Pfizer

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Caribou Biosciences develops cellular engineering solutions based on CRISPR technologies, offering a gene editing platform with applications in human therapeutics, disease modeling, genomics research, and agricultural biotechnology.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2016. It specializes in drug discovery, particularly focusing on solute carrier (SLC) transporters, which are vital for understanding various disease pathways. The company’s efforts are directed towards immunometabolism, lysosomal function, and mucosal defense, with applications in immuno-oncology, inflammatory disorders, and neurological diseases. Utilizing its proprietary RAPID platform, Jnana accelerates the identification of therapeutic targets and the development of small molecule therapies. By leveraging advanced chemoproteomics techniques, the company aims to enhance the efficiency of drug discovery and address significant unmet medical needs in patient care.

Nucleome Therapeutics Limited is a biotechnology company based in Oxford, United Kingdom, founded in 2019. The company focuses on decoding the "dark matter" of the human genome, which comprises over 95% of disease-linked genetic variants that have not yet been fully explored. By leveraging a unique platform that creates high-resolution three-dimensional genome structure maps and employs machine learning techniques, Nucleome aims to link these genetic variants to gene functions and map disease pathways. This innovative approach facilitates the functional validation of variants in primary cell types, enabling the discovery and development of novel therapeutics. Initially concentrating on lymphocytes and autoimmune diseases, Nucleome Therapeutics seeks to establish a robust pipeline of drug assets along with corresponding biomarkers to improve patient outcomes through genetically guided therapeutics.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

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

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2016. It specializes in drug discovery, particularly focusing on solute carrier (SLC) transporters, which are vital for understanding various disease pathways. The company’s efforts are directed towards immunometabolism, lysosomal function, and mucosal defense, with applications in immuno-oncology, inflammatory disorders, and neurological diseases. Utilizing its proprietary RAPID platform, Jnana accelerates the identification of therapeutic targets and the development of small molecule therapies. By leveraging advanced chemoproteomics techniques, the company aims to enhance the efficiency of drug discovery and address significant unmet medical needs in patient care.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Triplet Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies for triplet repeat expansion disorders, such as Huntington's disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these conditions at their source by utilizing a unique approach that involves a single oligonucleotide targeting the DNA Damage Response pathway. This strategy is designed to provide effective treatments for multiple repeat expansion disorders, enabling healthcare professionals to offer advanced biotherapeutic options to their patients. Through its research and development efforts, Triplet Therapeutics is committed to transforming the landscape of treatment for these challenging genetic disorders.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Vivet Therapeutics SAS is a biotechnology company based in Paris, France, focused on the research, development, and commercialization of gene therapy treatments for inherited liver disorders and orphan diseases. Founded in 2016, the company utilizes a liver-tropic adeno-associated virus (AAV) to deliver therapeutic genes directly to hepatocytes, aiming to correct genetic disorders at the source. Vivet is particularly engaged in developing innovative solutions for conditions such as Wilson disease, Progressive Familial Intrahepatic Cholestasis (PFIC), and Citrullinemia Type I (CTLN1). To enhance its gene therapy approaches, Vivet collaborates with the Fundación para la Investigación Médica Aplicada at the Centro de Investigación Médica Aplicada, focusing on optimizing AAV vectors for targeted liver delivery and ensuring long-term expression of therapeutic genes.

Gliknik Inc. is a biopharmaceutical company focused on developing therapies for patients with cancer and immune disorders. Founded in 2007 and based in Baltimore, Maryland, Gliknik's product portfolio includes GL-0817, aimed at preventing the recurrence of squamous cell carcinoma of the oral cavity, and GL-2045, which targets various autoimmune diseases such as immune thrombocytopenic purpura, chronic inflammatory demyelinating polyneuropathy, and Myasthenia gravis. Additionally, the company develops GB-4542, an anti-CD20 monoclonal antibody therapy. Gliknik employs several innovative platforms, including an Immunomodulator Platform that utilizes off-the-shelf peptide drugs to enhance immune responses against cancer, the Stradomer Platform that creates drugs mimicking the efficacy of human intravenous immunoglobulin, and the Stradobody Platform, which produces compounds akin to monoclonal antibodies for tumor cell targeting. Through these initiatives, Gliknik aims to provide meaningful therapies that alleviate suffering for patients facing these challenging health conditions.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

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.

