Deerfield Management

The Oncology Institute of Hope and Innovation is a prominent community oncology practice in the United States, established in 2007 and headquartered in Cerritos, California, with additional facilities in Arizona and Nevada. The organization provides a wide range of services aimed at cancer awareness, prevention, diagnosis, and treatment. Its offerings include chemotherapy education, comprehensive lab testing, pain management, financial and dietary counseling, and an in-house pharmacy. The institute also features an infusion center, support groups, and conducts clinical trials, ensuring patients have access to the latest treatments. Notably, it operates the Hope, Health, and Healing Center, which focuses on alternative and holistic therapies alongside conventional chemotherapy. With over 80 physicians and advanced practice providers across more than 50 clinic locations, the Oncology Institute serves over 1 million patients, delivering evidence-based cancer care in community settings.

Elion Therapeutics is a biotechnology company dedicated to addressing life-threatening invasive fungal infections (IFIs). The company develops innovative treatments by enhancing the existing Amphotericin B drug, aiming to provide a broad spectrum of antifungal options that are non-toxic to mammalian cells. Elion's approach is grounded in mechanistic insights, which facilitate the targeted optimization of natural compounds for improved therapeutic efficacy. Through its focus on clinical research and development, Elion seeks to deliver safer and more effective treatments for patients suffering from serious fungal infections.

BridgeBio Oncology Therapeutics operates as a biopharmaceutical company.

BiomX discovers and develops innovative microbiome therapeutics. The Company's mission is to develop novel therapeutics for preventing and treating diseases in which microbiome imbalances have been implicated. BiomX's microbiome modulation technologies are based on the cutting-edge innovative research of its scientific collaborators: Professor Rotem Sorek, PhD and Dr. Eran Elinav, MD, PhD both of the Weizmann Institute of Science and Professor Timothy K. Lu, MD, PhD of the Massachusetts Institute of Technology.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.

enGene is a developer of a mucosal immunotherapy platform designed to treat inflammatory bowel disease and diabetes. The company's mucosal immunotherapy platform has developed a flexible nucleotide delivery technology targeting mucosal tissues to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels, enabling physicians and doctors to regenerate physiologic, meal-regulated insulin secretion from the gut of subjects with diabetes.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Actio Biosciences focuses on the development of precision medicines tailored for homogeneous rare patient populations. The company is engaged in creating a pipeline of innovative drugs aimed at treating rare diseases, with the potential to extend their application to more common illnesses. By utilizing biotechnology to modulate the activity of proteins linked to rare diseases, Actio Biosciences aims to enhance the likelihood of clinical success and deliver effective treatments for conditions that are often difficult to cure. Their commitment to addressing both rare and common diseases positions them to make a significant impact in the field of medicine.

Bitterroot Bio is a biotechnology company focused on developing innovative immunotherapy-based treatments for cardiovascular disease. By harnessing the immune system's natural capabilities to combat illness, the company conducts research to understand the significant roles of immune cells and immune modulators in the onset and advancement of cardiovascular conditions. Through its commitment to advancing medical treatments, Bitterroot Bio aims to provide effective therapies that can transform cardiovascular disease management and improve patient outcomes.

Abivax is a clinical-stage biotechnology company based in France that develops therapeutics aimed at harnessing the body's natural immune response to treat autoimmune diseases, viral infections, and cancer. The company is currently evaluating its drug candidate, obefazimod, in Phase 3 clinical trials for the treatment of adults with moderately to severely active ulcerative colitis. Abivax focuses on addressing significant unmet medical needs in chronic inflammatory diseases, particularly inflammatory bowel diseases, which include ulcerative colitis and Crohn's disease. Through its innovative drug development platforms, Abivax aims to provide novel and effective treatment options for patients suffering from these conditions.

