Deerfield Management

Singular Genomics is a life science technology company that develops innovative sequencing platforms. Its core product, the Singular Sequencing Engine, powers two integrated solutions: G4 for next-generation sequencing (NGS) markets, offering high accuracy and speed; and PX, which combines single-cell analysis, spatial analysis, genomics, and proteomics in one versatile multi-omics instrument. These products aim to empower researchers and clinicians across diverse fields such as basic biology, oncology, immunology, and more.

Pairwise Plants is an innovative agricultural company based in San Diego, California, focused on gene editing to enhance the quality of fruits and vegetables. Founded in 2017 by a team of experts in biotechnology and agriculture, Pairwise utilizes advanced techniques, including base editing and high-fidelity enzymes, which are licensed from renowned institutions such as Harvard and Massachusetts General Hospital. The company develops new varieties of crops, including leafy greens, berries, and stone fruits, in addition to working with traditional crops like corn, soybeans, wheat, cotton, and canola. By collaborating with food and agriculture organizations, Pairwise aims to create healthier and more sustainable produce, with initial products expected to be available in the coming years.

Relation Therapeutics is a biotechnology company focused on advancing drug discovery and development. It utilizes human genetics and clinical omics data, alongside patient-derived tissue samples, to identify and validate therapeutic programs. The company employs machine learning to explore the relationships between genes, proteins, and drugs, generating insights that have real translational validity. By combining single-cell multi-omics from patient tissues with functional assays, Relation Therapeutics aims to deepen the understanding of biological processes in human health and disease. This approach enables healthcare providers to discover and develop novel treatments, particularly in areas with unmet medical needs.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a 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. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Actio Biosciences specializes in developing precision medicines tailored to homogeneous rare patient populations. Its pipeline focuses on creating new drugs designed to enhance clinical success rates and ultimately deliver treatments to all patients in need, including those with challenging-to-cure illnesses.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy-based treatments for severe and life-threatening rare genetic diseases. Its pipeline includes therapies for Sanfilippo syndrome types A and B, epidermolysis bullosa, Batten disease, cystic fibrosis, and genetic eye disorders.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

UTR Therapeutics Inc is a biotechnology company focused on developing treatments for aggressive cancers driven by the overexpression of the c-MYC gene. The company, spun out from City University of New York and Harvard Medical School, specializes in researching and developing therapeutics that target and degrade specific gene transcripts involved in tumor progression. Their primary focus is on cancers like triple negative breast cancer, medulloblastoma, and osteosarcoma, aiming to provide clinicians with treatments that directly act on cancer-causing genetic expressions.

Apertura Gene Therapy focuses on advancing gene therapy treatments for severe diseases. It innovates by simultaneously engineering adeno-associated virus (AAV) capsids, regulatory elements, and payloads to overcome current limitations in gene therapy.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

GeneDx specializes in delivering personalized insights from genomic data to improve health outcomes. It combines extensive genetic expertise with advanced data science capabilities.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Singular Genomics is a life science technology company that develops innovative sequencing platforms. Its core product, the Singular Sequencing Engine, powers two integrated solutions: G4 for next-generation sequencing (NGS) markets, offering high accuracy and speed; and PX, which combines single-cell analysis, spatial analysis, genomics, and proteomics in one versatile multi-omics instrument. These products aim to empower researchers and clinicians across diverse fields such as basic biology, oncology, immunology, and more.

Jaguar Gene Therapy is developing gene therapy treatments for severe genetic diseases. Its clinical-stage pipeline focuses on neurodevelopmental disorders, Type 1 galactosemia, and Type 1 diabetes.

Jaguar Gene Therapy is developing gene therapy treatments for severe genetic diseases. Its clinical-stage pipeline focuses on neurodevelopmental disorders, Type 1 galactosemia, and Type 1 diabetes.

Pairwise Plants is an innovative agricultural company based in San Diego, California, focused on gene editing to enhance the quality of fruits and vegetables. Founded in 2017 by a team of experts in biotechnology and agriculture, Pairwise utilizes advanced techniques, including base editing and high-fidelity enzymes, which are licensed from renowned institutions such as Harvard and Massachusetts General Hospital. The company develops new varieties of crops, including leafy greens, berries, and stone fruits, in addition to working with traditional crops like corn, soybeans, wheat, cotton, and canola. By collaborating with food and agriculture organizations, Pairwise aims to create healthier and more sustainable produce, with initial products expected to be available in the coming years.

Founded in San Diego, California in 2015, Encodia specializes in proteomics research. It develops scalable and parallelized approaches to protein analysis, aiming to accelerate novel disease treatments through personalized medicine.

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

GeneDx specializes in delivering personalized insights from genomic data to improve health outcomes. It combines extensive genetic expertise with advanced data science capabilities.

