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.

Founded in 2021, Commit Biologics is a Danish biotechnology company dedicated to developing innovative immunotherapy platforms for cancer treatment. Its proprietary platform employs b-specific domain antibodies to engage the body's innate immune response, targeting and destroying cancer cells.

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).

Booster Therapeutics is a biotechnology company focused on developing drug modalities to regulate cellular homeostasis and eliminate toxic accumulations. Its primary goal is to reinstate balance within cells, enabling the prevention and treatment of diseases associated with aging.

Founded in 2021, Commit Biologics is a Danish biotechnology company dedicated to developing innovative immunotherapy platforms for cancer treatment. Its proprietary platform employs b-specific domain antibodies to engage the body's innate immune response, targeting and destroying cancer cells.

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.

MinervaX is a biotechnology company focused on developing maternal vaccines to prevent infections caused by Group B streptococcus (GBS), which can lead to serious adverse pregnancy outcomes and life-threatening conditions for newborns. The company leverages a specific fusion protein that has shown promise in eliciting protective immunity against various clinically relevant GBS strains. By concentrating on this innovative vaccine approach, MinervaX aims to provide medical professionals with effective tools to safeguard newborns from bacterial infections, ultimately enhancing maternal and infant health outcomes.

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.

Developer of biotechnology research company designed for the next generation of T cell receptor therapies against cancer. The company engages in the development of TCR-based immunotherapies including T-cell therapies and biologics combining genome editing, functional high-throughput screening, deep sequencing, and machine learning, addressing key efficacy and safety challenges, enabling patients to get the treatment of serious diseases with high unmet medical need.

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.

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 specializing in systemic adeno-associated virus (AAV)-based gene therapies. Its pipeline includes FLT180a for hemophilia B, FLT190 for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A, all in various stages of clinical trials.

Aligos Therapeutics is a biopharmaceutical company dedicated to developing innovative therapeutics targeting viral infections and liver diseases. Founded in 2018, the company focuses on chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH), leveraging its expertise in liver disease drug development.

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.

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.

MinervaX is a biotechnology company focused on developing maternal vaccines to prevent infections caused by Group B streptococcus (GBS), which can lead to serious adverse pregnancy outcomes and life-threatening conditions for newborns. The company leverages a specific fusion protein that has shown promise in eliciting protective immunity against various clinically relevant GBS strains. By concentrating on this innovative vaccine approach, MinervaX aims to provide medical professionals with effective tools to safeguard newborns from bacterial infections, ultimately enhancing maternal and infant health outcomes.

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.

Aligos Therapeutics is a biopharmaceutical company dedicated to developing innovative therapeutics targeting viral infections and liver diseases. Founded in 2018, the company focuses on chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH), leveraging its expertise in liver disease drug development.

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.

MinervaX is a biotechnology company focused on developing maternal vaccines to prevent infections caused by Group B streptococcus (GBS), which can lead to serious adverse pregnancy outcomes and life-threatening conditions for newborns. The company leverages a specific fusion protein that has shown promise in eliciting protective immunity against various clinically relevant GBS strains. By concentrating on this innovative vaccine approach, MinervaX aims to provide medical professionals with effective tools to safeguard newborns from bacterial infections, ultimately enhancing maternal and infant health outcomes.

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.

Alios BioPharma is focused on discovering and developing innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company utilizes a range of proprietary platform technologies, including small molecule activators of innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein-engineering of interferons. These technologies enable the development of distinct therapeutic products targeting serious viral conditions such as chronic hepatitis B and C, HIV, and respiratory viruses, including pandemic influenza and emerging diseases like SARS. Alios BioPharma's approach is supported by a proprietary chemical library of nucleoside analogs and advanced virology-based screening systems, positioning the company as a key player in the field of antiviral therapeutics.

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.

MinervaX is a biotechnology company focused on developing maternal vaccines to prevent infections caused by Group B streptococcus (GBS), which can lead to serious adverse pregnancy outcomes and life-threatening conditions for newborns. The company leverages a specific fusion protein that has shown promise in eliciting protective immunity against various clinically relevant GBS strains. By concentrating on this innovative vaccine approach, MinervaX aims to provide medical professionals with effective tools to safeguard newborns from bacterial infections, ultimately enhancing maternal and infant health outcomes.

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.

Alios BioPharma is focused on discovering and developing innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company utilizes a range of proprietary platform technologies, including small molecule activators of innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein-engineering of interferons. These technologies enable the development of distinct therapeutic products targeting serious viral conditions such as chronic hepatitis B and C, HIV, and respiratory viruses, including pandemic influenza and emerging diseases like SARS. Alios BioPharma's approach is supported by a proprietary chemical library of nucleoside analogs and advanced virology-based screening systems, positioning the company as a key player in the field of antiviral therapeutics.

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.

Alios BioPharma is focused on discovering and developing innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company utilizes a range of proprietary platform technologies, including small molecule activators of innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein-engineering of interferons. These technologies enable the development of distinct therapeutic products targeting serious viral conditions such as chronic hepatitis B and C, HIV, and respiratory viruses, including pandemic influenza and emerging diseases like SARS. Alios BioPharma's approach is supported by a proprietary chemical library of nucleoside analogs and advanced virology-based screening systems, positioning the company as a key player in the field of antiviral therapeutics.

Alios BioPharma is focused on discovering and developing innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company utilizes a range of proprietary platform technologies, including small molecule activators of innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein-engineering of interferons. These technologies enable the development of distinct therapeutic products targeting serious viral conditions such as chronic hepatitis B and C, HIV, and respiratory viruses, including pandemic influenza and emerging diseases like SARS. Alios BioPharma's approach is supported by a proprietary chemical library of nucleoside analogs and advanced virology-based screening systems, positioning the company as a key player in the field of antiviral therapeutics.

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.

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.

GlycoEra is a biotechnology company focused on developing novel therapeutics by harnessing the power of glycan-mediated biology.