Deerfield Management

Vicinitas Therapeutics is a biotech startup that specializes in developing medicines that impact patient lives. Its vision is to use its proprietary Deubiquitinase Targeting Chimera (DUBTAC) platform to restore the levels of aberrantly degraded proteins that cause disease to confer therapeutic benefit.

InSilico Medicine, Inc. is an artificial intelligence company focused on drug discovery, biomarker development, and aging research. Founded in 2014 and headquartered in Baltimore, Maryland, the company employs advanced AI technologies, including deep learning and generative adversarial networks, to create novel molecular structures for diseases with known and unknown targets. Its comprehensive platform covers all stages of drug development, from discovery to clinical trials. Key initiatives include Pharma.AI, which offers machine learning services to various industries, and Young.AI, which predicts biological age. InSilico Medicine also engages in internal drug discovery programs targeting conditions such as cancer, Parkinson's Disease, and age-related disorders. Additionally, the company has partnered with Life Extension to develop nutraceutical products using its bioinformatics and deep learning techniques. Through its innovative solutions, InSilico Medicine aims to enhance personalized healthcare and mitigate the effects of aging.

Operator of a biotechnology company intended for pioneering the development of genetic medicines for treating intractable diseases. The company specializes in developing genetic drugs by simultaneously engineering (Adeno-associated viruses) AAV capsids that bind to the human Transferrin Receptor 1 (TfR1) to enable intravenous delivery of therapeutics that use receptor-mediated transcytosis across the blood brain barrier allowing broad penetration of the central nervous system, and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity and manufacturability, thereby enabling patients to treat debilitating diseases with limited options.

Vibliome Therapeutics, Inc. is a biotechnology company based in Bozeman, Montana, that focuses on researching and developing innovative therapeutics. The company is pioneering a non-hormonal birth control pill for men, utilizing the homeodomain-interacting protein kinase 4 (HIPK4) as a key component of its approach. Additionally, Vibliome develops cancer treatments through a specialized library of kinase inhibitors that target specific pathways. With a systematic methodology for creating small molecule kinase inhibitors, Vibliome aims to achieve unique and, in some cases, extraordinary selectivity among the over 500 kinases in the human genome. This versatility in their chemistry platform presents numerous opportunities for advancements in both reproductive health and oncology. Vibliome was incorporated in 2017.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, specializing in the development of specific and selective antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create novel monoclonal antibody and cell-based therapeutics. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate aimed at treating soft tissue and bone sarcoma, non-small cell lung cancer, and other tumors. BioAtla's pipeline also includes BA3021, targeting non-small cell lung cancer and melanoma, and BA3071, which addresses various cancers such as renal cell carcinoma and bladder cancer. The company's innovative approach enhances drug selectivity for cancer tissues, potentially improving safety and expanding treatment options for previously untreatable cancers. BioAtla holds over 150 issued patents and patent applications, underscoring its commitment to developing safer and more effective cancer therapies.

ARS Pharmaceuticals, Inc., founded in 2015 and based in San Diego, California, is a biopharmaceutical company specializing in the development of innovative treatments for severe allergic reactions, including anaphylaxis. The company's flagship product, neffy, is a novel intranasal epinephrine spray that utilizes a proprietary absorption technology known as Intravail. This unique formulation allows for effective, injection-like absorption of epinephrine at a low dose, making it a convenient and reliable option for patients at risk of life-threatening allergic reactions to foods, medications, and insect stings. With its focus on enhancing accessibility and ease of use, ARS Pharmaceuticals aims to improve emergency responses for individuals experiencing severe allergic reactions.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the delivery of nucleic acid therapeutics, particularly antisense oligonucleotides. Established in 2018, PepGen aims to enhance the clinical efficacy of these therapeutics through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative technology employs engineered peptides designed for optimized tissue penetration, cellular uptake, and nuclear delivery, enabling effective transport of oligonucleotides to various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. By leveraging its drug delivery capabilities, PepGen seeks to transform the treatment landscape for severe neuromuscular and neurological diseases.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company specializes in discovering and developing medicines that target cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational techniques and machine learning to identify new binding pockets on proteins, thereby facilitating the discovery and development of small-molecule drugs. By enhancing the drug development process, Frontier Medicines aims to deliver breakthrough therapies that can significantly change the treatment landscape for debilitating diseases.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.

ADC Therapeutics is a clinical-stage oncology drug discovery and development company focused on advancing highly potent and targeted antibody drug conjugates (ADCs) for the treatment of hematological cancers and solid tumors. Our ADCs are developed using the latest-generation pyrrolobenzodiazepine (PBD) dimer technology providing a superior therapeutic index compared to first- generation PBD ADCs in preclinical studies. PBD dimers are very potent toxins that actively kill cancer cells and have a differentiated mechanism of action than warheads commonly used in other ADCs.

Adaptive Phage Therapeutics, Inc. specializes in developing therapeutic solutions to combat the increasing threat of multi-drug resistant (MDR) bacteria. Founded in 2016 and based in Gaithersburg, Maryland, the company utilizes a unique resource known as PhageBank, which is an extensive and dynamic collection of bacteriophages—viruses that specifically target and attack harmful bacteria. Through a rapid system that matches these phages to individual patient bacterial infections, Adaptive Phage Therapeutics aims to enhance treatment options for patients suffering from MDR infections. Their innovative approach enables medical researchers to create tailored therapies that effectively detect and eliminate resistant bacterial strains.

GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders. The company is focused on developing novel and proprietary Mebufotenin 5-Methoxy-N, N-Dimethyltryptamine, or 5-MeO-DMT, therapies for the treatment of patients with Treatment-Resistant Depression, or TRD.

Xeris Biopharma is a specialty pharmaceutical company focused on developing and commercializing ready-to-use injectable and infusible drug formulations. Utilizing its proprietary XeriSol and XeriJect technology platforms, the company delivers highly concentrated, liquid-stable formulations of peptides, small molecules, and proteins through various delivery methods, including syringes and auto-injectors. Xeris Biopharma's lead product is Gvoke, a ready-to-use glucagon for treating severe hypoglycemia, while its other products include Keveyis, the first FDA-approved treatment for primary periodic paralysis, and Recorlev, approved for endogenous hypercortisolemia in Cushing's Syndrome. The company is also advancing several product candidates in clinical trials, targeting conditions in endocrinology, neurology, and gastroenterology. Founded in 2005 and headquartered in Chicago, Illinois, Xeris Biopharma aims to improve patient care by providing easier-to-use therapies that enhance treatment outcomes.

