specializes in small molecule therapeutics. Its lead product, RAIN-32, is a small molecule MDM2 inhibitor aimed at treating well-differentiated and de-differentiated liposarcoma. Additionally, Rain Therapeutics is developing tarloxotinib, a hypoxia-activated pan-HER inhibitor currently undergoing clinical trials for non-small cell lung cancer. The company emphasizes identifying cancers driven by specific oncogenic mutations and evaluates sub-populations where existing treatment options are inadequate. Through collaboration with a global network of scientific and thought leaders, Rain Therapeutics seeks to align its therapeutic innovations with the needs of these cancer populations.
ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, that specializes in the discovery and development of antiviral drugs targeting the respiratory syncytial virus (RSV). Founded in 2011, ReViral focuses on creating novel first-in-class compounds, including a highly potent fusion inhibitor designed to treat severe RSV infections, particularly in vulnerable populations such as neonates. The company's innovative approach aims to expand the antiviral market by providing effective treatment options for patients affected by RSV, a virus known for causing significant respiratory illness. Through its research and development efforts, ReViral seeks to address unmet medical needs in the realm of viral infections.
Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and based in San Mateo, California, the company concentrates on targeting dysregulated transcription factors and the associated transcriptional regulatory networks that contribute to cancer progression. Its lead candidate, entospletinib, is a selective inhibitor aimed at spleen tyrosine kinase for treating acute myeloid leukemia. Additionally, Kronos Bio is advancing KB-0742, an orally bioavailable inhibitor of cyclin dependent kinase 9, intended for the treatment of MYC-amplified solid tumors. The company employs advanced techniques, including high-throughput small-molecule microarrays and targeted protein degradation, to discover potent compounds that can effectively modulate challenging cancer targets.
Taysha Gene Therapies is a company focused on developing adeno-associated virus (AAV) based gene therapies aimed at treating monogenic diseases of the central nervous system. Founded in 2019 and based in Dallas, Texas, the company is advancing several therapeutic candidates, including TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies collaborates with The University of Texas Southwestern Medical Center to enhance the development and commercialization of its innovative treatments, with a mission to eradicate severe and life-threatening CNS diseases through curative gene therapies. The company's expertise in drug development and its partnership with a leading gene therapy program enable it to effectively progress its pipeline of potential therapies.
Thrive Earlier Detection Corp. is a healthcare company dedicated to improving cancer detection through innovative technology. It focuses on the development of CancerSEEK, a liquid biopsy test that analyzes genomic mutations in tumor DNA and protein markers in plasma to identify multiple types of cancer at their earliest stages, often before symptoms become apparent. Founded in 2018 and headquartered in Cambridge, Massachusetts, with an additional office in Baltimore, Maryland, Thrive aims to integrate early cancer detection into routine medical care. The company was previously known as Thrive Sciences Inc. and has been a subsidiary of Exact Sciences Corporation since early 2021. Thrive was launched with support from various investors, including Third Rock Ventures and Section 32, reflecting a strong commitment to advancing cancer diagnostics.
Cerevel (www.cerevel.com) is a biopharmaceutical company focused on developing drug candidates to treat disorders of the central nervous system (CNS). The company has a portfolio of pre-commercial neuroscience assets, which include three clinical-stage compounds and several pre-clinical compounds designed to target a broad range of CNS disorders including Parkinson’s, Alzheimer’s, epilepsy, schizophrenia and addiction. Headquartered in the Greater Boston area, Cerevel was formed in 2018 through a partnership between Bain Capital and Pfizer.
Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, dedicated to leveraging the body's natural biology to repair or reverse damage from various degenerative diseases. The company employs a unique Progenitor Cell Activation approach, utilizing combinations of small molecules to stimulate progenitor cells to regenerate functional tissue. Its lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at addressing the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is developing therapies for a variety of degenerative conditions, including multiple sclerosis and ailments affecting muscle, skin, gastrointestinal tract, and bone health. The company has also established a collaboration agreement with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics is focused on disease modification through innovative therapeutic strategies.
Verona Pharma is a clinical-stage biopharmaceutical company headquartered in London, United Kingdom, that specializes in developing therapies for chronic respiratory diseases with significant unmet medical needs. The company's lead product candidate, ensifentrine, is an inhaled dual inhibitor of phosphodiesterase 3 and 4, functioning as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is currently in Phase 2b clinical development as a nebulized formulation for the maintenance treatment of chronic obstructive pulmonary disease (COPD), and alternative delivery methods, including a dry powder inhaler and a pressurized metered-dose inhaler, are also being explored. In addition to COPD, Verona Pharma aims to develop ensifentrine for other respiratory conditions such as cystic fibrosis and asthma. Founded in 2005, the company is committed to improving the health and quality of life for individuals affected by these challenging diseases.
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.