BioGraph 55 is an immuno-oncology target discovery and drug development company focused on creating novel therapies for cancer and immune-related diseases. It develops B-cell–targeted therapies and bispecific antibodies for immuno-oncology and autoimmunity, employing a reverse translational approach to identify cellular targets within diseased tissues and validate mechanisms that drive disease. The company aims to address significant unmet clinical needs by leveraging insights from the tumor microenvironment to guide therapy development and translational science.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The company identifies and de-risks compounds that hold therapeutic and commercial potential, employing a rigorous process to generate data that supports both development pathways and the formation of new companies. With a team that boasts deep expertise in drug development and strong relationships within academia and patient advocacy, Cydan accelerates therapies through various stages, including clinical, regulatory, and commercial development. By facilitating the creation of effective treatments, Cydan aims to improve the lives of patients and families affected by rare diseases.

Superfluid DX is a company focused on developing advanced molecular diagnostics technology aimed at early disease detection and monitoring. By leveraging innovative research in transcriptomics, Superfluid DX analyzes tissue-specific cell-free circulating RNA found in blood samples. This non-invasive approach enables the identification of genetic information linked to neurodegenerative diseases and other conditions, providing healthcare professionals with critical insights into a patient's health status. The company's platform is designed to facilitate early diagnosis and ongoing monitoring of organ damage, ultimately improving patient outcomes by enabling timely interventions.

Bamboo Therapeutics is a biotechnology company focused on advancing gene therapies for the treatment of monogenic disorders, particularly those affecting the central nervous system and neuromuscular conditions. Founded to translate the pioneering work of Dr. Richard Jude Samulski, a leading figure in gene therapy, the company aims to move innovative treatments from the laboratory into human clinical trials, ultimately seeking drug approval. Dr. Samulski, recognized for his groundbreaking use of adeno-associated virus (AAV) as a vehicle for gene replacement, has significantly contributed to the field through the development of over twenty patents related to AAV therapeutic applications. Bamboo Therapeutics leverages his expertise in vector optimization and AAV re-engineering to enhance the safety and efficacy of its gene therapies.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is primarily engaged in the clinical study of VTS-270 for the treatment of Niemann-Pick Disease Type C (NPC) and is also involved in the pre-clinical discovery and development of additional novel drugs for NPC and other lysosomal storage diseases. Vtesse operates as a subsidiary of Sucampo Pharmaceuticals, Inc. and is recognized as the first spin-out from Cydan Development, Inc., an orphan-drug accelerator. The company collaborates with the National Institutes of Health (NIH) to advance its clinical programs and is supported by a seasoned management team with extensive experience in drug development. Vtesse's initiatives aim not only to provide innovative therapies but also to offer resources and support for patients and families affected by NPC and related conditions.

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.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is primarily engaged in the clinical study of VTS-270 for the treatment of Niemann-Pick Disease Type C (NPC) and is also involved in the pre-clinical discovery and development of additional novel drugs for NPC and other lysosomal storage diseases. Vtesse operates as a subsidiary of Sucampo Pharmaceuticals, Inc. and is recognized as the first spin-out from Cydan Development, Inc., an orphan-drug accelerator. The company collaborates with the National Institutes of Health (NIH) to advance its clinical programs and is supported by a seasoned management team with extensive experience in drug development. Vtesse's initiatives aim not only to provide innovative therapies but also to offer resources and support for patients and families affected by NPC and related conditions.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The company identifies and de-risks compounds that hold therapeutic and commercial potential, employing a rigorous process to generate data that supports both development pathways and the formation of new companies. With a team that boasts deep expertise in drug development and strong relationships within academia and patient advocacy, Cydan accelerates therapies through various stages, including clinical, regulatory, and commercial development. By facilitating the creation of effective treatments, Cydan aims to improve the lives of patients and families affected by rare diseases.

Catapult Genetics Pty., Ltd. is a developer and provider of DNA testing services focused on the livestock and aquaculture industries. The company specializes in commercializing DNA tests and gene markers designed to enhance meat production and quality, thus improving efficiency for food producers, processors, and retailers. Its product offerings include GeneSTAR tests for feed efficiency, marbling, and tenderness, as well as SireTRACE for parentage identification. Additionally, Catapult Genetics provides SureTRAK for traceability of meat products, GH Exon 5 for evaluating wagyu cattle, and SCD for selecting cattle based on genotype. The company serves a diverse clientele, including stud and commercial breeders, pastoral companies, and biotechnology firms, through distribution partners in regions such as the United Kingdom, North and South America, Europe, and South Africa. Founded in 1998 and based in Albion, Australia, Catapult Genetics operates in Australia and New Zealand and was previously known as Genetic Solutions Pty Ltd.

Genizon Biosciences Inc. discovers genes and biomarkers in common diseases using genome wide association studies (GWAS), leveraging its proven and proprietary technology platform and access to patients from the Quebec Founder Population.

Antibody discovery and optimization company developing a rational approach to antibody engineering.