Terns Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing oral, small-molecule drugs for the treatment of cancer and liver diseases, particularly non-alcoholic steatohepatitis (NASH). Founded in 2017 and headquartered in Foster City, California, with additional offices in Shanghai, the company leverages its expertise in disease biology and medicinal chemistry to advance a pipeline of innovative therapeutic candidates. Key products include TERN-101, a non-bile acid farnesoid X receptor agonist, and TERN-201, an inhibitor of semicarbazide-sensitive amine oxidase. Terns Pharmaceuticals aims to address significant unmet medical needs both in China and globally through its efficient drug discovery model and extensive clinical development capabilities.

FogPharma is a healthcare company founded in 2015 and headquartered in Gloucester, Massachusetts. The firm is dedicated to the pharmaceutical sector, with a primary focus on developing innovative treatments for cancer. It operates with the support of a diverse group of private and institutional investors, fostering collaboration among its founders, employees, and advisors. FogPharma aims to create a new class of medicines designed to extend and enhance the lives of cancer patients. The company's commitment to its mission drives its efforts to pioneer effective solutions in cancer treatment.

Neomorph, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing protein degradation solutions, specifically through the use of molecular glue degraders. This innovative approach repurposes the cellular machinery to eliminate proteins associated with various diseases, particularly targeting those considered "undruggable." Neomorph's mission is to address critical challenges in human health by discovering and creating new medicines aimed at these difficult targets, thereby advancing its projects through clinical development.

Cystetic Medicines develops a treatment that potentially could help people with cystic fibrosis (CF), regardless of genetic mutation.

Bridge Medicines LLC offers drug discovery services. It focuses on developing technologies in academic institutions from human proof-of-concept to clinical development. The company develops inhibitors of ENL-YEATS for the treatment of acute leukemias, such as acute myelogenous leukemia and a series of orally active small molecule inhibitors of activated factor XII for the treatment of hereditary angioedema and other inflammatory disorders. Bridge Medicines LLC was founded in 2016 and is based in New York, New York.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing targeted therapies for women's cancers, particularly estrogen receptor (ER)-positive breast cancer. The company's lead candidate, OP-1250, is an ER antagonist and selective ER degrader currently undergoing Phase 1/2 clinical trials for the treatment of metastatic or locally advanced, ER-positive, and human epidermal growth factor receptor 2-negative breast cancer. Olema's research focuses on utilizing its expertise in endocrine-driven cancers and the molecular mechanisms of the ER to create more effective treatments that aim to improve patient outcomes. In addition to OP-1250, Olema is advancing another drug candidate, OP-3136, further enriching its pipeline. Established in 2006 and headquartered in San Francisco, California, Olema is committed to transforming the standard of care for women facing these challenging diagnoses.

ARTMS Products Inc. is a Vancouver-based company that specializes in manufacturing diagnostic imaging systems for the production and separation of medical isotopes. Founded in 2013, the company has developed the QUANTM Irradiation System, which facilitates decentralized and local production of critical isotopes such as gallium-68, zirconium-89, technetium-99m, and copper-64. ARTMS's primary offering, QUANTM99, is a system that combines hardware and consumables for the effective production of technetium-99m using medical cyclotrons. This innovative approach aims to enhance the accessibility and cost-effectiveness of medical isotopes, improving diagnostic imaging services.

ADC Therapeutics is a clinical-stage oncology drug discovery and development company focused on advancing highly potent and targeted antibody drug conjugates (ADCs) for the treatment of hematological cancers and solid tumors. Our ADCs are developed using the latest-generation pyrrolobenzodiazepine (PBD) dimer technology providing a superior therapeutic index compared to first- generation PBD ADCs in preclinical studies. PBD dimers are very potent toxins that actively kill cancer cells and have a differentiated mechanism of action than warheads commonly used in other ADCs.

Neomorph, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing protein degradation solutions, specifically through the use of molecular glue degraders. This innovative approach repurposes the cellular machinery to eliminate proteins associated with various diseases, particularly targeting those considered "undruggable." Neomorph's mission is to address critical challenges in human health by discovering and creating new medicines aimed at these difficult targets, thereby advancing its projects through clinical development.

Four Points Innovation supports Duke R&D projects throughout preclinical stages of drug discovery and development. Beginning approximately in March 2020, Duke researchers can submit proposals on projects for consideration by a Four Points Innovation committee comprised of scientific leadership representing Duke and Deerfield.

Civetta Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on developing small molecule therapeutics that target beta-propeller proteins for the treatment of cancers and other diseases. Founded in 2018, Civetta leverages its expertise in biochemistry, biology, and medicinal chemistry to create innovative therapies aimed at modulating the diverse functional roles of propeller proteins, which are critical in various disease pathways, including cancer and neurodegeneration. The company aims to build a robust portfolio of therapeutics that provide both immediate treatment options and long-term value in the biopharmaceutical landscape. Through its targeted approach, Civetta seeks to enhance the availability of effective treatment options for healthcare providers and patients facing serious health challenges.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, specializing in the development of small molecule therapies for severe musculoskeletal diseases. Founded in 2017, the company leverages its expertise in skeletal muscle physiology to create innovative treatments for genetically defined muscle disorders, including Duchenne, Becker, and limb girdle muscular dystrophies. Edgewise Therapeutics employs a precision medicine platform to identify and target key muscle proteins and modulators, aiming to address the underlying causes of these conditions. Additionally, the company has established a state-of-the-art research facility to enhance its capabilities in understanding muscle adaptation and injury related to disease.

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.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company specializes in discovering and developing medicines that target cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational techniques and machine learning to identify new binding pockets on proteins, thereby facilitating the discovery and development of small-molecule drugs. By enhancing the drug development process, Frontier Medicines aims to deliver breakthrough therapies that can significantly change the treatment landscape for debilitating diseases.

Aerie Pharmaceuticals is an ophthalmic pharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapies for various eye diseases, including glaucoma, dry eye, and retinal conditions. The company's key products include Rhopressa, a once-daily eye drop that lowers elevated intraocular pressure in glaucoma patients, and Rocklatan, a fixed-dose combination of Rhopressa and latanoprost for treating open-angle glaucoma and ocular hypertension. Additionally, Aerie is advancing its clinical-stage product AVX-012 for dry eye and developing sustained-release implants AR-1105 and AR-13503 for retinal diseases. Established in 2005, Aerie is headquartered in Durham, North Carolina, with research facilities in Research Triangle Park. The company has also formed a collaborative research and development agreement with DSM.

Schrödinger is a healthcare software company that specializes in computational drug design, serving the biopharmaceutical and biotechnology sectors. Founded in 1990, the company operates through two main segments: Software and Drug Discovery. Its software solutions facilitate drug discovery processes for pharmaceutical and industrial clients, as well as academic and governmental research institutions. In addition to software sales, Schrödinger engages in drug discovery through its own preclinical and clinical programs, often collaborating with other organizations. The company is committed to advancing its computational platform by investing in scientific research and talent, and it has established partnerships and collaborations across more than 70 countries. With a workforce of over 400 employees, Schrödinger continues to influence the life sciences and materials science industries through innovative approaches to drug discovery.