AsclepiX Therapeutics, Inc. is a biopharmaceutical company focused on the design and development of innovative treatments for diseases related to abnormal blood vessel growth, specifically through the use of short biomimetic anti-angiogenic and anti-lymphangiogenic peptides. The company's lead candidates, including AXT107, AXT201, AXT301, and AXT501, target conditions such as diabetic macular edema, wet age-related macular degeneration, and macular edema following retinal vein occlusion, which are among the leading causes of blindness. Additionally, AXT201, AXT301, and AXT501 are being explored for their potential in treating various solid tumors. AsclepiX leverages advanced computational biology, bioinformatics, and the latest developments in biomaterials to enhance drug delivery systems, aiming to create long-lasting biodegradable nanoparticles that can effectively deliver its therapeutic peptides. Incorporated in 2011 and based in Baltimore, Maryland, AsclepiX Therapeutics is committed to addressing unmet medical needs in ophthalmology, oncology, and other diseases driven by angiogenesis and lymphangiogenesis.
C4 is building on the knowledge with synthetic chemical compounds which it calls Degronimids. These compounds can be engineered and linked together to bind with many molecular targets that today’s drugs can’t bind with, Cohen said. The key insight, he said, is that the scientific founders have discovered a way to bind with select disease-causing proteins, and flag them as cellular trash, so that the ordinary proteasome garbage disposal system can get rid of them. Selecting the precise disease-related proteins to send to the trash is obviously critical, because any drug like this could cause serious side effects if it sent healthy proteins involved in normal cell processes to the trash. C4 Therapeutics is developing a new class of targeted protein degradation (TPD) therapeutics for the treatment of a broad range of diseases. Our Degronimid™ platform incorporates highly selective small molecule binders to target disease-causing proteins and facilitate their rapid destruction and clearance from the cell through the natural ubiquitin/proteasome system (UPS). Because of this distinctive mechanism, Degronimids are capable of hitting many more targets, including those previously thought to be undruggable, while reducing the potential for drug resistance. The broad applicability of Degronimids, and our chemical biology platform designed for accelerated validation, have the potential to make an unprecedented impact across many diseases through multiple industry collaborations as well as proprietary programs. The Company has received an exclusive worldwide license to the Degronimid platform and related IP from Dana-Farber.
4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing targeted gene therapy products using evolved adeno-associated virus vectors. The company has a diverse portfolio focusing on three main therapeutic areas: ophthalmology, cardiology, and pulmonology. Key product candidates include 4D-125, currently in a Phase I/II clinical trial for treating X-linked retinitis pigmentosa; 4D-110, in a Phase I trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is developing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. The company aims to address unmet medical needs through its innovative gene therapies.
C4 is building on the knowledge with synthetic chemical compounds which it calls Degronimids. These compounds can be engineered and linked together to bind with many molecular targets that today’s drugs can’t bind with, Cohen said. The key insight, he said, is that the scientific founders have discovered a way to bind with select disease-causing proteins, and flag them as cellular trash, so that the ordinary proteasome garbage disposal system can get rid of them. Selecting the precise disease-related proteins to send to the trash is obviously critical, because any drug like this could cause serious side effects if it sent healthy proteins involved in normal cell processes to the trash. C4 Therapeutics is developing a new class of targeted protein degradation (TPD) therapeutics for the treatment of a broad range of diseases. Our Degronimid™ platform incorporates highly selective small molecule binders to target disease-causing proteins and facilitate their rapid destruction and clearance from the cell through the natural ubiquitin/proteasome system (UPS). Because of this distinctive mechanism, Degronimids are capable of hitting many more targets, including those previously thought to be undruggable, while reducing the potential for drug resistance. The broad applicability of Degronimids, and our chemical biology platform designed for accelerated validation, have the potential to make an unprecedented impact across many diseases through multiple industry collaborations as well as proprietary programs. The Company has received an exclusive worldwide license to the Degronimid platform and related IP from Dana-Farber.
Athira Pharma is a clinical-stage drug development company striving to improve human health by advancing bold and innovative therapies with the potential to restore the lives of people impacted by brain disorders. Athira is derived from the word Athir, the energy that reaches everyone. It captures its mission to develop therapies that can reach and positively impact everyone.
COMPASS Pathways is a mental health care company focused on developing innovative treatments for mental health conditions. It specializes in the formulation of COMP360, a psilocybin-based therapy currently undergoing Phase IIb clinical trials aimed at treating patients with treatment-resistant depression. The company operates across multiple sites in Europe and North America, conducting large-scale clinical trials to evaluate the efficacy and safety of its psilocybin therapy. Founded in 2016 and incorporated in 2020, COMPASS Pathways is headquartered in Altrincham, Cheshire, United Kingdom. The company's mission is to enhance patient access to evidence-based mental health solutions.
Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases.The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drugs. Dynacure is developing DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 for the treatment of Centronuclear Myopathies, with Ionis Pharmaceuticals. Dynacure is also building a complementary research portfolio targeting other orphan disorders.
Zucara Therapeutics Inc. is focused on developing innovative therapeutics aimed at preventing hypoglycemia in patients with diabetes. Founded in 2014 and based in Toronto, Canada, with an additional office in Vancouver, the company is working on a once-daily treatment that targets somatostatin type 2 receptors in the pancreas. This pre-clinical therapy aims to regulate hormone levels that are often uncontrolled in Type 1 diabetes, thereby preventing dangerous drops in blood sugar and restoring natural glucose levels. Zucara's approach offers a significant advantage over existing treatments, which typically only address hypoglycemia after it occurs, thereby enhancing diabetes management and improving patient outcomes. The therapeutic is designed to be administered via subcutaneous injection, addressing the serious health risks associated with low blood sugar episodes.
