Novo Holdings

Glycomine, Inc. is a biotech company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, the company addresses the significant unmet medical needs of patients suffering from rare diseases, of which approximately 7,000 exist globally, with a majority lacking FDA-approved treatments. Glycomine specializes in creating replacement therapies that combine substrates, enzymes, and proteins with advanced bio-nano materials. These therapeutics are designed to facilitate the targeted delivery of treatment to clinically relevant organs, thereby enhancing the efficacy of care for conditions characterized by metabolic disorders and protein misfolding.

Coave Therapeutics specializes in developing gene therapies for rare ocular and central nervous system (CNS) diseases. Its platform enables targeted delivery of therapeutic genes to the CNS, enhancing autophagy and lysosomal function, with a focus on improving patient outcomes for conditions like ALS.

Benchmark Genetics is a prominent company in aquaculture genetics, specializing in the development of genetic material for various species, particularly salmon, shrimp, and tilapia. With extensive experience from 30 applied breeding programs across 16 countries, the company is recognized as a leading provider of genetics services in the industry. Their approach is innovation-driven, utilizing advanced breeding technologies such as Quantitative Trait Loci (QTL) and Genomic Selection. Benchmark Genetics places significant emphasis on customer collaboration, fostering long-term partnerships with leading aquaculture businesses to enhance genetic improvements and develop new products. In addition to their genetic offerings, the company provides comprehensive technical support to ensure clients maximize the potential of their genetic resources. Their dedicated sales teams include technical specialists knowledgeable about their focus species and various production systems, including Recirculating Aquaculture Systems (RAS).

Oxford Nanopore Technologies Limited is a biotechnology company specializing in the development and commercialization of nanopore-based sequencing technology for the analysis of DNA, RNA, proteins, and small molecules. Its innovative product lineup includes the portable MinION device, the benchtop PromethION, and the GridION system, all of which enable real-time data streaming and scalable sequencing for a variety of applications in scientific research, personalized medicine, environmental monitoring, and agriculture. The Flongle offers smaller, on-demand sequencing solutions, catering to users requiring rapid insights. Operating in over 80 countries, Oxford Nanopore's technology is designed for accessibility and ease of use, featuring straightforward library preparation and automated solutions. The company maintains a robust patent portfolio, supporting its ongoing research and development in both biological and solid-state nanopore technologies. Founded in 2005 and based in Oxford, United Kingdom, Oxford Nanopore Technologies also has offices in multiple countries, including the United States, China, and Germany.

CARGO Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Clasp Therapeutics develops precision oncology platforms to identify neoantigens from driver mutations in hard-to-treat cancers. Its technology enables the creation of targeted therapies that selectively bind to HLA-presented peptides, allowing for precise destruction of cancer cells across various tumor types.

Asgard Therapeutics is a private biotechnology company spun out from Lund University that develops in vivo direct cell reprogramming approaches for cancer immunotherapy. It advances a gene therapy platform called TrojanDC that reprograms cells to function as professional antigen-presenting cells, with the goal of initiating and shaping personalized anti-tumor immune responses. The company focuses on generating immune therapies capable of addressing cancers resistant to conventional treatments.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Elo Life Systems is a biotechnology company dedicated to enhancing human health through innovative food solutions. It aims to bridge gaps between food, agriculture, and health by partnering with stakeholders across the food systems value chain. Elo employs precision breeding technologies, genomics, data analytics, machine learning, and gene editing capabilities to develop healthier, more sustainable food options.

Ray Therapeutics develops optogenetic gene therapies to restore vision in patients with blinding retinal diseases. The company designs therapies that use bioengineered light-sensitive proteins delivered to targeted retinal cells via intravitreal injections, with the goal of improving visual function across various forms of retinal blindness and inherited retinal diseases. By combining gene delivery with optogenetic control, Ray Therapeutics aims to provide durable, long-term benefits from a single treatment, addressing diseases that lead to severe retinal degeneration. The company focuses on advancing research and development in this niche field to enable patients with limited or no light perception to regain functional sight, supporting efforts to study rare retinal conditions and translate laboratory insights into potential clinical therapies.