SHINE Medical Technologies, LLC is a manufacturer and supplier of medical tracers and cancer treatment elements, specializing in isotopes used for diagnostic and therapeutic purposes. The company produces molybdenum-99, which is essential for imaging and diagnosing conditions such as cancer and heart disease. Additionally, SHINE offers Iodine-131 for the treatment of thyroid cancer and related conditions, as well as Iodine-125, which is utilized in brachytherapy for various cancers. The company also provides Xenon-133, an inhalable gas that assists in evaluating lung function and blood flow in the brain. Founded in 2010 and headquartered in Janesville, Wisconsin, SHINE serves both domestic and international markets with its innovative medical solutions.

Tris Pharma, Inc. is a specialty pharmaceutical company based in Monmouth Junction, New Jersey, that focuses on the research, development, manufacturing, and marketing of over-the-counter, prescription branded, and specialty generic products in the United States. The company specializes in extended release formulations tailored for children, seniors, and adults who have difficulty swallowing pills. Tris Pharma has developed several proprietary technologies, including OralXR+, which delivers medication over time using small particles, and Nobuse, an abuse-resistant technology. The product portfolio includes immediate and extended release formulations, lipophilic drugs, and Tuzistra XR, an extended-release oral suspension for cough relief. The company offers a diverse range of dosage forms, including suspensions, chewable tablets, and film strips, and has a pipeline of products targeting cough and cold, pain management, and neurological disorders. Established in 2000, Tris Pharma holds over 30 U.S. patents and has successfully commercialized numerous products in the market through both internal resources and licensing agreements.

Trinity Biotech is engaged in the development, manufacturing, and marketing of medical diagnostic products for clinical laboratories and point-of-care settings. The company offers a wide range of diagnostic tests and instrumentation that detect infectious diseases, sexually transmitted infections, autoimmune disorders, and conditions related to diabetes and liver health. With a portfolio of over 400 products, it employs various diagnostic formats, including immunofluorescence assays and enzyme-linked immunosorbent assays. Additionally, Trinity Biotech supplies raw materials to the life sciences and research sectors. Its products are marketed under several brand names, including Recombigen and Unigold, and are sold through a direct sales force and a network of independent distributors. The company serves a diverse clientele, including public health facilities, hospitals, and clinical laboratories. Founded in 1992 and headquartered in Bray, Ireland, Trinity Biotech has established a strong presence in the diagnostic market across regions including the Americas, Europe, Africa, and Asia.

Pinnacle Hill is a collaboration between the University of North Carolina at Chapel Hill and Deerfield Management, focused on discovering new medicines to address significant unmet medical needs. The company develops therapeutic medical drugs targeting diseases such as multiple myeloma and Angelman syndrome. Its innovative approach aims to replace or reactivate the faulty UBE3A gene in affected nerve cells, providing healthcare professionals with new treatment options for patients facing life-threatening conditions. Deerfield Management contributes up to $65 million in targeted funding and offers drug development expertise to enhance the promising therapeutic research at UNC-Chapel Hill.

ARS Pharmaceuticals, Inc., founded in 2015 and based in San Diego, California, is a biopharmaceutical company specializing in the development of innovative treatments for severe allergic reactions, including anaphylaxis. The company's flagship product, neffy, is a novel intranasal epinephrine spray that utilizes a proprietary absorption technology known as Intravail. This unique formulation allows for effective, injection-like absorption of epinephrine at a low dose, making it a convenient and reliable option for patients at risk of life-threatening allergic reactions to foods, medications, and insect stings. With its focus on enhancing accessibility and ease of use, ARS Pharmaceuticals aims to improve emergency responses for individuals experiencing severe allergic reactions.

Poseidon Innovation specializes in the development of advanced therapeutics aimed at curing diseases, particularly for patients in risky early-stage conditions. Operating primarily in New York, the company focuses on streamlining the drug development cycle, allowing for quicker access to treatments. By addressing the complexities and uncertainties associated with early-stage therapeutic processes, Poseidon Innovation seeks to enhance the efficiency of bringing new medical solutions to patients in need.

Appello Pharmaceuticals, Inc. is a preclinical-stage company based in Nashville, Tennessee, specializing in the development of innovative positive allosteric modulators of mGlu4. These compounds are being investigated for their potential in treating Parkinson’s disease. The company aims to advance its research to provide new therapeutic options for this neurological disorder.

Ablexis, LLC, a biotechnology company, provides a platform for human antibody drug discovery and development. The company offers AlivaMab Mouse, a transgenic mouse platform for the discovery and development of therapeutic antibodies to treat human diseases. It commercializes its platform through creative partnering strategies to biotechnology and pharmaceutical companies. The company was founded in 2009 and is based in San Francisco, California.

Cureatr Inc. offers a mobile care coordination solution aimed at improving medication management for healthcare providers. By integrating HIPAA-secure text messaging with workflow navigation tools, Cureatr facilitates efficient communication among medical teams. The platform is accessible on Web, Android, and iPhone devices, ensuring real-time, universal access to accurate medication data for over 265 million patients. The company addresses the significant challenges of suboptimal medication management in the United States, which leads to substantial healthcare costs and mortality. With a focus on high-quality interventions provided by board-certified telepharmacists, Cureatr aims to enhance patient safety and reduce hospitalizations. Established in 2011, the company is headquartered in New York, New York.

Sollis Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Columbus, Ohio, focused on developing non-steroid, non-opioid analgesics and anti-inflammatory medications. Its primary product, Radicular Pain RelIEf Via Epidural injection of Clonidine Micropellets (RePRIEVE-CM), is designed for targeted local delivery to address lumbosacral radiculopathy, sciatica, and other neuropathic pain syndromes. Founded in 2017, Sollis Therapeutics aims to provide effective pain management solutions while avoiding the complications associated with traditional opioid treatments. Through its innovative approach, the company seeks to improve therapeutic options for healthcare providers managing pain conditions that affect a significant number of individuals.

Arvinas is a pharmaceutical company that focuses on developing novel therapeutics to treat a variety of diseases including cancer, pro-inflammatory, autoimmune and rare diseases. Its proprietary approach is the targeting of disease-causing proteins for degradation thus eliminating them from the body. The company was founded in 2013 and is headquartered in Connecticut, United States.

Operator of a novel therapeutics company intended to accelerate early-stage therapeutics based on novel biology. The company focuses on identifying and establishing development plans for the advancement of novel drugs to market, enabling scientists to execute novel and potentially transformative research projects.