Agile Therapeutics, Inc. is a women's healthcare company dedicated to developing and commercializing prescription contraceptive products. The company’s lead product, Twirla, is a once-weekly transdermal contraceptive patch that delivers a low dose of levonorgestrel and ethinyl estradiol. Agile Therapeutics is also advancing a pipeline of related products, including various line extensions of Twirla and additional transdermal contraceptive options. These include regimens designed to extend menstrual cycles or provide shorter and lighter periods, as well as a progestin-only patch intended for women who cannot or prefer not to use estrogen. Founded in 1997 and headquartered in Princeton, New Jersey, Agile Therapeutics aims to address the unmet healthcare needs of women through innovative contraceptive solutions.
Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.
Cerebral Therapeutics, Inc. is a pharmaceutical company that develops implanted drug-device combination therapies for the treatment of neurologic diseases. The company uses implanted, refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose for the formulation of anti-epileptic drug valproic acid, called CT-010, to treat refractory epilepsy. Cerebral Therapeutics, Inc. was formerly known as Cerebral Therapeutics LLC. The company was incorporated in 2010 and is based in Aurora, Colorado.
Quellis Biosciences, Inc. is a biopharmaceutical company based in Waltham, Massachusetts, dedicated to developing innovative monoclonal antibody therapies for patients with rare diseases. Founded in 2017, the company aims to create next-generation treatments that enhance patient outcomes, particularly for conditions that are underserved by existing therapies. Quellis is supported by a team with extensive expertise in monoclonal antibody discovery and development, as well as experience in biotech investment and company formation. Collaborating with partners from the biotech incubator Viridian LLC and the accelerator Xontogeny LLC, Quellis is focused on optimizing multiple antibody leads to ensure effective therapeutic performance. Their mission is to deliver meaningful medicines that significantly improve the lives of patients facing serious health challenges.
Zentalis Pharmaceuticals is a clinical-stage biopharmaceutical company based in New York, focused on developing innovative small molecule therapeutics for cancer treatment. Established in 2014, the company aims to target fundamental biological pathways associated with various cancers. Its lead product candidate, ZN-c5, is an oral selective estrogen receptor degrader currently undergoing Phase I/II clinical trials for advanced or metastatic breast cancer. Additionally, Zentalis is advancing several other candidates: ZN-c3, a WEE1 inhibitor in trials for advanced solid tumors; ZN-d5, a selective B-cell lymphoma 2 inhibitor for hematological cancers; and ZN-e4, an irreversible inhibitor of the mutant epidermal growth factor receptor, also in Phase I/II trials for advanced non-small cell lung cancer. Through its Integrated Discovery Engine, Zentalis seeks to discover and develop novel compounds with potentially differentiated therapeutic profiles.
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.
Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, focused on developing patient-specific immunotherapies to treat cancer. Founded in 2016, the company creates personalized vaccines and T cell therapies that harness the immune system to target and eliminate cancer cells. By identifying truncal tumor neo-antigens and other cancer-specific markers, Achilles Therapeutics aims to develop next-generation therapies that selectively attack tumors while sparing healthy tissues. Currently, the company is conducting two clinical trials: the CHIRON trial for patients with non-small-cell lung cancer and the THETIS trial for those with recurrent or metastatic melanoma.
Oncorus, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative viral immunotherapies for cancer treatment. The company's lead candidate, ONCR-177, utilizes an oncolytic herpes simplex virus type 1 platform and is currently in Phase I clinical trials aimed at treating various cancers. In addition to ONCR-177, Oncorus is advancing the ONCR-GBM program for brain cancer and exploring synthetic viral immunotherapies based on Coxsackievirus A21 and Seneca Valley Virus. The company has entered a collaboration with MSD International GmbH to evaluate the safety and tolerability of ONCR-177 in combination with Merck’s immunotherapy. Founded in 2015 and headquartered in Cambridge, Massachusetts, Oncorus is committed to advancing cancer care and has pledged to support cancer research and healthcare initiatives in developing regions through corporate philanthropy.
Landos Biopharma, Inc. is a clinical-stage biopharmaceutical company based in Blacksburg, Virginia, specializing in the development of oral therapeutics for autoimmune diseases. Founded in 2017, the company focuses on innovative treatments for conditions such as inflammatory bowel disease (IBD), Crohn's disease, and ulcerative colitis. Its lead product candidate, NX-13, is a novel, gut-selective agonist targeting the NLRX1 pathway. The company also has a diverse pipeline that includes other internally discovered compounds aimed at novel immunometabolic pathways. By developing first-in-class therapeutics, Landos Biopharma seeks to provide effective treatment options for patients suffering from autoimmune conditions.