Sangon Biotech Co. is a biotechnology company founded in 2003 as a Sino-foreign joint venture, specializing in the research and development of chemical synthesis of DNA. Over the years, Sangon has established itself as a key player in the global biotechnology market, known for its expertise in DNA synthesis, sequencing, and gene synthesis. The company has invested significantly in its infrastructure, including a manufacturing and industrial base in Songjiang Industrial Park. Sangon offers a wide range of products and services, including molecular biology kits, biochemicals, synthetic genes, oligos, peptide synthesis, enzymes, and laboratory consumables. By providing affordable life science research tools and services, Sangon supports researchers in advancing basic research and product development in the life sciences field.

Elo Life Systems is a biotechnology company dedicated to enhancing human health through innovative food solutions. It aims to bridge gaps between food, agriculture, and health by partnering with stakeholders across the food systems value chain. Elo employs precision breeding technologies, genomics, data analytics, machine learning, and gene editing capabilities to develop healthier, more sustainable food options.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

iECURE is a gene editing company developing mutation-agnostic in vivo gene insertion therapies to treat rare, life-threatening liver disorders in children. Its approach seeks long-term, potentially curative treatment for metabolic liver diseases caused by loss-of-function mutations, with initial programs targeting ornithine transcarbamylase deficiency, citrullinemia type 1, and phenylketonuria.

MedGenome develops sequencing-based diagnostics and a research platform that combines genomics technologies, bioinformatics, computing, and big data analytics to understand the genetic basis of cancer, metabolic disorders, eye disorders, and other rare diseases in diverse populations. The platform enables high-end genetic diagnostic tests across immuno-oncology, infectious diseases, reproductive health, and rare diseases, and serves patients, pharmaceutical companies, and research institutions worldwide by delivering deep insights into diseases at the genetic and molecular level to advance global health.

Inspirna is a clinical-stage biopharmaceutical company focused on developing first-in-class cancer therapies that target RNA-regulated drivers of tumor growth. Using RNA biology-based discovery platforms, including miRNA-DRIVEr and RNA-DRIVEr, it identifies novel cancer targets and advances them with oral small-molecule medicines and antibody-drug conjugates. Its lead programs include RGX-202 for RAS-mutant colorectal cancer and RGX-104 for small cell and non-small cell lung cancers, currently in early-phase trials (Phase 1b/2). The company aims to translate RNA-centric targets into treatments for patients with cancers that lack effective options.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

Asgard Therapeutics is a private biotechnology company spun out from Lund University that develops in vivo direct cell reprogramming approaches for cancer immunotherapy. It advances a gene therapy platform called TrojanDC that reprograms cells to function as professional antigen-presenting cells, with the goal of initiating and shaping personalized anti-tumor immune responses. The company focuses on generating immune therapies capable of addressing cancers resistant to conventional treatments.

Glycomine, Inc. is a biotech company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, the company addresses the significant unmet medical needs of patients suffering from rare diseases, of which approximately 7,000 exist globally, with a majority lacking FDA-approved treatments. Glycomine specializes in creating replacement therapies that combine substrates, enzymes, and proteins with advanced bio-nano materials. These therapeutics are designed to facilitate the targeted delivery of treatment to clinically relevant organs, thereby enhancing the efficacy of care for conditions characterized by metabolic disorders and protein misfolding.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, focused on developing innovative genetic medicines for rare diseases. Founded in 2015, the company utilizes a proprietary structure-inspired adeno-associated virus (AAV) vector engineering platform to create unique and differentiated vectors. These vectors are designed to improve upon naturally occurring AAV serotypes, addressing limitations of first-generation gene therapies. StrideBio's technologies facilitate gene addition, silencing, and editing, providing potential curative options for patients with severe medical conditions. By combining structural insights with accelerated evolution, StrideBio aims to produce novel AAV capsids capable of evading neutralizing antibodies, ultimately enhancing treatment outcomes for those affected by rare diseases.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Reneo Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in San Diego, California, dedicated to developing therapies for patients with rare genetic mitochondrial diseases. These conditions are characterized by deficits in cellular metabolism and energy production, often leading to significant health challenges. The company is focused on creating treatments that improve mitochondrial function, preserve muscle integrity, and enhance the overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes critical to the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. Founded in 2014, Reneo aims to address the unmet needs of patients suffering from orphan metabolic diseases linked to mitochondrial dysfunction.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