Abeona Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing gene and cell therapies for severe, life-threatening rare genetic diseases. The company's leading programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, and ABO-102 and ABO-101, which are AAV-based gene therapies targeting Sanfilippo syndrome types A and B, respectively. Additional therapies under development include ABO-201 and ABO-202 for Batten disease, ABO-401 for cystic fibrosis, and ABO-5OX for genetic eye disorders. Abeona also operates the AIM Vector Platform, which focuses on advancing gene therapy delivery methods using next-generation AAV capsids. The company is engaged in a licensing agreement with REGENXBIO for gene therapies addressing multiple genetic disorders. Founded in 1974 and headquartered in New York, Abeona has a fully operational manufacturing facility that supports its preclinical and clinical research efforts.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

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.

Humanity Health is a platform dedicated to supporting underrepresented leaders in healthcare and life sciences by facilitating career advancement and talent acquisition. The company employs a personalized service model to provide women and people of color with access to career-accelerating opportunities and valuable introductions. It also assists employer partners in identifying, engaging, and recruiting diverse healthcare talent, leading to more inclusive hiring outcomes. The Humanity Talent Network (HTN), a private membership network, serves to amplify and connect experienced underrepresented professionals, helping them excel in competitive executive roles, board positions, and entrepreneurial opportunities. Through its proprietary screening technology and leadership development programs, Humanity Health aims to transform the landscape of healthcare recruitment and promote diversity within the industry.

UTR Therapeutics Inc is an IND stage biotechnology company advancing mRNA Overwriting which is a spin out from City University of New York and Harvard Medical School.

Valneva is a pharmaceutical company specializing in the development of prophylactic vaccines aimed at addressing infectious diseases with significant unmet medical needs. The company has successfully commercialized several vaccines, including IXIARO/JESPECT, DUKORAL, and IXCHIQ, primarily targeting travelers. In addition to its marketed products, Valneva is advancing a diverse pipeline of vaccine candidates, including those for Lyme disease, chikungunya, and COVID-19. The company is involved in the entire lifecycle of vaccine development, encompassing research, manufacturing, and distribution. Valneva also offers technologies and services aimed at enhancing vaccine commercialization, which further supports its mission to deliver life-saving immunizations globally.

Aurion Biotechnologies is a clinical-stage biotech company focused on developing advanced therapies for ocular diseases, particularly those leading to blindness. Based in Seattle, Boston, and Tokyo, the company aims to transform the lives of millions by restoring vision through innovative cell therapies. Its lead candidate targets corneal edema and is one of the first clinically validated cell therapies in corneal care. The process involves culturing healthy cells from a donor cornea using a proprietary method, allowing cells from a single donor to potentially treat over 100 recipient eyes. Clinical trials in Japan have shown that patients experience significant and lasting improvements in corneal health indicators, including visual acuity and corneal thickness.

Vibliome has discovered a new systematic approach that enables the development of small molecule kinase inhibitors with unique selectivity profiles and, in some cases, extraordinary selectivity. There are over 500 kinases in the human genome which control nearly all cellular functions by transferring a polar phosphate group from ATP to regulatory serine, threonine, or tyrosine residues on proteins. The versatility of our chemistry platform offers many opportunities for variation while maintaining a common general architecture.

The Oncology Institute of Hope and Innovation is a prominent community oncology practice in the United States, established in 2007 and headquartered in Cerritos, California, with additional facilities in Arizona and Nevada. The organization provides a wide range of services aimed at cancer awareness, prevention, diagnosis, and treatment. Its offerings include chemotherapy education, comprehensive lab testing, pain management, financial and dietary counseling, and an in-house pharmacy. The institute also features an infusion center, support groups, and conducts clinical trials, ensuring patients have access to the latest treatments. Notably, it operates the Hope, Health, and Healing Center, which focuses on alternative and holistic therapies alongside conventional chemotherapy. With over 80 physicians and advanced practice providers across more than 50 clinic locations, the Oncology Institute serves over 1 million patients, delivering evidence-based cancer care in community settings.