Foamix Pharmaceuticals Inc. a pharmaceutical company, engages in development and commercialization of proprietary, innovative, and differentiated topical drugs for dermatological conditions. It offers Molecule Stabilizing Technology (MST) platform. The company was incorporated in 2014 and is based in Bridgewater, New Jersey. Foamix Pharmaceuticals Inc. operates as a subsidiary of Menlo Therapeutics Inc.
Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, dedicated to leveraging the body's natural biology to repair or reverse damage from various degenerative diseases. The company employs a unique Progenitor Cell Activation approach, utilizing combinations of small molecules to stimulate progenitor cells to regenerate functional tissue. Its lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at addressing the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is developing therapies for a variety of degenerative conditions, including multiple sclerosis and ailments affecting muscle, skin, gastrointestinal tract, and bone health. The company has also established a collaboration agreement with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics is focused on disease modification through innovative therapeutic strategies.
Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and based in San Mateo, California, the company concentrates on targeting dysregulated transcription factors and the associated transcriptional regulatory networks that contribute to cancer progression. Its lead candidate, entospletinib, is a selective inhibitor aimed at spleen tyrosine kinase for treating acute myeloid leukemia. Additionally, Kronos Bio is advancing KB-0742, an orally bioavailable inhibitor of cyclin dependent kinase 9, intended for the treatment of MYC-amplified solid tumors. The company employs advanced techniques, including high-throughput small-molecule microarrays and targeted protein degradation, to discover potent compounds that can effectively modulate challenging cancer targets.
Confo Therapeutics is a drug discovery company based in Brussels, Belgium, founded in 2015. The company specializes in developing internal drug discovery programs focused on G-protein coupled receptors (GPCRs) to address unmet medical needs. Utilizing its proprietary CONFO® technology, Confo Therapeutics stabilizes inherently unstable functional conformations of GPCRs, providing a robust starting point for drug discovery. This approach reveals previously inaccessible structural features of these receptors, facilitating the identification of novel agonists that enhance therapeutic interventions.
Athenex, Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for cancer and related conditions. Founded in 2003 and headquartered in Buffalo, New York, Athenex operates across North America and Asia, aiming to become a global leader in cancer treatment. The company has three primary segments: Oncology Innovation Platform, Global Supply Chain Platform, and Commercial Platform. Its clinical pipeline includes notable Orascovery product candidates, such as Oral Paclitaxel for metastatic breast cancer and various other oral formulations targeting different cancer types. Additionally, Athenex is developing Src Kinase inhibitors for skin cancers and immunomodulatory therapies, including T Cell Receptor Engineered T Cells and arginine deprivation therapy. Through collaborations and strategic partnerships, Athenex is engaged in extensive research and development activities to advance its therapeutic offerings and improve patient outcomes.
Poseida Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in San Diego, California, focused on developing innovative therapies for patients with significant unmet medical needs. The company is advancing a diverse pipeline that includes treatments for hematological malignancies and solid tumors, as well as liver-directed gene therapies aimed at rare diseases. Poseida is known for its proprietary gene engineering platforms, including the non-viral piggyBac DNA Modification System and the Cas-CLOVER site-specific gene editing system, which facilitate the creation of next-generation cell and gene therapeutics. Additionally, the company's portfolio encompasses CAR-T therapies for cancer and gene therapies targeting rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia. Founded in 2014, Poseida Therapeutics is committed to addressing critical health challenges through its advanced therapeutic solutions.
Fusion Pharmaceuticals Inc., a clinical-stage oncology company, focuses on developing radiopharmaceuticals as precision medicines. The company has developed Targeted Alpha Therapies platform together with its proprietary Fast-Clear linker technology to enable us to connect alpha particle emitting isotopes to antibodies and other targeting molecules in order to selectively deliver the alpha particle payloads to tumors. Its lead product candidate is FPI-1434 that is in Phase 1 clinical trials as a monotherapy in patients with solid tumors expressing insulin-like growth factor 1 receptor. The company is also conducting additional preclinical studies of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors to further assess the anti-tumor activity, and dosing schedule and pharmacodynamics of the combinations. In addition, it is progressing its earlier-stage product candidate, FPI-1966 into clinical development for the treatment of head and neck, and bladder cancers expressing fibroblast growth factor receptor. The company was founded in 2014 and is headquartered in Hamilton, Canada.
SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for underserved patient populations affected by rare diseases and cancer. Founded in 2017 and headquartered in Stamford, Connecticut, the company’s lead product candidate, nirogacestat, is an oral small molecule gamma secretase inhibitor currently in Phase III trials for treating desmoid tumors. Other significant candidates include mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for relapsed or refractory multiple myeloma. The company is also exploring additional therapies, such as BGB-3245, an investigational oral inhibitor targeting BRAF mutations, currently in Phase I trials. SpringWorks has established collaborations with several biotechnology and pharmaceutical firms to enhance its research and development efforts.