Mission Bio develops and delivers tools for studying cellular heterogeneity in human health and life science research. Its flagship product is the Tapestri Platform, a single-cell platform that detects DNA and protein changes, enabling precise detection of heterogeneity in disease progression and treatment response. The company's solutions are primarily applied in oncology, including blood cancers and solid tumors.

Freeline Therapeutics is a clinical-stage biotechnology company focused on systemic adeno-associated virus (AAV)-based gene therapies delivered via liver-directed strategies to restore functional protein expression in patients. Its pipeline includes FLT180a for hemophilia B, FLT190 for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A, with additional research programs across indications. Founded in 2015 and headquartered in Stevenage, United Kingdom, the company aims to deliver durable therapeutic proteins through engineered AAV vectors.

Allievex is a clinical-stage biotechnology company developing novel, non-opioid therapeutics for treating rare pediatric neurodegenerative diseases. Its lead product, tralesinidase alfa, is an investigational enzyme replacement therapy targeting Sanfilippo syndrome type B and mucopolysaccharidosis IIIB in children.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Glycomine, Inc. is a biotech company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, the company addresses the significant unmet medical needs of patients suffering from rare diseases, of which approximately 7,000 exist globally, with a majority lacking FDA-approved treatments. Glycomine specializes in creating replacement therapies that combine substrates, enzymes, and proteins with advanced bio-nano materials. These therapeutics are designed to facilitate the targeted delivery of treatment to clinically relevant organs, thereby enhancing the efficacy of care for conditions characterized by metabolic disorders and protein misfolding.

Oxford Biomedica is a biopharmaceutical company focused on gene and cell therapy, specializing in the development, manufacturing, and advancement of lentiviral vector-based medicines and related cell therapies. The company leverages its LentiVector platform to deliver therapeutic genes into non-dividing cells and collaborates with major pharmaceutical firms to progress both internal candidates and externally funded programs. Its internal pipeline targets diseases of the eye and the central nervous system, while ongoing partnerships span multiple therapeutic areas across Phase I/II and beyond. Oxford Biomedica also operates as a contract development and manufacturing organization, offering viral vector development and manufacturing services through OXB Solutions. Founded in 1995 and headquartered in Oxford, United Kingdom, the company maintains a global collaboration network with industry and academic partners.

Oxford Biomedica is a biopharmaceutical company focused on gene and cell therapy, specializing in the development, manufacturing, and advancement of lentiviral vector-based medicines and related cell therapies. The company leverages its LentiVector platform to deliver therapeutic genes into non-dividing cells and collaborates with major pharmaceutical firms to progress both internal candidates and externally funded programs. Its internal pipeline targets diseases of the eye and the central nervous system, while ongoing partnerships span multiple therapeutic areas across Phase I/II and beyond. Oxford Biomedica also operates as a contract development and manufacturing organization, offering viral vector development and manufacturing services through OXB Solutions. Founded in 1995 and headquartered in Oxford, United Kingdom, the company maintains a global collaboration network with industry and academic partners.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

Inspirna is a clinical-stage biopharmaceutical company focused on developing first-in-class cancer therapies that target RNA-regulated drivers of tumor growth. Using RNA biology-based discovery platforms, including miRNA-DRIVEr and RNA-DRIVEr, it identifies novel cancer targets and advances them with oral small-molecule medicines and antibody-drug conjugates. Its lead programs include RGX-202 for RAS-mutant colorectal cancer and RGX-104 for small cell and non-small cell lung cancers, currently in early-phase trials (Phase 1b/2). The company aims to translate RNA-centric targets into treatments for patients with cancers that lack effective options.

