Deerfield Management

Relation Therapeutics is a biotechnology company that focuses on humanizing drug discovery and development. It leverages human genetics and clinical omics data and utilizes patient-derived tissue samples with functional genomics interventional experiments to identify, validate, and prosecute therapeutic programs.

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.

At Kura Oncology, They are committed to realizing the promise of precision medicines for cancer. The genomics revolution is transforming how they treat cancer. They now understand that how a patient responds to treatment depends in part on the genetic makeup of the cancer and, importantly, we have the knowledge and tools to create targeted treatments and companion diagnostics to identify those patients most likely to benefit. This new era offers the potential for innovative treatments that are safer and more effective for patients with particular cancers. They are leveraging our insights into cancer genetics as well as our core strength of translating novel science into life-saving medicines to advance a pipeline of precision medicines. Their development programs target cancers with high unmet need, including lung, colorectal, thyroid and pancreatic cancers as well as blood cancers such as lymphoma and leukemias.

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.

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.

Apertura Gene Therapy is a biotechnology company focused on developing innovative genetic medicines aimed at treating intractable diseases with limited treatment options. The company specializes in the engineering of adeno-associated virus (AAV) capsids that effectively bind to the human Transferrin Receptor 1 (TfR1), facilitating intravenous delivery of therapeutics through receptor-mediated transcytosis across the blood-brain barrier. This approach allows for broad penetration of the central nervous system. In addition to AAV capsids, Apertura also develops regulatory elements and therapeutic payloads to address challenges related to cellular access, gene expression, pre-existing immunity, and manufacturability. By collaborating with corporate and academic partners, Apertura is committed to advancing gene therapy and improving outcomes for patients suffering from debilitating diseases.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

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.

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.

Jaguar Gene Therapy is accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases.

Pairwise Plants is an agricultural gene editing company based in San Diego, California, focused on enhancing crops such as corn, soybeans, wheat, cotton, and canola. Founded in 2017 by a team of experts in food technology and genetics, Pairwise aims to improve the quality and nutritional value of fruits and vegetables, including leafy greens, berries, and stone fruits. The company collaborates with food and agriculture organizations to provide innovative solutions for both row and specialty crops. Pairwise holds licenses for advanced gene editing technologies from prestigious institutions, enabling the development of new crop varieties. With a commitment to building a healthier world through better food, Pairwise anticipates launching its first products in the coming years.

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.

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

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.

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.

Pairwise Plants is an agricultural gene editing company based in San Diego, California, focused on enhancing crops such as corn, soybeans, wheat, cotton, and canola. Founded in 2017 by a team of experts in food technology and genetics, Pairwise aims to improve the quality and nutritional value of fruits and vegetables, including leafy greens, berries, and stone fruits. The company collaborates with food and agriculture organizations to provide innovative solutions for both row and specialty crops. Pairwise holds licenses for advanced gene editing technologies from prestigious institutions, enabling the development of new crop varieties. With a commitment to building a healthier world through better food, Pairwise anticipates launching its first products in the coming years.

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.

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.

Audentes Therapeutics, a clinical-stage biotechnology company based in San Francisco, is dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapies, including AT132 and AT342, which are in Phase I/II clinical studies for treating X-linked myotubular myopathy and Crigler-Najjar syndrome, respectively. Additionally, it is exploring treatments for Pompe disease and catecholaminergic polymorphic ventricular tachycardia, along with vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Audentes has established collaborative agreements for research and development with Genethon and the University of Pennsylvania. Founded in 2012, Audentes Therapeutics became a subsidiary of Astellas US Holding in 2020 and operates as Astellas Gene Therapies, focusing on leveraging its expertise in gene therapy research and commercialization.

Rhythm Metabolic is the subsidiary of Rhythm Pharmaceuticals that develops setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway. It was established in 2013 and is headquartered in Boston, Massachusetts.

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.

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.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

PacBio designs, develops, and manufactures sequencing systems to resolve genetically complex problems. Its single molecule real-time (SMRT) sequencing technology enables single molecule real-time detection of biological processes.

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.

PacBio designs, develops, and manufactures sequencing systems to resolve genetically complex problems. Its single molecule real-time (SMRT) sequencing technology enables single molecule real-time detection of biological processes.

ZymoGenetics is dedicated to the discovery, development, and early manufacturing of therapeutic proteins aimed at treating human diseases. As a fully integrated research and development organization, it possesses a diverse array of expertise and technologies, encompassing scientific computing, molecular and cellular biology, protein chemistry, and antibody generation and engineering. The company is actively engaged in advancing a pipeline of therapeutic proteins, including recothrom, which targets hemostasis. ZymoGenetics also covers various aspects of clinical development, regulatory affairs, quality assurance, drug formulation, process development, and protein manufacturing, allowing for a comprehensive approach to therapeutic innovation.

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.