GeneDx is dedicated to providing personalized health insights that enhance diagnosis, treatment, and drug discovery. By integrating extensive genomic expertise with advanced data interpretation capabilities, the company aims to establish precision medicine as a standard in healthcare. GeneDx specializes in exome and genome testing and interpretation, leveraging one of the largest rare disease data sets available. Its operations are divided into two segments: GeneDx, which generates the majority of revenue, and Legacy Sema4 diagnostics. Through these efforts, GeneDx is positioned to significantly impact health outcomes by using genomic and large-scale clinical information.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Singular Genomics is a life science technology company focused on advancing next generation sequencing (NGS) and multiomics technologies to support researchers and clinicians. The company's core platform, the Singular Sequencing Engine, underpins its product offerings, emphasizing accuracy, speed, flexibility, and scalability. Singular Genomics has developed two primary integrated solutions: the G4 Integrated Solution, which targets the NGS market with a combination of instrument and consumable kits, and the PX Integrated Solution, which integrates single cell analysis, spatial analysis, genomics, and proteomics into a versatile platform. These solutions cater to various market segments, including basic biology, oncology, and immunology, enhancing research capabilities across diverse applications.

Werewolf Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapeutics that enhance the body’s immune response to cancer. Founded in 2017, the company utilizes its proprietary PREDATOR platform to engineer conditionally activated molecules, known as INDUKINE molecules. These therapeutics are designed to remain inactive in peripheral tissues but activate selectively within the tumor microenvironment, addressing the limitations of traditional proinflammatory immune therapies. By stimulating both adaptive and innate immunity, Werewolf Therapeutics aims to improve cancer treatment outcomes.

Encodia, Inc., founded in 2015 and headquartered in San Diego, California, specializes in proteomics research and the development of innovative protein analysis technology. The company offers a platform that utilizes reverse-translation technology to convert peptide sequence information into DNA libraries, facilitating scalable and efficient protein sequencing. This capability enables researchers to analyze protein samples comprehensively, thereby advancing the field of personalized medicine. By democratizing access to detailed information on cellular processes, Encodia aims to accelerate the discovery of novel approaches to addressing challenging diseases.

Neomorph, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing protein degradation solutions, particularly through innovative molecular glue degraders. These targeted protein degradation drugs leverage the body's cellular machinery to eliminate proteins associated with various diseases. Neomorph aims to address critical health challenges by focusing on "undruggable targets," which are proteins that have been difficult to treat with traditional medicines. Through its research and development efforts, Neomorph is committed to advancing a pipeline of projects that hold promise for improving patient outcomes in the medical field.

Bridge Medicines LLC is a biotechnology company based in New York, established in 2016. It specializes in drug discovery and the development of innovative therapeutics for various human diseases, particularly in oncology and rare diseases. The company collaborates with prominent institutions such as Memorial Sloan Kettering Cancer Center, The Rockefeller University, and Weill Cornell Medicine, as well as industry partners like Takeda Pharmaceutical Company. Bridge Medicines focuses on advancing technologies from academic research through to clinical development, with a notable emphasis on developing inhibitors for acute leukemias and small molecule inhibitors for hereditary angioedema and inflammatory disorders. The initiative aims to provide comprehensive support throughout the drug development process, from validating preclinical studies to advancing candidates into human clinical trials.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

GeneDx is dedicated to providing personalized health insights that enhance diagnosis, treatment, and drug discovery. By integrating extensive genomic expertise with advanced data interpretation capabilities, the company aims to establish precision medicine as a standard in healthcare. GeneDx specializes in exome and genome testing and interpretation, leveraging one of the largest rare disease data sets available. Its operations are divided into two segments: GeneDx, which generates the majority of revenue, and Legacy Sema4 diagnostics. Through these efforts, GeneDx is positioned to significantly impact health outcomes by using genomic and large-scale clinical information.