BridgeBio is a biotechnology company dedicated to the discovery, development, and delivery of medicines for genetic diseases. The company has a diverse pipeline of 20 development programs, which range from early discovery to late-stage clinical trials. Among its notable candidates are BBP-265, an oral small molecule targeting transthyretin amyloidosis currently in Phase 3 trials, and infigratinib, a selective tyrosine kinase inhibitor aimed at treating FGFR-driven cancers and achondroplasia. Additionally, BridgeBio is developing BBP-631, a preclinical gene therapy for congenital adrenal hyperplasia, and BBP-454, a program focused on small molecule inhibitors for KRAS-driven cancers. The company collaborates with several prestigious institutions, including Stanford University and Johns Hopkins University, to enhance its research and development efforts. Founded in 2015 and headquartered in Palo Alto, California, BridgeBio aims to accelerate the development of therapies for Mendelian diseases, genetic dermatology, and oncology.
Vyome Therapeutics focuses on developing innovative treatments for antibiotic-resistant acne and other pathogens through a unique pipeline of antibiotics designed to slow the development of resistance. The company's product portfolio includes VB 1953, its lead molecule targeting the significant unmet needs in the resistant acne market, along with various anti-dandruff products such as VB 001 (a leave-on scalp care treatment), VB 2421 (a hair gel), VB 3222 (a wash-off shampoo), and VB 7731 (a Ketoconazole emulsion gel). Established in 2010 and based in Princeton, New Jersey, the company incorporates advanced research and development capabilities, including formulation, analytical, and clinical divisions. Vyome's management team boasts extensive expertise in dermatology and has a proven track record of successfully launching numerous products. The firm is committed to addressing refractory skin conditions and aims to bring its innovative therapies to market effectively.
Relay Therapeutics is a clinical-stage precision medicines company based in Cambridge, Massachusetts, focused on transforming the drug discovery process, particularly in targeted oncology. The company employs an innovative allosteric drug-discovery platform that integrates computational techniques with experimental methods to analyze protein motion and interactions. This approach aids in the development of therapies for complex diseases, including cancer. Relay's product candidates include RLY-1971, an oral small molecule inhibitor targeting SHP2, currently undergoing Phase 1 trials for advanced solid tumors; RLY-4008, a selective inhibitor of FGFR2 for tumors with specific oncogenic alterations; and RLY-PI3K1047, which targets cancer-associated mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, Relay Therapeutics is dedicated to enhancing small molecule therapeutic discovery through its advanced platform and targeted treatment strategies.
Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide medicines to treat the underlying causes of severe genetic diseases. Its lead product candidate, STK-001 used to treat Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics, Inc. has a partnership with Invitae Corporation to offer genetic testing. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was founded in 2014 and is headquartered in Bedford, Massachusetts.
Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.
Allogene Therapeutics is a clinical-stage biotechnology company focused on immuno-oncology, particularly the development of genetically engineered allogeneic T cell therapies for cancer treatment. The company is advancing a pipeline of allogeneic T cell product candidates that leverage validated gene editing and proprietary cell manufacturing technologies. Key offerings include UCART19, which targets relapsed or refractory acute lymphoblastic leukemia, alongside several preclinical allogeneic CAR T therapies aimed at various malignancies and autoimmune diseases. By using T cells derived from healthy donors, Allogene Therapeutics aims to provide off-the-shelf treatment options that enhance patient eligibility and scalability. The company seeks to redefine CAR T therapy through its innovative approaches, addressing conditions like lymphoma, leukemia, and solid tumors.
Allogene Therapeutics is a clinical-stage biotechnology company focused on immuno-oncology, particularly the development of genetically engineered allogeneic T cell therapies for cancer treatment. The company is advancing a pipeline of allogeneic T cell product candidates that leverage validated gene editing and proprietary cell manufacturing technologies. Key offerings include UCART19, which targets relapsed or refractory acute lymphoblastic leukemia, alongside several preclinical allogeneic CAR T therapies aimed at various malignancies and autoimmune diseases. By using T cells derived from healthy donors, Allogene Therapeutics aims to provide off-the-shelf treatment options that enhance patient eligibility and scalability. The company seeks to redefine CAR T therapy through its innovative approaches, addressing conditions like lymphoma, leukemia, and solid tumors.
4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing targeted gene therapy products using evolved adeno-associated virus vectors. The company has a diverse portfolio focusing on three main therapeutic areas: ophthalmology, cardiology, and pulmonology. Key product candidates include 4D-125, currently in a Phase I/II clinical trial for treating X-linked retinitis pigmentosa; 4D-110, in a Phase I trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is developing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. The company aims to address unmet medical needs through its innovative gene therapies.
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.
ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, that specializes in the discovery and development of antiviral drugs targeting the respiratory syncytial virus (RSV). Founded in 2011, ReViral focuses on creating novel first-in-class compounds, including a highly potent fusion inhibitor designed to treat severe RSV infections, particularly in vulnerable populations such as neonates. The company's innovative approach aims to expand the antiviral market by providing effective treatment options for patients affected by RSV, a virus known for causing significant respiratory illness. Through its research and development efforts, ReViral seeks to address unmet medical needs in the realm of viral infections.