Dracen Pharmaceuticals, Inc. is a biotechnology company based in Baltimore, Maryland, founded in 2016. The company specializes in the discovery and development of anticancer therapies, focusing on novel glutamine antagonists. These innovative therapies aim to directly reduce tumor size and improve the tumor microenvironment, thereby enhancing the effectiveness of immuno-oncology treatments. Dracen's approach seeks to increase the number of patients who respond to cancer therapies and extend survival rates, particularly in cases where traditional immuno-oncology methods have been less effective. Through its commitment to advancing cancer treatment, Dracen Pharmaceuticals is dedicated to improving outcomes for cancer patients.

Kiniksa Pharmaceuticals is a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing, and commercializing therapeutic medicines for patients suffering from debilitating diseases with a significant unmet medical need. It has a pipeline of product candidates across various stages of development, currently focused on autoinflammatory and autoimmune conditions. It has three clinical-stage product candidates, one of which is anticipated to commence a Phase 3 clinical trial in 2018. They follow a disciplined and methodical approach to selectively identify and acquire product candidates with strong biologic rationales or validated mechanisms of action. The company's portfolio of product candidates offers multiple development opportunities. By modulating different parts of the innate and adaptive immune system, these product candidates together have the potential to provide a variety of mechanisms to address multiple devastating diseases.

Melinta Therapeutics, Inc. is a commercial-stage pharmaceutical company specializing in the discovery, development, and commercialization of anti-infective therapies targeting bacterial infections. The company offers several products, including Baxdela, Vabomere, Orbactiv, and Minocin, which address various acute bacterial skin infections and gram-negative infections. Recognizing the urgent need for new antibiotics to combat drug-resistant infections, Melinta is advancing delafloxacin, currently in Phase 3 development for treating acute bacterial skin and skin structure infections. The company also focuses on developing innovative antibiotics aimed at overcoming resistant pathogens associated with serious hospital infections. Established in 2000, Melinta Therapeutics is headquartered in Morristown, New Jersey, and operates with support from strategic partners in the healthcare sector.

Homology Medicines, Inc. is a genetic medicines company based in Bedford, Massachusetts, focused on improving the lives of patients with rare genetic diseases. The company employs a proprietary platform that utilizes human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies in vivo, either through gene therapy or nuclease-free gene editing. This innovative approach allows for targeted treatment of various disease-relevant tissues, including the liver and central nervous system, through a single injection. Homology's lead product candidate, HMI-102, is currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, while HMI-103 targets pediatric PKU patients, and HMI-202 is being developed for metachromatic leukodystrophy. Founded in 2015, Homology Medicines aims to address significant unmet medical needs by targeting the underlying causes of genetic disorders.

Proteon Therapeutics, Inc is a biopharmaceutical company developing pharmaceuticals to address the medical needs of patients with renal and vascular diseases. The company leverages a unique understanding of tissue remodeling to develop a pipeline of proprietary therapeutics. Proteon Therapeutics’ first drug candidate (PRT-201) is in development for the improvement of blood flow following vascular surgery procedures. The company’s initial clinical focus is vascular access for hemodialysis and peripheral arterial disease (PAD).

Xeris Biopharma is a specialty pharmaceutical company focused on developing and commercializing ready-to-use injectable and infusible drug formulations. Utilizing its proprietary XeriSol and XeriJect technology platforms, the company delivers highly concentrated, liquid-stable formulations of peptides, small molecules, and proteins through various delivery methods, including syringes and auto-injectors. Xeris Biopharma's lead product is Gvoke, a ready-to-use glucagon for treating severe hypoglycemia, while its other products include Keveyis, the first FDA-approved treatment for primary periodic paralysis, and Recorlev, approved for endogenous hypercortisolemia in Cushing's Syndrome. The company is also advancing several product candidates in clinical trials, targeting conditions in endocrinology, neurology, and gastroenterology. Founded in 2005 and headquartered in Chicago, Illinois, Xeris Biopharma aims to improve patient care by providing easier-to-use therapies that enhance treatment outcomes.

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.

Chondrial Therapeutics LLC is a clinical-stage biotechnology company based in Indianapolis, Indiana, founded in 2013. The company specializes in discovering and developing orphan drugs aimed at treating rare mitochondrial diseases, with a particular focus on Friedreich’s Ataxia. Through its commitment to scientific research, Chondrial Therapeutics seeks to advance innovative therapies for patients affected by these challenging conditions.

Bridge Medicines LLC offers drug discovery services. It focuses on developing technologies in academic institutions from human proof-of-concept to clinical development. The company develops inhibitors of ENL-YEATS for the treatment of acute leukemias, such as acute myelogenous leukemia and a series of orally active small molecule inhibitors of activated factor XII for the treatment of hereditary angioedema and other inflammatory disorders. Bridge Medicines LLC was founded in 2016 and is based in New York, New York.

Ardelyx, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for cardiorenal diseases. Headquartered in Fremont, California, Ardelyx's lead product candidate, tenapanor, has successfully completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and is also in Phase 3 trials for managing hyperphosphatemia in end-stage renal disease patients undergoing dialysis. The company is advancing RDX013, a small molecule potassium secretagogue, aimed at treating hyperkalemia. Ardelyx is also developing RDX5791, an oral NHE3 sodium transport inhibitor targeting constipation-predominant irritable bowel syndrome and excess dietary sodium absorption, currently in Phase 2 trials. Additionally, RDX002 and RDX009, which inhibit phosphate absorption and target type 2 diabetes, respectively, are in preclinical development. Ardelyx emphasizes non-systemic oral therapeutics that minimize side effects while effectively addressing metabolic disorders. Since its founding in 2007, the company has secured significant funding to support its research and development efforts.

Blade Therapeutics is a discovery stage drug development company. Their mission is to create antifibrotic treatment strategies that address a wide variety of congenital and acquired diseases of fibrosis. Their leadership team has extensive experience at developing drugs that treat fibrotic disorders. They are also partnering with international experts in order to identify and in-license cutting-edge technologies to build a robust pipeline of antifibrotic compounds.

FogPharma is a healthcare company founded in 2015 and headquartered in Gloucester, Massachusetts. The firm is dedicated to the pharmaceutical sector, with a primary focus on developing innovative treatments for cancer. It operates with the support of a diverse group of private and institutional investors, fostering collaboration among its founders, employees, and advisors. FogPharma aims to create a new class of medicines designed to extend and enhance the lives of cancer patients. The company's commitment to its mission drives its efforts to pioneer effective solutions in cancer treatment.

The Neos Therapeutics team is dedicated to creating high-quality products and partnering with pharmaceutical and consumer healthcare companies to address key medical needs. Neos Therapeutics’ research and development (R&D) and fully integrated state of the art, FDA approved manufacturing facility is located in the Dallas/Fort Worth area.