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.

Orphazyme develops innovative therapies for treating lysosomal storage diseases, a family of serious genetic disorders. Based in Copenhagen, Denmark, the company collaborates with leading academic institutions worldwide.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

Orphazyme develops innovative therapies for treating lysosomal storage diseases, a family of serious genetic disorders. Based in Copenhagen, Denmark, the company collaborates with leading academic institutions worldwide.

Inspirna is a clinical-stage biopharmaceutical company focused on developing first-in-class cancer therapies that target RNA-regulated drivers of tumor growth. Using RNA biology-based discovery platforms, including miRNA-DRIVEr and RNA-DRIVEr, it identifies novel cancer targets and advances them with oral small-molecule medicines and antibody-drug conjugates. Its lead programs include RGX-202 for RAS-mutant colorectal cancer and RGX-104 for small cell and non-small cell lung cancers, currently in early-phase trials (Phase 1b/2). The company aims to translate RNA-centric targets into treatments for patients with cancers that lack effective options.

Synlab is a provision of medical diagnostic services with a complete offer of laboratory medicine services for patients, doctors, clinics, and the pharmaceutical industry. They provide services that include laboratory analysis, synlab iunic, clinical diagnostic services, business services, service and professionals, and genetic analysis.

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).

Lysogene S.A. is a biotechnology company based in Neuilly-sur-Seine, France, focused on the research and clinical development of gene therapy for neurodegenerative disorders. Founded in 2009, the company is advancing several clinical programs, including LYS-SAF302, currently in Phase II/III trials for the treatment of mucopolysaccharidosis type IIIA, and LYS-GM101, which is in Phase I/II trials for GM1 gangliosidosis. Lysogene is also engaged in a collaborative research effort with the Weizmann Institute of Science to develop a novel AAV gene therapy approach targeting neuronopathic Gaucher disease, Parkinson's disease, and other conditions. The company aims to enhance the health outcomes of patients suffering from life-threatening, incurable disorders by utilizing AAV vectors that effectively deliver genetic material to the central nervous system.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatments for retinal diseases that have significant unmet medical needs. The company is actively working on therapeutic product candidates aimed at age-related retinal diseases, as well as gene therapy product candidates designed for orphan inherited retinal diseases. By focusing on these areas, IVERIC bio aims to address critical gaps in the current treatment landscape for patients suffering from various retinal conditions.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatments for retinal diseases that have significant unmet medical needs. The company is actively working on therapeutic product candidates aimed at age-related retinal diseases, as well as gene therapy product candidates designed for orphan inherited retinal diseases. By focusing on these areas, IVERIC bio aims to address critical gaps in the current treatment landscape for patients suffering from various retinal conditions.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

Orphazyme develops innovative therapies for treating lysosomal storage diseases, a family of serious genetic disorders. Based in Copenhagen, Denmark, the company collaborates with leading academic institutions worldwide.

HTG Molecular Diagnostics is a commercial-stage life sciences company based in Tucson, Arizona, specializing in precision medicine through its innovative HTG EdgeSeq platform. Established in 1997, the company offers a suite of molecular-based technology solutions for gene expression profiling and the measurement of various RNA types, including miRNA and mRNA. The HTG EdgeSeq technology automates sample processing, allowing for the rapid and simultaneous profiling of hundreds to thousands of molecular targets using minimal biological material. HTG Molecular Diagnostics markets a range of products, including instruments, assay kits, and software, which work together to facilitate efficient molecular profiling. Its offerings include several specialized panels designed for oncology and immuno-oncology applications. The company serves a diverse clientele comprising biopharmaceutical firms, academic research institutions, and molecular testing laboratories, distributing its products directly in the United States and Europe, as well as through international distributors.

Orphazyme develops innovative therapies for treating lysosomal storage diseases, a family of serious genetic disorders. Based in Copenhagen, Denmark, the company collaborates with leading academic institutions worldwide.