Alector, Inc. is a clinical stage biopharmaceutical company focused on developing therapies for neurodegenerative diseases, including Alzheimer's and frontotemporal dementia. The company's lead product, AL001, is a humanized recombinant monoclonal antibody currently in phase II clinical trials for frontotemporal dementia. Additionally, AL101 is in phase I clinical trials for various neurodegenerative diseases, while AL002 and AL003 are undergoing phase 1b trials specifically for Alzheimer's disease. Alector is leveraging advancements in antibody technology, neuroimmunology, and human genetics to create innovative therapeutics. The company maintains a collaboration with Adimab, LLC, which aids in the discovery and optimization of antibody therapeutics. Alector has a pipeline of ten programs in the research and development stage and aims to advance several candidates through pre-clinical development within the next two years. Founded in 2013, Alector is headquartered in South San Francisco, California.
Athenex, Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for cancer and related conditions. Founded in 2003 and headquartered in Buffalo, New York, Athenex operates across North America and Asia, aiming to become a global leader in cancer treatment. The company has three primary segments: Oncology Innovation Platform, Global Supply Chain Platform, and Commercial Platform. Its clinical pipeline includes notable Orascovery product candidates, such as Oral Paclitaxel for metastatic breast cancer and various other oral formulations targeting different cancer types. Additionally, Athenex is developing Src Kinase inhibitors for skin cancers and immunomodulatory therapies, including T Cell Receptor Engineered T Cells and arginine deprivation therapy. Through collaborations and strategic partnerships, Athenex is engaged in extensive research and development activities to advance its therapeutic offerings and improve patient outcomes.
Rain Therapeutics Inc. is a biotechnology company focused on developing targeted therapies for cancer patients, particularly those with unmet medical needs. Established in 2017 and based in Newark, California, the company specializes in small molecule therapeutics. Its lead product, RAIN-32, is a small molecule MDM2 inhibitor aimed at treating well-differentiated and de-differentiated liposarcoma. Additionally, Rain Therapeutics is developing tarloxotinib, a hypoxia-activated pan-HER inhibitor currently undergoing clinical trials for non-small cell lung cancer. The company emphasizes identifying cancers driven by specific oncogenic mutations and evaluates sub-populations where existing treatment options are inadequate. Through collaboration with a global network of scientific and thought leaders, Rain Therapeutics seeks to align its therapeutic innovations with the needs of these cancer populations.
MEI Pharma, Inc. (Nasdaq: MEIP) is a San Diego-based oncology company focused on the clinical development of novel therapies for cancer. The Company’s clinical development pipeline includes lead drug candidate Pracinostat, a potential best-in-class, oral histone deacetylase (HDAC) inhibitor. Pracinostat has been tested in more than 150 patients in multiple Phase I and exploratory Phase II clinical trials, including advanced hematologic malignancies such as myelodysplastic syndrome, acute myeloid leukemia and myelofibrosis. The Company expects to initiate a randomized Phase II trial of Pracinostat in combination with standard-of-care in at least one hematologic malignancy toward the middle of 2013. In addition, MEI Pharma is developing two drug candidates derived from its isoflavone-based technology platform, ME-143 and ME-344. Results from a Phase I trial of intravenous ME-143 in heavily treated patients with solid refractory tumors were presented at the American Society of Clinical Oncology Annual Meeting in June 2012. A Phase I clinical trial of intravenous ME-344 in patients with solid refractory tumors is ongoing.
Kodiak Sciences Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for retinal diseases. The company's leading candidate, KSI-301, is a vascular endothelial growth factor (VEGF)-biological agent currently undergoing Phase 1b clinical trials for the treatment of wet age-related macular degeneration and diabetic retinopathy. Additionally, Kodiak is advancing KSI-501, a bispecific anti-interleukin 6/VEGF bioconjugate aimed at addressing inflammation and abnormal blood vessel growth associated with retinal vascular diseases, which is in the preclinical stage. The company's early research pipeline also includes KSI-601, a triplet inhibitor targeting dry AMD. Founded in 2009 and headquartered in Palo Alto, California, Kodiak Sciences was previously known as Oligasis, LLC before its name change in 2015.
Eidos Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Francisco, California, focused on developing innovative treatments for transthyretin amyloidosis (ATTR), a disease caused by the misfolding of the transthyretin protein. The company is advancing its lead drug candidate, AG10, which is currently in phase 3 clinical trials. AG10 is an orally administered small molecule designed to stabilize the tetrameric form of transthyretin, effectively preventing the molecular processes that lead to the development of ATTR. Founded in 2013, Eidos Therapeutics operates as a subsidiary of BridgeBio Pharma, Inc., which is dedicated to creating genetically targeted therapies to enhance patient outcomes.
IDEAYA Biosciences is an oncology-focused biotechnology company based in South San Francisco, California, dedicated to the discovery and development of targeted therapeutics for genetically defined patient populations. The company specializes in precision medicine using molecular diagnostics and synthetic lethality approaches to address cancer treatment. Its lead product candidate, IDE196, is a protein kinase C inhibitor currently undergoing Phase 1/2 clinical trials for cancers associated with GNAQ or GNA11 gene mutations. IDEAYA's preclinical pipeline includes programs targeting various mechanisms related to DNA damage and repair, such as MAT2A for tumors with MTAP deletions, Pol-theta for homologous recombination deficiency, and PARG for BRCA2 mutation context. The company collaborates with Cancer Research UK and the University of Manchester to develop small molecule inhibitors of Poly (ADP-ribose) glycohydrolase, while also engaging in partnerships for clinical trials with Pfizer and GlaxoSmithKline. Founded in 2015, IDEAYA aims to advance innovative therapies for enhancing immunotherapy responses and improving patient outcomes in oncology.