Homology Medicines, Inc. is a genetic medicines company based in Bedford, Massachusetts, focused on improving the lives of patients with rare genetic diseases. The company employs a proprietary platform that utilizes human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies in vivo, either through gene therapy or nuclease-free gene editing. This innovative approach allows for targeted treatment of various disease-relevant tissues, including the liver and central nervous system, through a single injection. Homology's lead product candidate, HMI-102, is currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, while HMI-103 targets pediatric PKU patients, and HMI-202 is being developed for metachromatic leukodystrophy. Founded in 2015, Homology Medicines aims to address significant unmet medical needs by targeting the underlying causes of genetic disorders.

Graybug Vision, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for diseases affecting the retina and optic nerve. Founded in 2011 and based in Redwood City, California, the company is advancing several product candidates, including GB-102, an intravitreal injection formulation currently undergoing Phase I/IIa and IIb clinical trials for wet age-related macular degeneration and Phase IIa trials for diabetic macular edema. Additionally, Graybug is developing GB-103, an annual formulation of GB-102 for diabetic retinopathy, and GB-401, a depot formulation aimed at treating primary open-angle glaucoma. The company's proprietary micro- and nanoparticle controlled release technologies are designed to deliver medications at a sustained rate, enhancing patient compliance and improving clinical outcomes.

Lumos Pharma is a clinical-stage biopharmaceutical company based in Austin, Texas, dedicated to developing and commercializing therapeutics for severe, rare, and genetic diseases. Its lead candidate, LUM-201, is an oral growth hormone stimulating small molecule currently undergoing Phase 2 clinical trials for pediatric growth hormone deficiency. The company also holds a licensing agreement with Ellipses Pharma Limited to develop and commercialize nanoparticle formulations for oncology indications. Additionally, Lumos Pharma is focused on treating Creatine Transporter Deficiency, leveraging technology from the University of Cincinnati and collaborating with Key Opinion Leaders and the National Institutes of Health. Founded in 2011, Lumos Pharma aims to address unmet medical needs through innovative therapies.

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.

Ribon Therapeutics, Inc. is a biotechnology company based in Lexington, Massachusetts, founded in 2015. The company is focused on developing innovative cancer therapies that target monoPARP proteins, which are essential regulators of cancer cell survival. Ribon's scientific founders have made significant contributions to the understanding of PARPs, or poly(ADP-ribose) polymerases, and their roles in cancer. Leveraging this expertise, Ribon is establishing a proprietary drug discovery platform aimed at exploring the molecular actions and biological functions of monoPARPs. This platform supports the creation of a pipeline of therapeutics that selectively target these proteins to potentially treat cancer and other diseases. Ribon Therapeutics is supported by notable life science investors, enhancing its capability to advance its research and development efforts.

Aprecia Pharmaceuticals, LLC is a pharmaceutical company specializing in the development and manufacture of innovative drug delivery systems. Founded in 2003 and headquartered in Blue Ash, Ohio, the company focuses on advanced technologies such as ZipDose, which provides orodispersible formulations that combine high-dose capabilities with taste-masking options. Aprecia's product portfolio includes SPRITAM, a medication for treating various types of seizures, and CustomRelease, which offers tailored time release profiles for medications. The company also develops NanoDose for submilligram dosing and ImplantaDose for post-surgical management of infections and pain. Aprecia's commitment to transforming patient and caregiver experiences is evident in its use of three-dimensional printing technology to create fast-melt pharmaceutical products. The company's manufacturing facilities are located in East Windsor and New Jersey.

ESSA Pharma Inc. is a clinical-stage pharmaceutical company headquartered in Vancouver, Canada, established in 2009. The company specializes in developing novel therapies for the treatment of advanced prostate cancer, particularly metastatic castration-resistant prostate cancer (CRPC). ESSA is advancing its lead product candidate, EPI-7386, which is an oral small molecule designed to selectively block the amino-terminal domain of the androgen receptor (AR). This mechanism targets a critical component necessary for the growth and survival of prostate cancer cells, aiming to address the resistance mechanisms associated with current therapies. By focusing on this specific target, ESSA seeks to enhance the treatment options and improve survival outcomes for patients suffering from CRPC.

Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.

Xeris Biopharma is a specialty pharmaceutical company focused on developing and commercializing ready-to-use injectable and infusible drug formulations. Utilizing its proprietary XeriSol and XeriJect technology platforms, the company delivers highly concentrated, liquid-stable formulations of peptides, small molecules, and proteins through various delivery methods, including syringes and auto-injectors. Xeris Biopharma's lead product is Gvoke, a ready-to-use glucagon for treating severe hypoglycemia, while its other products include Keveyis, the first FDA-approved treatment for primary periodic paralysis, and Recorlev, approved for endogenous hypercortisolemia in Cushing's Syndrome. The company is also advancing several product candidates in clinical trials, targeting conditions in endocrinology, neurology, and gastroenterology. Founded in 2005 and headquartered in Chicago, Illinois, Xeris Biopharma aims to improve patient care by providing easier-to-use therapies that enhance treatment outcomes.

Hallux Inc. is a clinical-stage pharmaceutical company dedicated to the research and development of a novel micro-insert designed for subungual delivery to treat distal lateral subungual onychomycosis. Founded in 2012 and based in Laguna Hills, California, Hallux focuses on a biodegradable topical dosage form that delivers high concentrations of terbinafine hydrochloride directly to the site of infection. This targeted approach aims to improve treatment efficacy while minimizing systemic exposure, thus reducing the risk of liver complications and drug interactions. Initial pilot studies have shown promising results, indicating that the subungual treatment procedure is both safe and well-tolerated. Hallux's innovative technology represents a significant advancement in the treatment of onychomycosis and other nail disorders.

Akari Therapeutics is a clinical-stage biopharmaceutical company based in London, focused on developing and commercializing innovative treatments for rare and orphan autoimmune and inflammatory diseases. The company's lead product candidate, Coversin, is a second-generation complement inhibitor currently undergoing Phase II clinical trials for conditions such as paroxysmal nocturnal hemoglobinuria, Guillain-Barré syndrome, and atypical hemolytic uremic syndrome. Akari Therapeutics aims to provide life-transforming therapies that target specific pathways, including the complement component 5 (C5) and leukotriene B4 (LTB4) pathways. Founded in 2015, the company is dedicated to addressing unmet medical needs in the field of autoimmune and inflammatory diseases.

Cureatr Inc. offers a mobile care coordination solution aimed at improving medication management for healthcare providers. By integrating HIPAA-secure text messaging with workflow navigation tools, Cureatr facilitates efficient communication among medical teams. The platform is accessible on Web, Android, and iPhone devices, ensuring real-time, universal access to accurate medication data for over 265 million patients. The company addresses the significant challenges of suboptimal medication management in the United States, which leads to substantial healthcare costs and mortality. With a focus on high-quality interventions provided by board-certified telepharmacists, Cureatr aims to enhance patient safety and reduce hospitalizations. Established in 2011, the company is headquartered in New York, New York.