Great Lakes Discoveries fosters preclinical and commercial clinical development to catalyze translational research initiatives. The company's new exploratory biologics initiative aims to develop a therapeutic treatment for systemic inflammation. Great Lakes Discovery is a product of the collaboration of Deerfield Management and the University of Michigan.

Element Science is a medical device and digital health company that develops solutions at the intersection of clinical-grade wearable devices, machine learning algorithms, and lifesaving therapies to address the needs of high-risk cardiovascular patients, primarily as they transition from the hospital to home. The company developing a proprietary next-generation wearable digital platform that unites patient-centric human factor engineering, sophisticated machine learning algorithm development principles, and rigorous electromechanical medical device development standards. The company was founded in 2011 and is headquartered in San Francisco, California.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.

Stelexis Therapeutics, LLC is engaged in the research and development of innovative therapies targeting pre-cancerous stem cells. Founded in 2017 and based in New York, the company utilizes a proprietary drug discovery platform to identify pre-cancerous stem and progenitor cells that contribute to the development of primary and recurrent tumors. Its focus is on developing cancer treatments that specifically target pre-cancerous events associated with hematopoietic malignancies, such as acute myeloid leukemia and myelodysplastic syndrome, as well as solid tumors. By addressing these early-stage cancer cells, Stelexis Therapeutics aims to improve therapeutic interventions and reduce the risk of relapse in patients.

Schrödinger develops a computational platform that facilitates the research efforts of biopharmaceutical and industrial companies, academic institutions, and government laboratories worldwide. Schrödinger also has wholly-owned and collaborative drug discovery programs in a broad range of therapeutic areas. Schrödinger is deeply committed to investing in the science and talent that drive its computational platform.

Ablexis, LLC is a biotechnology company based in San Francisco, California, specializing in human antibody drug discovery and development. Founded in December 2009, the company offers the AlivaMab Mouse, a transgenic mouse platform designed to enhance the efficiency of discovering therapeutic antibodies for human diseases. This innovative platform significantly improves upon traditional antibody discovery methods, facilitating biotechnology and pharmaceutical companies in developing antibody and antibody-derivative drug candidates. Ablexis commercializes its technology through strategic partnerships, aiming to enable the next generation of breakthrough antibody drugs.

Lakeside Discovery is an innovative drug discovery collaboration leveraging Northwestern’s most promising biomedical research and Deerfield’s strategic expertise to develop life-changing medicines.

Ancora Innovation is a partnership that combines Vanderbilt University's life science discovery initiatives with Deerfield's focus on advancing drug development. The organization aims to accelerate the progression of early-stage therapeutics that are grounded in innovative biological research. With a commitment of up to $65 million in funding from Deerfield, Ancora Innovation seeks to support the development of novel therapeutics and to provide capital for successful start-up companies emerging from its initiatives. By identifying promising research avenues and establishing comprehensive development plans, Ancora Innovation enables scientists to pursue potentially transformative drug discoveries and advance them toward market readiness.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Broad Institute brings together a diverse group of individuals from across its partner institutions — undergraduate and graduate students, postdoctoral fellows, professional scientists, administrative professionals, and academic faculty.

Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases and a robust intellectual property portfolio with issued composition of matter patents in the United States for its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines.

Chondrial Therapeutics s a clinical-stage biotechnology company focused on the treatment of rare diseases. It is dedicated to the pursuit of scientific research leading to the treatment for Friedreich’s Ataxia and other mitochondrial diseases.

Bridge Medicines LLC is a biotechnology company based in New York, established in 2016. It specializes in drug discovery and the development of innovative therapeutics for various human diseases, particularly in oncology and rare diseases. The company collaborates with prominent institutions such as Memorial Sloan Kettering Cancer Center, The Rockefeller University, and Weill Cornell Medicine, as well as industry partners like Takeda Pharmaceutical Company. Bridge Medicines focuses on advancing technologies from academic research through to clinical development, with a notable emphasis on developing inhibitors for acute leukemias and small molecule inhibitors for hereditary angioedema and inflammatory disorders. The initiative aims to provide comprehensive support throughout the drug development process, from validating preclinical studies to advancing candidates into human clinical trials.

Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases and a robust intellectual property portfolio with issued composition of matter patents in the United States for its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines.

Lumos Pharma, based in Austin, Texas, is an early-stage biotechnology company created to develop and commercialize a novel treatment for the rare disease Creatine Transporter Deficiency. Lumos Pharma is the exclusive licensee of technology and discoveries made in laboratories at the University of Cincinnati and has partnered with Key Opinion Leaders in the field and the United States National Institutes of Health to ensure success in developing a treatment for Creatine Transporter Deficiency. It was founded in 2014 and is headquartered in Austin, Texas.

REGENXBIO Inc. is a clinical-stage biotechnology company specializing in gene therapy aimed at addressing genetic defects and enabling cells to produce therapeutic proteins and antibodies. The company utilizes its proprietary NAV Technology Platform, which involves recombinant adeno-associated virus vectors for gene delivery. REGENXBIO’s lead candidate, RGX-314, is currently in Phase I/IIa clinical trials for the treatment of wet age-related macular degeneration. Additional product candidates under development include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Beyond its own product candidates, REGENXBIO also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has a collaboration agreement with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.

AveXis, now known as Novartis Gene Therapies, is a biotechnology company focused on developing and commercializing gene therapies aimed at treating rare and life-threatening neurological genetic disorders. Acquired by Novartis in 2018, the company specializes in motor neuron cell-targeted gene replacement therapies for conditions such as spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. By addressing these severe diseases, which often result in progressive muscle weakness, paralysis, and death, AveXis plays a crucial role in enhancing treatment options for affected patients and their families.

Zevra Therapeutics is a development-stage biopharmaceutical company dedicated to the discovery and development of innovative therapies for pain, ADHD, and other central nervous system diseases. The company is particularly focused on creating safer, abuse-resistant opioid pain relievers. Utilizing its proprietary Ligand Activated Therapy (LAT) platform, Zevra aims to enhance existing drugs and streamline the development process while safeguarding its intellectual property. Additionally, Zevra addresses the needs of patients with rare diseases by employing unique, data-driven strategies to overcome challenges in drug development. The company's mission centers on delivering transformative therapies to individuals facing limited or no treatment options, ultimately striving to improve the quality of life for those affected by these conditions.

Spark Therapeutics, Inc. is a biotechnology company focused on developing gene therapy products aimed at treating debilitating genetic diseases. Headquartered in Philadelphia, Pennsylvania, the company was founded in 2013 and has established itself as a leader in the field of gene therapy, with a robust pipeline that includes LUXTURNA for retinal dystrophy, as well as several candidates for hemophilia and choroideremia. Spark is also exploring therapies for neurodegenerative diseases and inherited retinal diseases. The company holds a collaboration agreement with Pfizer for the development of hemophilia B treatments and has partnered with Novartis for the commercialization of its lead product outside the United States. Spark Therapeutics leverages a proprietary manufacturing platform and a team with extensive expertise in research, clinical development, and regulatory affairs, building on a legacy of innovation in gene therapy from its origins at The Children’s Hospital of Philadelphia. As of December 2019, Spark operates as a subsidiary of Roche Holding AG.

Infinity Pharmaceuticals is a clinical-stage biotechnology company dedicated to developing innovative treatments for various cancers. The company's lead candidate, eganelisib, is an oral immuno-oncology therapy that aims to reprogram macrophages to counteract immune suppression in cancer. Infinity is conducting multiple clinical trials to evaluate eganelisib in combination with established therapies, including global studies such as MARIO-275, which tests eganelisib alongside Opdivo® in patients with urothelial cancer, and MARIO-3, which explores combinations with Tecentriq® and Abraxane® in triple-negative breast cancer as well as with Tecentriq and Avastin® in renal cell carcinoma. Additionally, Infinity is collaborating with Arcus Biosciences to test a novel regimen that combines eganelisib with etrumadenant and Doxil® for advanced triple-negative breast cancer patients. The company's research also encompasses treatments for hematologic malignancies and solid tumors, reflecting its commitment to addressing various oncological challenges.