Crinetics Pharmaceuticals discovers and develops novel therapeutics targeting peptide hormone receptors for the treatment of endocrine-related diseases and cancers. Their programs are derived from internal discovery efforts and use objective hormonal biomarker endpoints for preclinical and clinical studies to reduce both the risk and cost to achieve meaningful clinical data. Crinetics is seeking motivated partners to help advance Their programs to become commercially successful products.
Molecular Templates, Inc. is a clinical-stage biopharmaceutical company based in Austin, Texas, dedicated to discovering and developing biologic therapeutics for cancer treatment. The company utilizes a proprietary platform known as engineered toxin bodies to create targeted therapies. Its lead candidate, MT-3724, is currently undergoing Phase II clinical trials for relapsed and refractory diffuse large B-cell lymphoma and non-Hodgkin’s lymphoma. In addition, the company is developing other candidates, including MT-4019, which targets CD38, and has collaborations with Takeda Pharmaceutical Company and Vertex Pharmaceuticals. These partnerships aim to enhance treatment options for conditions like multiple myeloma and improve outcomes in hematopoietic stem cell transplants. Molecular Templates continues to expand its pipeline with additional compounds targeting various cancers.
Partner Therapeutics, Inc. is an oncology company based in Lexington, Massachusetts, focused on developing and commercializing approved and late-stage cancer therapies. The company aims to improve treatment outcomes and reduce healthcare costs by providing a range of cancer treatments, from primary therapies to supportive care. One of its key products is Leukine, which promotes the growth of white blood cells, enhancing patients' immune responses during cancer treatment. Formerly known as Partners Oncology, Inc., the company rebranded in November 2017. Partner Therapeutics serves as a vital link between researchers and healthcare professionals, facilitating the advancement of cancer therapies.
Vitruvias Therapeutics is a start-up generic pharmaceutical company based in Montgomery, Alabama, founded in 2013. The company specializes in developing niche topical generic products that are available only by prescription. Focusing on complex active pharmaceutical ingredients (APIs) and challenging finished dosage forms, Vitruvias Therapeutics aims to address the difficulties associated with manufacturing these products. By leveraging strategic partnerships, the company seeks to overcome manufacturing complexities and raw material sourcing challenges, thereby enhancing the availability of essential generic medications. The name Vitruvias is inspired by Leonardo da Vinci’s "Vitruvian Man," symbolizing the ideal proportions of the human figure, reflecting the company's commitment to precision in pharmaceutical development.
Landos Biopharma, Inc. is a clinical-stage biopharmaceutical company based in Blacksburg, Virginia, specializing in the development of oral therapeutics for autoimmune diseases. Founded in 2017, the company focuses on innovative treatments for conditions such as inflammatory bowel disease (IBD), Crohn's disease, and ulcerative colitis. Its lead product candidate, NX-13, is a novel, gut-selective agonist targeting the NLRX1 pathway. The company also has a diverse pipeline that includes other internally discovered compounds aimed at novel immunometabolic pathways. By developing first-in-class therapeutics, Landos Biopharma seeks to provide effective treatment options for patients suffering from autoimmune conditions.
BridgeBio is a biotechnology company dedicated to the discovery, development, and delivery of medicines for genetic diseases. The company has a diverse pipeline of 20 development programs, which range from early discovery to late-stage clinical trials. Among its notable candidates are BBP-265, an oral small molecule targeting transthyretin amyloidosis currently in Phase 3 trials, and infigratinib, a selective tyrosine kinase inhibitor aimed at treating FGFR-driven cancers and achondroplasia. Additionally, BridgeBio is developing BBP-631, a preclinical gene therapy for congenital adrenal hyperplasia, and BBP-454, a program focused on small molecule inhibitors for KRAS-driven cancers. The company collaborates with several prestigious institutions, including Stanford University and Johns Hopkins University, to enhance its research and development efforts. Founded in 2015 and headquartered in Palo Alto, California, BridgeBio aims to accelerate the development of therapies for Mendelian diseases, genetic dermatology, and oncology.
Molecular Templates, Inc. is a clinical-stage biopharmaceutical company based in Austin, Texas, dedicated to discovering and developing biologic therapeutics for cancer treatment. The company utilizes a proprietary platform known as engineered toxin bodies to create targeted therapies. Its lead candidate, MT-3724, is currently undergoing Phase II clinical trials for relapsed and refractory diffuse large B-cell lymphoma and non-Hodgkin’s lymphoma. In addition, the company is developing other candidates, including MT-4019, which targets CD38, and has collaborations with Takeda Pharmaceutical Company and Vertex Pharmaceuticals. These partnerships aim to enhance treatment options for conditions like multiple myeloma and improve outcomes in hematopoietic stem cell transplants. Molecular Templates continues to expand its pipeline with additional compounds targeting various cancers.