CytomX Therapeutics is an early stage, privately-funded biotechnology company developing Probodiesâ„¢, proteolytically-activated antibodies. Probodies, by their ability to site-direct the activity of antibodies, will result in an improved therapeutic index for validated targets as well enable the drugging of targets with broad tissue expression. Their mission, alone and with the help of partners, is to provide patients with more effective and less toxic therapies for severe illnesses such as cancer and inflammatory diseases.

Aralez Pharmaceuticals is a specialty pharmaceutical company that focuses on the acquisition, development, and commercialization of products primarily in cardiovascular health, pain management, and other specialty areas. The company offers a range of products, including Fiorinal, Proferrin, Fibricor, Uracyst, Neovisc, and Cambia, among others. Operating mainly in the United States and Canada, Aralez generates the majority of its revenue from the U.S. market, serving customers worldwide with its innovative therapeutic solutions.

Trinity Biotech is engaged in the development, manufacturing, and marketing of medical diagnostic products for clinical laboratories and point-of-care settings. The company offers a wide range of diagnostic tests and instrumentation that detect infectious diseases, sexually transmitted infections, autoimmune disorders, and conditions related to diabetes and liver health. With a portfolio of over 400 products, it employs various diagnostic formats, including immunofluorescence assays and enzyme-linked immunosorbent assays. Additionally, Trinity Biotech supplies raw materials to the life sciences and research sectors. Its products are marketed under several brand names, including Recombigen and Unigold, and are sold through a direct sales force and a network of independent distributors. The company serves a diverse clientele, including public health facilities, hospitals, and clinical laboratories. Founded in 1992 and headquartered in Bray, Ireland, Trinity Biotech has established a strong presence in the diagnostic market across regions including the Americas, Europe, Africa, and Asia.

aTyr Pharma is a biotherapeutics company that discovers and develops protein biologics for human therapeutics. The company has established a dominant intellectual property estate surrounding Physiocrine-based compositions and potential therapeutic applications. aTyr’s key programs are focused on immunomodulation disorders in the areas of inflammation and immunity and are in preclinical development. The company's primary focus is ATYR1923, a clinical-stage product candidate which binds to the neuropilin-2 receptor and is designed to down-regulate immune engagement in interstitial lung diseases. The privately held biotech was founded by The Scripps Research Institute Professor Paul Schimmel, a leading aminoacyl tRNA synthetase scientist, and is backed by top life sciences investors Alta Partners, Cardinal Partners, Domain Associates and Polaris Ventures.

DepoMed is a specialty pharmaceutical company that specializes in developing and commercializing products aimed at treating pain and conditions related to the central nervous system. Founded in 1995, the company has built a reputation for integrating innovative technology into its therapeutic offerings and successfully guiding these products through clinical approval. DepoMed's portfolio includes four FDA-approved medications: Gralise, which is used for managing postherpetic neuralgia; Cambia, an oral solution for the acute treatment of migraines in adults; Zipsor, liquid-filled capsules designed for mild to moderate acute pain relief; and Lazanda, a nasal spray for managing breakthrough pain in cancer patients. The company continues to focus on addressing unmet medical needs through its specialized product offerings.

ECPM is a medical device company focused on designing and commercializing a platform of products and services for gastrointestinal caregivers. The Company offers a range of products and services that span single-use devices and infection control products, pathology, and imaging systems. Its products are used in colonoscopy and procedures of the upper GI tract, including esophagogastroduodenoscopy (EGD). The company is headquartered in Alpharetta, Georgia.

Tonix Pharmaceuticals is a clinical-stage biopharmaceutical company based in New York, focused on the development of small molecules and biologics to treat and prevent various human diseases. The company has a diverse portfolio that includes immunology products, such as vaccines for infectious diseases and biologics aimed at treating immunosuppression, cancer, and autoimmune disorders. Notably, its lead vaccine candidate, TNX-1800, is designed to protect against COVID-19, while other candidates like TNX-801 and TNX-2300 target smallpox, monkeypox, and additional COVID-19 prevention. In the central nervous system (CNS) domain, Tonix's leading candidate, TNX-102 SL, is in Phase 3 development for fibromyalgia and is also being studied for agitation in Alzheimer’s disease and alcohol use disorder. Other CNS candidates include TNX-1300 for cocaine intoxication, TNX-601 CR for depression, and TNX-1900 for migraines. The preclinical pipeline features promising treatments for conditions like post-traumatic stress disorder and cancer. Founded in 2007, Tonix is dedicated to addressing critical health needs through its innovative therapeutic approaches.

SteadyMed Ltd. is a medical-device company pursuing innovative devices for delivering injectable therapeutic drugs for pain relief, diabetes and other chronic conditions. The company's products are pre-filled drug-infusion patches that will be sold in conjunction with a series of pharma and device companies. Specific applications of the device will be tailored in accordance with the requirements of the company's partners.

Alimera Sciences is a pharmaceutical company that specializes in the research, development, and commercialization of prescription ophthalmic pharmaceuticals. They are presently focused on diseases affecting the back of the eye, or retina because these diseases are not well treated with current therapies and will affect millions of people in their aging populations. It was founded in 2003 and is headquartered in Alpharetta, Georgia.

MedAvail operates as a technology-enabled pharmacy company that integrates automated pharmacy services within clinics and other healthcare settings using its proprietary technology. The company offers comprehensive services through its robotic dispensing platform, known as the MedAvail MedCenter, alongside home delivery options. Additionally, it runs SpotRx, a telehealth platform that facilitates remote pharmacist consultations through on-site dispensing kiosks, complemented by home delivery services. MedAvail is headquartered in Mississauga, Canada.

SHINE Medical Technologies, LLC is a manufacturer and supplier of medical tracers and cancer treatment elements, specializing in isotopes used for diagnostic and therapeutic purposes. The company produces molybdenum-99, which is essential for imaging and diagnosing conditions such as cancer and heart disease. Additionally, SHINE offers Iodine-131 for the treatment of thyroid cancer and related conditions, as well as Iodine-125, which is utilized in brachytherapy for various cancers. The company also provides Xenon-133, an inhalable gas that assists in evaluating lung function and blood flow in the brain. Founded in 2010 and headquartered in Janesville, Wisconsin, SHINE serves both domestic and international markets with its innovative medical solutions.

SHINE Medical Technologies, LLC is a manufacturer and supplier of medical tracers and cancer treatment elements, specializing in isotopes used for diagnostic and therapeutic purposes. The company produces molybdenum-99, which is essential for imaging and diagnosing conditions such as cancer and heart disease. Additionally, SHINE offers Iodine-131 for the treatment of thyroid cancer and related conditions, as well as Iodine-125, which is utilized in brachytherapy for various cancers. The company also provides Xenon-133, an inhalable gas that assists in evaluating lung function and blood flow in the brain. Founded in 2010 and headquartered in Janesville, Wisconsin, SHINE serves both domestic and international markets with its innovative medical solutions.