Kolltan Pharmaceuticals, Inc. is a clinical-stage biotechnology company focused on the discovery and development of monoclonal antibody drugs that target receptor tyrosine kinases (RTKs) for the treatment of cancer and other diseases. Founded in 2007 and based in New Haven, Connecticut, Kolltan's lead product candidate, KTN3379, is an antibody targeting the ErbB3 RTK and is currently undergoing Phase I clinical trials for adult patients with advanced solid tumors. The company is also advancing KTN0158, a KIT RTK inhibitor aimed at treating neurofibromatosis type 1 and other inflammatory diseases, as well as KIT-ADC, an antibody-drug conjugate for oncology. Kolltan's research is informed by groundbreaking work in the laboratory of Dr. Joseph Schlessinger, which elucidates the molecular mechanisms of RTK activation and its implications for cancer.

Intra-Cellular Therapies, Inc. is a biopharmaceutical company focused on developing innovative treatments for neuropsychiatric and neurologic diseases, as well as other disorders affecting the central nervous system (CNS) in the United States. The company’s lead drug candidate, CAPLYTA, targets schizophrenia, bipolar disorder, and behavioral disturbances associated with dementia and autism. In addition to CAPLYTA, the company is advancing several other candidates, including ITI-002, which inhibits phosphodiesterase type 1; ITI-214 for Parkinson's disease; and ITI-333, aimed at substance use disorders and related psychiatric conditions such as depression and anxiety. Intra-Cellular Therapies also utilizes its CNSProfile technology platform to generate molecular signatures for drug compounds, enhancing insights into the actions of CNS drugs and aiding in drug discovery. The company is headquartered in New York, New York.

Nektar Therapeutics is a biopharmaceutical company based in San Francisco, California, with additional operations in Huntsville, Alabama, and Hyderabad, India. The company focuses on researching and developing investigational drugs for oncology, immunology, and virology, alongside a portfolio of partnered approved medicines. Its clinical pipeline includes notable candidates such as NKTR-181 for chronic low back pain, ONZEALD for advanced metastatic breast cancer, and Bempegaldesleukin, designed to enhance cancer-killing T cell activity. Nektar is also developing therapies for autoimmune diseases, including NKTR-358, and various other conditions like hemophilia A, opioid-induced constipation, and systemic lupus erythematosus. The company's strategy involves leveraging the immune system to combat cancer and restore immune balance in autoimmune disorders. Nektar has established collaboration agreements with several major pharmaceutical companies, enhancing its research capabilities and development efforts. Incorporated in 1990, Nektar Therapeutics continues to advance its innovative drug candidates through various stages of clinical trials.

WaferGen Bio-systems, Inc. is an emerging genomic analysis company in the early stage of commercialization. The company offers the transformative SmartChip Real-Time PCR System—a next-generation Real-Time PCR System for profiling and validation of gene expression patterns (biomarkers) on a single platform. The SmartChip System provides a range of high-throughput capabilities including microRNA and mRNA gene expression profiling and single nucleotide polymorphism (SNP) genotyping.

Cytokinetics is a late-stage biopharmaceutical company dedicated to the discovery and development of innovative treatments aimed at improving muscle function in patients with debilitating diseases. The company focuses on creating first-in-class muscle activators and next-in-class muscle inhibitors to address conditions characterized by reduced muscle performance, such as amyotrophic lateral sclerosis, heart failure, spinal muscular atrophy, and chronic obstructive pulmonary disease. Utilizing small molecules specifically designed to enhance muscle function and contractility, Cytokinetics aims to advance investigational medicines that have the potential to improve health outcomes for individuals suffering from severe cardiovascular and neuromuscular disorders.