Dova Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in Durham, North Carolina, that specializes in acquiring, developing, and commercializing drug candidates for thrombocytopenia, a condition characterized by low platelet counts. The company's primary product candidate, DOPTELET, is approved for treating thrombocytopenia in adult patients with chronic liver disease who are preparing for a medical procedure. Additionally, Dova is developing DOPTELET for other indications, including chronic immune thrombocytopenia and chemotherapy-induced thrombocytopenia. Founded in 2016, Dova Pharmaceuticals operates as a subsidiary of Swedish Orphan Biovitrum AB, focusing on addressing rare diseases with significant unmet medical needs.
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.
Singulex, Inc., a biotechnology company, develops and commercializes direct molecular detection technology solutions. Its solutions enable life science researchers and clinicians to understand and manage disease. The company develops biomarker diagnostic systems that can detect and quantify normal and abnormal protein biomarkers. It is conducting pilot studies to validate Erenna BioAssay System, a digital molecule detection platform, which detects and quantifies molecule biomarkers in clinical samples. The company also offers various custom assay services, such as pilot study services, assay prototyping, assay characterization, and assay validation. The company was formerly known as BioProfile Corporation and changed its name to Singulex, Inc in September 2003. Singulex is based in Hayward, California
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
VBI Vaccines Inc. is a biopharmaceutical company focused on developing vaccines for infectious diseases and immuno-oncology, with operations in Israel, the United States, and internationally. The company offers Sci-B-Vac, a prophylactic hepatitis B vaccine suitable for various age groups, and is advancing VBI-2601, an immunotherapeutic candidate aimed at achieving a functional cure for chronic hepatitis B. Utilizing its enveloped virus-like particle (eVLP) platform technology, VBI develops vaccines that closely mimic target viruses to elicit strong immune responses. Key candidates in its eVLP program include VBI-1901, a glioblastoma vaccine in Phase I/IIa clinical trials, and VBI-1501, a cytomegalovirus vaccine that has completed Phase I trials. VBI Vaccines also engages in collaboration and licensing agreements with various pharmaceutical and biotechnology entities, including partnerships for the commercialization of its hepatitis B vaccine and the development of a pan-coronavirus vaccine candidate. Headquartered in Cambridge, Massachusetts, the company also has research facilities in Ottawa, Canada.
VBI Vaccines Inc. is a biopharmaceutical company focused on developing vaccines for infectious diseases and immuno-oncology, with operations in Israel, the United States, and internationally. The company offers Sci-B-Vac, a prophylactic hepatitis B vaccine suitable for various age groups, and is advancing VBI-2601, an immunotherapeutic candidate aimed at achieving a functional cure for chronic hepatitis B. Utilizing its enveloped virus-like particle (eVLP) platform technology, VBI develops vaccines that closely mimic target viruses to elicit strong immune responses. Key candidates in its eVLP program include VBI-1901, a glioblastoma vaccine in Phase I/IIa clinical trials, and VBI-1501, a cytomegalovirus vaccine that has completed Phase I trials. VBI Vaccines also engages in collaboration and licensing agreements with various pharmaceutical and biotechnology entities, including partnerships for the commercialization of its hepatitis B vaccine and the development of a pan-coronavirus vaccine candidate. Headquartered in Cambridge, Massachusetts, the company also has research facilities in Ottawa, Canada.
True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.
Vyome Therapeutics focuses on developing innovative treatments for antibiotic-resistant acne and other pathogens through a unique pipeline of antibiotics designed to slow the development of resistance. The company's product portfolio includes VB 1953, its lead molecule targeting the significant unmet needs in the resistant acne market, along with various anti-dandruff products such as VB 001 (a leave-on scalp care treatment), VB 2421 (a hair gel), VB 3222 (a wash-off shampoo), and VB 7731 (a Ketoconazole emulsion gel). Established in 2010 and based in Princeton, New Jersey, the company incorporates advanced research and development capabilities, including formulation, analytical, and clinical divisions. Vyome's management team boasts extensive expertise in dermatology and has a proven track record of successfully launching numerous products. The firm is committed to addressing refractory skin conditions and aims to bring its innovative therapies to market effectively.
Aquestive Therapeutics, Inc. is a specialty pharmaceutical company focused on developing and commercializing innovative medicines to meet unmet medical needs both in the United States and internationally. The company offers several marketed products, including Sympazan, an oral soluble film for treating Lennox-Gastaut syndrome; Suboxone, a sublingual film for opioid dependence; and Zuplenz, an oral soluble film for nausea and vomiting related to chemotherapy and post-operative recovery. Additionally, Aquestive's proprietary product candidates include Libervant, a buccal film for epileptic seizures, and Exservan, an oral film for Amyotrophic Lateral Sclerosis. The company's pipeline also features AQST-108, a sublingual film for anaphylaxis, AQST-305 for acromegaly, and APL-130277 for Parkinson's disease. Established in 2004 and headquartered in Warren, New Jersey, Aquestive Therapeutics leverages its proprietary technologies to address complex healthcare challenges and partners with other pharmaceutical companies to enhance drug delivery methods.
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