Horizon Therapeutics operates as a biopharmaceutical company. It focuses on the development of prescription drugs for mild to moderate pain relief and arthritis. The company's product candidates include HZT-501, a proprietary fixed-dose combination formulation of NSAID and ibuprofen; and HZT-602, a combination oral drug product consisting of naproxen and famotidine. Horizon Therapeutics is based in Northbrook, Illinois, and was founded in 2005.

Zevra Therapeutics is a development-stage biopharmaceutical company based in Celebration, Florida, focused on discovering and developing innovative therapies for pain, attention deficit hyperactivity disorder (ADHD), and other central nervous system (CNS) disorders. The company's primary initiatives include the creation of safer, abuse-resistant opioid pain relievers and proprietary prodrugs aimed at enhancing treatment efficacy and safety. Zevra's drug development platform employs its Ligand Activated Therapy (LAT) approach, utilizing patented technology to improve existing medications and expedite the development process. Among its product candidates are treatments for ADHD and stimulant use disorder, as well as an immediate-release combination product for acute pain management. The company has established a collaboration with KVK-Tech for the manufacturing and commercialization of its acute pain product. Founded in 2006, Zevra Therapeutics remains committed to addressing unmet medical needs in its therapeutic focus areas.

Spark Therapeutics, Inc. is dedicated to developing gene therapy products aimed at treating debilitating genetic diseases. The company is known for LUXTURNA, a treatment for patients with biallelic RPE65 mutation-associated retinal dystrophy, and has an extensive pipeline that includes candidates like SPK-8011 and SPK-8016 for hemophilia, SPK-7001 for choroideremia, and SPK-9001 for hemophilia B. Additionally, Spark is exploring liver-directed gene therapies such as SPK-3006 for Pompe disease and has programs targeting neurodegenerative diseases, including Huntington's disease and a form of Batten disease. Spark Therapeutics also engages in collaborations, notably with Pfizer for the SPK-FIX program for hemophilia B, and has a licensing agreement with Novartis for the commercialization of LUXTURNA outside the United States. Established in 2013 and based in Philadelphia, Spark Therapeutics operates as a subsidiary of Roche Holding AG and leverages a proprietary manufacturing platform to support its clinical studies across various therapeutic areas.

Proteon Therapeutics, Inc is a biopharmaceutical company developing pharmaceuticals to address the medical needs of patients with renal and vascular diseases. The company leverages a unique understanding of tissue remodeling to develop a pipeline of proprietary therapeutics. Proteon Therapeutics’ first drug candidate (PRT-201) is in development for the improvement of blood flow following vascular surgery procedures. The company’s initial clinical focus is vascular access for hemodialysis and peripheral arterial disease (PAD).

Infinity Pharmaceuticals, Inc. is engaged in the discovery and development of medicines aimed at treating cancer and related conditions in the United States. The company's lead product candidate is IPI-504 (retaspimycin hydrochloride), an intravenously administered small molecule inhibitor of heat shock protein 90 (Hsp90). Infinity is conducting several clinical trials for IPI-504, including a Phase II trial in combination with Herceptin for HER2-positive metastatic breast cancer and another Phase II trial for advanced non-small cell lung cancer. Additionally, the company is exploring IPI-493, an orally delivered Hsp90 inhibitor, as well as IPI-926 and IPI-940, which target advanced solid tumors and pain management, respectively. Infinity Pharmaceuticals has established strategic alliances with various partners, including Purdue Pharmaceutical Products and Novartis Institute for BioMedical Research, to advance its drug development efforts. The company is headquartered in Cambridge, Massachusetts.

Wellfount Corp. offers pharmacy services to nursing facilities. The company offers utilization and compliance reviews, multi dose, compliance packaging used for cycle and routine meds, proprietary SOPs and clinical programs, daily deliveries, intravenous therapy (IV), total parenteral nutrition (TPN), customized invoicing, EHR application integration, and medical records. It also offers medications and consulting services. Wellfount Corp. was founded in 2006 and is based in Indianapolis, Indiana.

Kolltan Pharmaceuticals, Inc. is a clinical-stage biotechnology company founded in 2007 and based in New Haven, Connecticut. The company focuses on the discovery and development of novel antibody-based drugs targeting receptor tyrosine kinases (RTKs) for the treatment of cancer and other diseases. Its lead product candidate, KTN3379, is an antibody targeting the ErbB3 RTK, currently undergoing Phase I clinical trials for advanced solid tumors. Additionally, Kolltan is developing KTN0158, a KIT RTK inhibitor aimed at treating neurofibromatosis type 1 and other inflammatory conditions, as well as an antibody-drug conjugate known as KIT-ADC for oncology applications. The company also engages in various research programs targeting RTKs, building on foundational discoveries made in collaboration with Dr. Joseph Schlessinger's laboratory at Yale University, which elucidate the molecular mechanisms linked to RTK activation and oncogenic mutations.

Auspex Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing innovative medicines for orphan diseases. Its pipeline features product candidates aimed at addressing unmet medical needs in conditions such as hyperkinetic movement disorders, which include chorea associated with Huntington’s disease, tardive dyskinesia, and Tourette syndrome. Additionally, the company is involved in creating deuterated analogs of clinically validated drugs, enhancing existing therapies across various therapeutic areas, including respiratory diseases and inflammation. Through its focused approach, Auspex Pharmaceuticals seeks to improve treatment options for patients with specific medical conditions.

Intra-Cellular Therapies, Inc., a biopharmaceutical company, develops novel drugs for the treatment of neuropsychiatric and neurologic diseases, and other disorders of the central nervous system (CNS) in the United States. The company is developing its lead drug candidate, CAPLYTA for the treatment of schizophrenia, bipolar disorder, behavioral disturbances associated with dementia, autism, and other CNS diseases. It is also developing ITI-002 that inhibits the enzyme phosphodiesterase type 1; ITI-214 for Parkinson’s disease; and ITI-333, for substance use disorders, pain, and psychiatric comorbidities, including depression and anxiety. Intra-Cellular Therapies, Inc. is headquartered in New York, New York.

MannKind Corporation is a biopharmaceutical company specializing in the development and commercialization of inhaled therapeutic products aimed at treating diabetes and pulmonary arterial hypertension. The company's flagship product, Afrezza, is an inhaled insulin designed to enhance glycemic control in adults with diabetes. Additionally, MannKind has a collaboration with United Therapeutics Corporation to develop a dry powder formulation of treprostinil for pulmonary arterial hypertension. Founded in 1991 and headquartered in Westlake Village, California, MannKind continues to focus on innovative solutions for patients with chronic conditions.

