Novo Holdings

Metagenomi, Inc. is a genetic medicines company based in Emeryville, California, focused on developing innovative gene editing systems for the treatment of genetic diseases. Since its incorporation in 2016, the company has utilized a proprietary metagenomics-derived genome editing toolbox that enables the creation of curative therapeutics. This toolbox encompasses a range of advanced technologies, including programmable nucleases, base editors, and various RNA and DNA-mediated integration systems, such as prime editing systems and CRISPR-associated transposases (CASTs). Metagenomi aims to address the challenges posed by diverse mutations that have traditionally been difficult to target with existing genome engineering approaches.

Genomatica, Inc. specializes in developing innovative manufacturing processes that convert renewable feedstocks into intermediate and basic chemicals. Utilizing its proprietary biotechnology platform, the company engineers microorganisms to facilitate fermentation-based production, focusing on a pipeline of 20 key chemicals, including butanediol and butadiene. Genomatica has established itself as a leader in bioengineering, aiming to foster a transition to sustainable materials. Its technology has gained recognition for commercializing processes for butanediol, used in biodegradable plastics and apparel, and butylene glycol, which is important in cosmetics and personal care products. The company is also advancing research in bio-nylon. Genomatica's achievements have been acknowledged through several awards, including the Kirkpatrick Award for outstanding chemical engineering technology and the ICIS Innovation Award for its Brontide butylene glycol. Founded in 1998 and headquartered in San Diego, California, Genomatica focuses on enhancing the economics, sustainability, and performance of chemical production.

Glycomine, Inc. is a biotechnology 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, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, specializing in the development of adeno-associated viral (AAV) vector technologies and therapeutics aimed at treating rare diseases. Founded in 2015, the company employs a unique STRucture Inspired DEsign approach that merges structural knowledge with accelerated evolution to create innovative AAV capsids. This technology is designed to evade neutralizing antibodies, thereby enhancing gene transfer efficiency. StrideBio's platform supports various gene therapy modalities, including gene addition, gene silencing, and gene editing, with applications across a range of rare genetic disorders.

Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.

Freeline Therapeutics is a clinical-stage biotechnology company focused on developing liver-directed gene therapies for bleeding disorders and other diseases. Based in Stevenage, United Kingdom, Freeline's lead product candidate, FLT180a, is undergoing Phase 1/2 clinical trials aimed at treating moderate to severe hemophilia B. The company's pipeline also includes FLT190, which is in dose-escalating Phase 1/2 trials for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A. Freeline utilizes a next-generation proprietary adeno-associated virus (AAV) vector platform, enhancing its potential for systemic gene therapy applications. Since its founding in 2015, the company has been dedicated to advancing innovative treatments in the field of gene therapy.

HepaRegeniX GmbH, founded in 2016 and based in Ulm, Germany, focuses on developing innovative drugs for the treatment of acute and chronic liver diseases. The company’s approach centers on the mitogen-activated protein kinase (MAPK) pathway, specifically targeting MAP kinase kinase 4 (MKK4), a crucial regulator of liver regeneration. Research conducted by Professor Lars Zender and his team at the University Hospital Tübingen has demonstrated that small RNA molecules can suppress MKK4 gene expression, thereby restoring the regenerative capacity of hepatocytes even in severely diseased livers. HepaRegeniX is advancing the development of small-molecule MKK4 inhibitors, which have the potential to address liver disorders, including nonalcoholic steatohepatitis, by enhancing liver regeneration.

Glycomine, Inc. is a biotechnology 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, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.

Oxford BioMedica is a pioneer of gene and cell therapy, with a leading industry position in lentiviral vector and cell therapy research, development and manufacture. Gene therapy is the treatment of disease by delivering therapeutic DNA into a patient’s cells. This can be either in vivo or ex vivo, the latter encompassing the field of cell therapy whereby genetically modified cells are put back into the body. Their pipeline of seven gene and cell therapy therapy products addresses diseases for which there is currently no treatment or that are inadequately treated today, including ocular and central nervous system disorders. Their product candidates have the potential to transform treatment landscapes. Their strategy is to develop their product candidates to their next value inflection points whilst continuing to build OXB Solutions into a valuable revenue-generating manufacturing and development services business.

Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.

HepaRegeniX GmbH, founded in 2016 and based in Ulm, Germany, focuses on developing innovative drugs for the treatment of acute and chronic liver diseases. The company’s approach centers on the mitogen-activated protein kinase (MAPK) pathway, specifically targeting MAP kinase kinase 4 (MKK4), a crucial regulator of liver regeneration. Research conducted by Professor Lars Zender and his team at the University Hospital Tübingen has demonstrated that small RNA molecules can suppress MKK4 gene expression, thereby restoring the regenerative capacity of hepatocytes even in severely diseased livers. HepaRegeniX is advancing the development of small-molecule MKK4 inhibitors, which have the potential to address liver disorders, including nonalcoholic steatohepatitis, by enhancing liver regeneration.

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.

HTG Molecular Diagnostics is a commercial stage life sciences company based in Tucson, Arizona, specializing in precision medicine. The company develops and markets molecular-based technology solutions for gene expression profiling and the measurement of miRNA, siRNA, and mRNA, which are essential for translational medicine and diagnostic applications. HTG offers a range of instrumentation, assay kits, and software that automate sample processing and analyze various molecular targets. Its platforms create molecular profiling libraries for next-generation sequencing, with marketed panels including oncology biomarker panels and immuno-oncology assays. HTG serves biopharmaceutical companies, academic research centers, and molecular testing laboratories, distributing its products directly within the United States and Europe, as well as through distributors in other regions. The company is also engaged in collaborations for the development of RNA and DNA profiling tests, enhancing its role in the evolving landscape of molecular diagnostics.

FoldRx Pharmaceuticals, Inc. is a biotechnology company focused on discovering and developing disease-modifying drug therapies for conditions related to protein misfolding and amyloidosis. The company’s pipeline includes advanced clinical programs targeting genetic neurological and cardiovascular disorders, specifically transthyretin-associated amyloidoses that affect both polyneuropathy and cardiomyopathy. Additionally, FoldRx is engaged in early-stage discovery efforts aimed at addressing diseases such as Parkinson's and cystic fibrosis. Established in 2003 and headquartered in Cambridge, Massachusetts, the company aims to provide effective treatments for the accumulation of misfolded proteins caused by genetic mutations and deficiencies in cellular quality control mechanisms.

EpiTherapeutics is focused on developing innovative cancer therapies through the exploration of epigenetics, an area pioneered by Professor Kristian Helin and his team at the Biotech Research & Innovation Centre in Copenhagen. The company specializes in small-molecule therapies that target enzymes crucial for regulating gene transcription, particularly histone demethylases. EpiTherapeutics has created a library of selective small molecule inhibitors aimed at modulating epigenetic regulation. Its lead pre-clinical compounds are currently being evaluated for their efficacy in treating various forms of cancer, positioning the company as a key player in advancing cancer treatment through epigenetic research.

Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Burnaby, Canada, focused on developing innovative therapeutics for neurological disorders. The company's clinical pipeline includes several product candidates targeting epilepsy, such as XEN496 and XEN1101, both Kv7 potassium channel modulators currently in Phase II trials, and XEN901, a selective Nav1.6 sodium channel inhibitor in Phase I trials. Additionally, XEN007, a central nervous system-acting calcium channel modulator, is also in Phase I trials. Xenon utilizes a proprietary discovery platform, known as Extreme Genetics, to identify genetic insights from families with severe phenotypes, enabling the development of novel medicines for both orphan and more common diseases. The company has established a collaboration with Neurocrine Biosciences to advance first-in-class treatments for epilepsy, highlighting its commitment to addressing unmet medical needs in the field of neurology.