Generation Bio is a genetic medicines company developing durable, redosable treatments for patients with rare and prevalent diseases. Its non-viral platform combines closed-ended DNA, cell-targeted lipid nanoparticles, and scalable manufacturing processes to deliver large and multiple genes across various tissues.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Allogene Therapeutics is a clinical-stage biotech company focused on immuno-oncology, developing genetically engineered allogeneic T-cell therapies for cancer. The company builds a pipeline of off-the-shelf donor-derived CAR-T products using gene-editing and proprietary cell-manufacturing technologies to enable broader patient eligibility and scalable production. Its programs include UCART19 for relapsed or refractory acute lymphoblastic leukemia and other allogeneic CAR-T candidates targeting lymphoma, leukemia, autoimmune diseases, and solid tumors. Revenue is primarily generated from collaborations and licensing agreements.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Pairwise Plants is an innovative agricultural company based in San Diego, California, focused on gene editing to enhance the quality of fruits and vegetables. Founded in 2017 by a team of experts in biotechnology and agriculture, Pairwise utilizes advanced techniques, including base editing and high-fidelity enzymes, which are licensed from renowned institutions such as Harvard and Massachusetts General Hospital. The company develops new varieties of crops, including leafy greens, berries, and stone fruits, in addition to working with traditional crops like corn, soybeans, wheat, cotton, and canola. By collaborating with food and agriculture organizations, Pairwise aims to create healthier and more sustainable produce, with initial products expected to be available in the coming years.

Generation Bio is a genetic medicines company developing durable, redosable treatments for patients with rare and prevalent diseases. Its non-viral platform combines closed-ended DNA, cell-targeted lipid nanoparticles, and scalable manufacturing processes to deliver large and multiple genes across various tissues.

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.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.

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.

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Spark Therapeutics focuses on developing potentially curative, one-time gene therapy products to treat debilitating genetic diseases. Its pipeline includes LUXTURNA for inherited retinal disease, SPK-FIX for hemophilia B (in collaboration with Pfizer), and preclinical programs targeting neurodegenerative diseases and other retinal degenerative conditions.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

Pacific Biosciences specializes in human, microbial, plant, and animal genomics, focusing on areas such as infectious disease research. The company designs and manufactures sequencing systems that address complex genetic challenges, utilizing its innovative single molecule real-time (SMRT) sequencing technology to detect biological processes in real time. Its product offerings include the PacBio RS II and Sequel Systems, which facilitate the monitoring and analysis of biochemical reactions at the single-molecule level, along with consumables like SMRT cells and various reagent kits. Customers range from research institutions and commercial laboratories to pharmaceutical and agricultural companies, with sales conducted through direct channels in North America and Europe, and distribution partners in Asia, the Middle East, and Latin America. Founded in 2000 and headquartered in Menlo Park, California, Pacific Biosciences continues to advance genomics research and applications.

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.

Pacific Biosciences specializes in human, microbial, plant, and animal genomics, focusing on areas such as infectious disease research. The company designs and manufactures sequencing systems that address complex genetic challenges, utilizing its innovative single molecule real-time (SMRT) sequencing technology to detect biological processes in real time. Its product offerings include the PacBio RS II and Sequel Systems, which facilitate the monitoring and analysis of biochemical reactions at the single-molecule level, along with consumables like SMRT cells and various reagent kits. Customers range from research institutions and commercial laboratories to pharmaceutical and agricultural companies, with sales conducted through direct channels in North America and Europe, and distribution partners in Asia, the Middle East, and Latin America. Founded in 2000 and headquartered in Menlo Park, California, Pacific Biosciences continues to advance genomics research and applications.

Pacific Biosciences specializes in human, microbial, plant, and animal genomics, focusing on areas such as infectious disease research. The company designs and manufactures sequencing systems that address complex genetic challenges, utilizing its innovative single molecule real-time (SMRT) sequencing technology to detect biological processes in real time. Its product offerings include the PacBio RS II and Sequel Systems, which facilitate the monitoring and analysis of biochemical reactions at the single-molecule level, along with consumables like SMRT cells and various reagent kits. Customers range from research institutions and commercial laboratories to pharmaceutical and agricultural companies, with sales conducted through direct channels in North America and Europe, and distribution partners in Asia, the Middle East, and Latin America. Founded in 2000 and headquartered in Menlo Park, California, Pacific Biosciences continues to advance genomics research and applications.

ZymoGenetics is a biotechnology company 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 competencies, including molecular and cellular biology, protein chemistry, and antibody generation and engineering. The company is actively involved in the clinical development and operations of its products and addresses regulatory affairs and quality control. ZymoGenetics is known for its therapeutic pipeline, which includes recothrom, a protein designed for hemostasis, underscoring its commitment to advancing innovative treatments in the biopharmaceutical sector.

Third Wave Technologies develops and markets molecular diagnostic products for DNA and RNA analysis, providing physicians and researchers with molecular solutions for disease analysis and treatment. Based in Madison, Wisconsin, the company offers products built on Invader chemistry used in women's health, infectious disease, genetics and pharmacogenetics, and oncology. Its portfolio includes in vitro diagnostic devices and analyte specific reagents that enable laboratories to perform hepatitis C genotyping, inherited disorders testing, and various mutations linked to genetic predispositions. The company also develops DNA and RNA analysis products for research and agricultural biotechnology markets. Its customers include clinical laboratories, pharmaceutical and biotechnology companies, academic research centers, and health care providers. Sales are executed through a combination of direct clinical-market personnel and collaborative partnerships in the United States and Europe.