Dicerna Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of innovative treatments primarily for liver-related diseases, including rare inherited conditions, chronic liver diseases, and viral infections. Utilizing its proprietary GalXC RNA interference technology, Dicerna focuses on silencing disease-causing genes to address various health issues. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and belcesiran for genetic liver disease, among others. The company's extensive research efforts also encompass treatments for cardiovascular diseases and neurodegeneration. Dicerna has established strategic collaborations with prominent pharmaceutical companies to enhance its development capabilities. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna is at the forefront of advancing RNA-based therapeutics.

MannKind Corporation is a biopharmaceutical company specializing in the development and commercialization of inhaled therapeutic products aimed at treating diabetes and pulmonary arterial hypertension. The company's flagship product, Afrezza, is an inhaled insulin designed to enhance glycemic control in adults with diabetes. Additionally, MannKind has a collaboration with United Therapeutics Corporation to develop a dry powder formulation of treprostinil for pulmonary arterial hypertension. Founded in 1991 and headquartered in Westlake Village, California, MannKind continues to focus on innovative solutions for patients with chronic conditions.

Windtree Therapeutics (formerly Discovery Laboratories) is a clinical-stage biotechnology company focused on developing novel surfactant therapies for respiratory diseases and other potential applications. Windtree proprietary technology platform includes a synthetic, peptide-containing surfactant (KL4 surfactant) that is structurally similar to endogenous pulmonary surfactant — and novel drug-delivery technologies being developed to enable noninvasive administration of aerosolized KL4 surfactant. Windtree is focused initially on improving the management of respiratory distress syndrome (RDS) in premature infants and believes that its proprietary technology may make it possible, over time, to develop a pipeline of KL4 surfactant product candidates to address a variety of respiratory diseases for which there are few or no approved therapies.

Flamel Technologies SA is a specialty pharmaceutical company utilizing its core competencies in formulation development and drug delivery to develop safer and more efficacious pharmaceutical products, addressing unmet medical needs and/or reducing overall healthcare costs. Flamel currently markets three previously Unapproved Marketed Drugs ("UMDs") in the United States, Bloxiverz® (neostigmine methylsulfate injection), Vazculep® (phenylephrine hydrochloride injection), and Akovaz™ (ephedrine sulfate injection). The Company also develops products utilizing its proprietary drug delivery platforms, Micropump® (oral sustained release microparticles platform), along with its tangent technologies, LiquiTime® (a Micropump-derivative platform for liquid oral products) and Trigger Lock™ (a Micropump-derivative platform for abuse-resistant opioids). Additionally, the Company has developed a long acting injectable platform, Medusa™, a hydrogel depot technology, particularly suited to the development of subcutaneously administered formulations. Current applications of Flamel's drug delivery products include sodium oxybate (Micropump®), extended-release of liquid medicines such as ibuprofen and guaifenesin (LiquiTime®, through a license arrangement with Elan Pharma International Limited for the U.S. Over-the-Counter market) and a current study of the delivery of exenatide utilizing the Medusa™ technology.

Éclat Pharmaceuticals, L.L.C. is a specialty pharmaceutical company dedicated to the development, manufacturing, and commercialization of niche brands and generic products. The company focuses on creating unique medications and has successfully brought to market products such as Hycet, an oral solution containing hydrocodone bitartrate, which is used for pain relief in patients aged two years and older. Through its commitment to addressing specific medical needs, Éclat Pharmaceuticals aims to provide effective therapeutic options within the pharmaceutical landscape.

Nektar Therapeutics is a biopharmaceutical company developing a robust pipeline of novel therapeutics based on its advanced polymer conjugate chemistry technology platform. Nektar also partners with the top biopharmaceutical companies to bring new products to market. To date, Nektar's technology and drug development expertise have enabled nine approved products for partners, which include leading biopharmaceutical companies.

Talon Therapeutics, Inc. is a San Francisco Bay Area-based biopharmaceutical company dedicated to seizing upon medical opportunities, efficiently and expertly leading product candidates through clinical development, and transferring value to patients, patient care providers, shareholders, corporate partners, and employees.

Flamel Technologies SA is a specialty pharmaceutical company utilizing its core competencies in formulation development and drug delivery to develop safer and more efficacious pharmaceutical products, addressing unmet medical needs and/or reducing overall healthcare costs. Flamel currently markets three previously Unapproved Marketed Drugs ("UMDs") in the United States, Bloxiverz® (neostigmine methylsulfate injection), Vazculep® (phenylephrine hydrochloride injection), and Akovaz™ (ephedrine sulfate injection). The Company also develops products utilizing its proprietary drug delivery platforms, Micropump® (oral sustained release microparticles platform), along with its tangent technologies, LiquiTime® (a Micropump-derivative platform for liquid oral products) and Trigger Lock™ (a Micropump-derivative platform for abuse-resistant opioids). Additionally, the Company has developed a long acting injectable platform, Medusa™, a hydrogel depot technology, particularly suited to the development of subcutaneously administered formulations. Current applications of Flamel's drug delivery products include sodium oxybate (Micropump®), extended-release of liquid medicines such as ibuprofen and guaifenesin (LiquiTime®, through a license arrangement with Elan Pharma International Limited for the U.S. Over-the-Counter market) and a current study of the delivery of exenatide utilizing the Medusa™ technology.

TESARO, Inc. is an oncology-focused biopharmaceutical company established in 2010 and based in Waltham, Massachusetts. The company specializes in the identification, acquisition, development, and commercialization of cancer therapeutics and supportive care products. Its notable offerings include ZEJULA, an oral poly polymerase inhibitor used for the maintenance treatment of recurrent ovarian cancer, and VARUBI, a neurokinin-1 receptor antagonist designed to prevent chemotherapy-induced nausea and vomiting. TESARO is also advancing a range of immunotherapy candidates, such as TSR-042, TSR-022, and TSR-033, all currently in phase I clinical trials. The company has formed various collaboration and licensing agreements with industry partners, including AnaptysBio, Janssen Biotech, and Genentech. In January 2019, TESARO became a subsidiary of GlaxoSmithKline plc, further enhancing its capabilities in cancer treatment innovation.

Cytokinetics, Incorporated is a late-stage biopharmaceutical company focused on developing innovative treatments aimed at enhancing muscle function and contractility for various debilitating diseases. The company specializes in small molecule drug candidates that target muscle biology, with notable products in its pipeline including omecamtiv mecarbil, a cardiac myosin activator currently in Phase III trials for heart failure, and reldesemtiv, a fast skeletal muscle troponin activator in Phase II trials for conditions such as amyotrophic lateral sclerosis and spinal muscular atrophy. Additionally, reldesemtiv is being evaluated for chronic obstructive pulmonary disease, while CK-3773274, a cardiac myosin inhibitor, is in Phase II trials for hypertrophic cardiomyopathy. The company also has AMG 594, a cardiac troponin activator in Phase I trials. Founded in 1997 and based in South San Francisco, California, Cytokinetics is committed to addressing significant unmet medical needs through its research and development efforts.