Vescor Therapeutics focuses on developing autophagy-targeted therapeutics aimed at enhancing cancer treatment. The company is engaged in clinical studies that highlight the essential role of autophagy in tumor growth and the mechanisms behind resistance to existing therapies. Vescor Therapeutics specializes in creating small molecule inhibitors that act on key proteins involved in the autophagy pathway. This innovative approach is designed to provide cancer patients with improved metabolic flexibility, enabling them to better meet their energy demands and cellular building block requirements during treatment.

Exelixis, Inc. is a biotechnology company based in Alameda, California, that specializes in the discovery, development, and commercialization of therapies for cancer and other serious diseases. The company’s primary focus is on small molecule drugs, with its lead product cabozantinib marketed under the names CABOMETYX and COMETRIQ, targeting advanced renal cell carcinoma and metastatic medullary thyroid cancer, respectively. Exelixis also offers COTELLIC, a treatment for advanced melanoma developed in collaboration with Roche. In addition to its commercial products, the company is advancing a pipeline of compounds, including XL092, an oral tyrosine kinase inhibitor, and other candidates targeting various kinases involved in tumor growth. Exelixis collaborates with several pharmaceutical companies to enhance its research and development efforts. Founded in 1994, Exelixis has built a reputation for innovation in oncology therapeutics.

Exelixis, Inc. is a biotechnology company based in Alameda, California, that specializes in the discovery, development, and commercialization of therapies for cancer and other serious diseases. The company’s primary focus is on small molecule drugs, with its lead product cabozantinib marketed under the names CABOMETYX and COMETRIQ, targeting advanced renal cell carcinoma and metastatic medullary thyroid cancer, respectively. Exelixis also offers COTELLIC, a treatment for advanced melanoma developed in collaboration with Roche. In addition to its commercial products, the company is advancing a pipeline of compounds, including XL092, an oral tyrosine kinase inhibitor, and other candidates targeting various kinases involved in tumor growth. Exelixis collaborates with several pharmaceutical companies to enhance its research and development efforts. Founded in 1994, Exelixis has built a reputation for innovation in oncology therapeutics.

Array BioPharma is a biopharmaceutical company headquartered in Boulder, Colorado, that specializes in the discovery, development, and commercialization of targeted small molecule drugs for cancer and other serious diseases. Established in 1998, the company has developed several clinical-stage drugs, including binimetinib, encorafenib, selumetinib, tucatinib, ARRY-382, LOXO-292, and LOXO-195. Array BioPharma has formed partnerships with leading pharmaceutical companies such as AstraZeneca, Roche, Genentech, Loxo Oncology, and Cascadian Therapeutics to enhance its drug development efforts. The company operates in North America, Europe, and Asia Pacific, demonstrating a global commitment to providing innovative therapies for patients in need.

Third Wave Technologies, Inc. specializes in the development and marketing of molecular diagnostic products aimed at DNA and RNA analysis applications. The company provides innovative solutions for physicians and researchers focused on disease analysis and treatment. Its proprietary Invader chemistry is utilized across various segments, including women's health, infectious diseases, genetics, pharmacogenetics, and oncology. Third Wave primarily offers in vitro diagnostic devices and analyte specific reagents, which enable laboratories to create customized assays for hepatitis C virus genotyping, inherited disorders, and other genetic mutations. Additionally, the company develops products for research and agricultural biotechnology markets. Its clientele includes clinical laboratories, pharmaceutical and biotechnology firms, academic institutions, and healthcare providers. Third Wave distributes its products through a combination of direct sales personnel and collaborative partnerships in the United States and Europe. Founded in 1993 and based in Madison, Wisconsin, Third Wave Technologies operates as a subsidiary of Hologic, Inc. as of 2008.