FoldRx Pharmaceuticals, Inc. is engaged in the discovery and development of disease-modifying drug therapies targeting diseases caused by protein misfolding and amyloidosis. Based in Cambridge, Massachusetts, the company focuses on addressing conditions related to the accumulation of misfolded proteins, which can result from genetic mutations or defects in cellular quality control mechanisms. Its pipeline includes advanced clinical programs aimed at treating transthyretin-associated amyloidoses, specifically those affecting the nervous and cardiovascular systems. Additionally, FoldRx is exploring therapeutic options for Parkinson's disease and cystic fibrosis. Founded in 2003, the company aims to provide healthcare professionals with effective treatments for these complex conditions.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatments for retinal diseases that have significant unmet medical needs. The company is actively working on therapeutic product candidates aimed at age-related retinal diseases, as well as gene therapy product candidates designed for orphan inherited retinal diseases. By focusing on these areas, IVERIC bio aims to address critical gaps in the current treatment landscape for patients suffering from various retinal conditions.

EpiTherapeutics is a biotechnology company focused on developing innovative cancer therapies that leverage the science of epigenetics. Founded on research conducted by Professor Kristian Helin and his team at the Biotech Research & Innovation Centre at the University of Copenhagen, the company specializes in small-molecule therapies targeting enzymes that regulate transcription in cancer cells. EpiTherapeutics has created a library of selective small molecule inhibitors, with a particular emphasis on histone demethylases, which play a critical role in gene transcription. The company's lead compounds are currently in pre-clinical development, aimed at addressing specific types of cancer.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatments for retinal diseases that have significant unmet medical needs. The company is actively working on therapeutic product candidates aimed at age-related retinal diseases, as well as gene therapy product candidates designed for orphan inherited retinal diseases. By focusing on these areas, IVERIC bio aims to address critical gaps in the current treatment landscape for patients suffering from various retinal conditions.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

Nuevolution is a prominent small molecule lead discovery company established in 2001 and headquartered in Copenhagen, Denmark. The firm specializes in developing innovative treatments for human diseases, particularly in oncology and inflammatory conditions. Central to its operations is Chemetics®, a proprietary drug discovery platform that combines wet chemistry with molecular biology, allowing for the rapid synthesis and DNA-tagging of millions of diverse small molecule compounds. This advanced technology enables efficient screening to identify potent drug leads at an unprecedented scale and speed. With a library exceeding 1 billion compounds, Nuevolution collaborates with various pharmaceutical and biotechnology companies, including notable partnerships with leading industry names. Additionally, the company is involved in a joint venture focused on the discovery and development of small molecule drugs targeting epigenetic factors. Nuevolution's capabilities have led to the identification of novel ligands with potential applications in addressing significant unmet medical needs across various therapeutic areas. The company is privately owned by a group of key Scandinavian investors.

Nuevolution is a prominent small molecule lead discovery company established in 2001 and headquartered in Copenhagen, Denmark. The firm specializes in developing innovative treatments for human diseases, particularly in oncology and inflammatory conditions. Central to its operations is Chemetics®, a proprietary drug discovery platform that combines wet chemistry with molecular biology, allowing for the rapid synthesis and DNA-tagging of millions of diverse small molecule compounds. This advanced technology enables efficient screening to identify potent drug leads at an unprecedented scale and speed. With a library exceeding 1 billion compounds, Nuevolution collaborates with various pharmaceutical and biotechnology companies, including notable partnerships with leading industry names. Additionally, the company is involved in a joint venture focused on the discovery and development of small molecule drugs targeting epigenetic factors. Nuevolution's capabilities have led to the identification of novel ligands with potential applications in addressing significant unmet medical needs across various therapeutic areas. The company is privately owned by a group of key Scandinavian investors.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

Xenon Pharmaceuticals is a biopharmaceutical company dedicated to developing novel medicines for neurological disorders. It focuses on rare genetic defects as drug targets, leveraging its proprietary discovery platform called Extreme Genetics. The company's pipeline includes XEN1101 and XEN496 for epilepsy treatment.