Novo Holdings

Glycomine, Inc. is a biotech company focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation, which can severely impact patients' health. Established in 2014 and located in San Carlos, California, Glycomine aims to address the significant unmet medical need for effective treatments in the realm of rare diseases, where approximately 95% lack any FDA-approved therapies. The company specializes in creating replacement therapies that utilize a combination of substrates, enzymes, and proteins, delivered through bio-nano materials that target specific cells within clinically relevant organs. By innovating in this area, Glycomine seeks to provide new therapeutic options for patients suffering from disorders of metabolism and protein misfolding.

Benchmark Genetics is a prominent player in aquaculture genetics, specializing in the genetic improvement of key species such as salmon, shrimp, and tilapia. With five in-house breeding programs and extensive experience from 30 applied breeding initiatives across 16 countries, the company provides a wide range of genetic services tailored to various aquaculture markets. Benchmark Genetics employs modern breeding technologies like quantitative trait loci (QTL) and genomic selection to enhance the quality and sustainability of aquatic species. The firm prioritizes customer collaboration, partnering with leading aquaculture businesses to drive genetic advancements and develop innovative products and services. Additionally, they offer technical support to ensure clients maximize the benefits of their genetic materials, with specialized sales teams focused on different production systems, including recirculating aquaculture systems (RAS).

Asceneuron develops effective therapeutics for orphan tauopathies and Alzheimer’s disease. By focusing on areas of high unmet medical need, Asceneuron aspires to become a leading biotech company specialized in small molecule drug discovery for neurodegenerative diseases. Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as e.g. neurofibrillary tangles in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major contributor to neurodegeneration and mutations in the tau gene are causative of the tauopathy fronto-temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17). Due to increasing life expectancy, Alzheimer’s disease is viewed as one of the largest healthcare problems of this century, imposing a major economic burden on societies in the Western and developing world. Current treatment options provide limited benefits supporting the urgent need for more efficacious and better tolerated medicines that address symptomatic relief as well as disease progression.

Asgard Therapeutics is a private biotech company focused on developing innovative cancer immunotherapies through direct cell reprogramming technologies. Emerging as a spin-off from Lund University, the company utilizes its proprietary TrojanDC technology to initiate immune responses by leveraging the unique properties of professional antigen-presenting cells. This approach aims to create efficient and personalized gene therapy products that activate robust anti-cancer immune defenses. By offering off-the-shelf solutions, Asgard Therapeutics seeks to provide new treatment options for patients suffering from cancers that are resistant to traditional therapies, thereby expanding the possibilities for effective cancer treatment.

LEXEO Therapeutics is a biotechnology company that specializes in the development of gene therapies, particularly those utilizing adeno-associated virus (AAV) technology. The company’s pipeline includes both rare and non-rare monogenic diseases, with a strong emphasis on hereditary and acquired conditions that present significant unmet medical needs. LEXEO's lead programs and preclinical candidates are primarily developed in collaboration with Weill Cornell Medicine’s Department of Genetic Medicine. The company aims to advance its clinical programs toward commercialization while maintaining an ongoing research partnership with Weill Cornell to enhance its preclinical efforts. Through its innovative approach, LEXEO Therapeutics seeks to transform the treatment landscape for diseases that impose a substantial burden on patients and society.

Elo Life Systems is a biotechnology company dedicated to enhancing human health and wellness through innovative food solutions. The company aims to connect food, agriculture, and health by addressing challenges related to agricultural productivity, nutritional needs, food security, and overall wellness. Elo employs advanced techniques such as precision breeding, genomics, data analytics, and machine learning to develop healthier food alternatives. By focusing on replacing sugar and artificial sweeteners, Elo strives to empower consumers to enjoy their favorite foods while leading healthier lives. Through strategic partnerships within the food systems value chain, the company seeks to create sustainable solutions that benefit both individuals and communities.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

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 and wellness through innovative food solutions. The company aims to connect food, agriculture, and health by addressing challenges related to agricultural productivity, nutritional needs, food security, and overall wellness. Elo employs advanced techniques such as precision breeding, genomics, data analytics, and machine learning to develop healthier food alternatives. By focusing on replacing sugar and artificial sweeteners, Elo strives to empower consumers to enjoy their favorite foods while leading healthier lives. Through strategic partnerships within the food systems value chain, the company seeks to create sustainable solutions that benefit both individuals and communities.

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

iECURE is a mutation-agnostic in vivo gene editing company striving to cure devastating diseases with high unmet needs.

Roslin Technologies is an ag-tech and food tech company focused on advancing biotechnologies to enhance protein production. The company specializes in animal stem cells, having developed perpetually self-renewing, pluripotent stem cells that have the potential to revolutionize the cultivated meat market. By providing these innovative cell lines and associated media formulations, Roslin Technologies enables companies in the cultivated meat industry to reduce costs and accelerate their market entry. Additionally, the company leverages its expertise in genomic capabilities to explore cell therapies aimed at treating incurable diseases in animals. Through these efforts, Roslin Technologies is committed to transforming the future of protein production.

MedGenome is a company focused on developing genomics-based diagnostics and research platforms to enhance global health. Its innovative platform leverages advanced genomics technologies, bioinformatics, and big data analytics to analyze the genetic diversity of large populations. This approach aims to uncover the genetic foundations of various health conditions, including cancer, metabolic disorders, eye diseases, and rare diseases. MedGenome provides high-quality genetic diagnostic tests across multiple disease categories, such as immuno-oncology, infectious diseases, and reproductive health. By offering these insights, the company supports patients, pharmaceutical firms, and research institutions in understanding diseases at their genetic and molecular levels, ultimately contributing to improved healthcare outcomes.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

Asgard Therapeutics is a private biotech company focused on developing innovative cancer immunotherapies through direct cell reprogramming technologies. Emerging as a spin-off from Lund University, the company utilizes its proprietary TrojanDC technology to initiate immune responses by leveraging the unique properties of professional antigen-presenting cells. This approach aims to create efficient and personalized gene therapy products that activate robust anti-cancer immune defenses. By offering off-the-shelf solutions, Asgard Therapeutics seeks to provide new treatment options for patients suffering from cancers that are resistant to traditional therapies, thereby expanding the possibilities for effective cancer treatment.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

Glycomine, Inc. is a biotech company focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation, which can severely impact patients' health. Established in 2014 and located in San Carlos, California, Glycomine aims to address the significant unmet medical need for effective treatments in the realm of rare diseases, where approximately 95% lack any FDA-approved therapies. The company specializes in creating replacement therapies that utilize a combination of substrates, enzymes, and proteins, delivered through bio-nano materials that target specific cells within clinically relevant organs. By innovating in this area, Glycomine seeks to provide new therapeutic options for patients suffering from disorders of metabolism and protein misfolding.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, established in 2015. The company focuses on developing innovative adeno-associated viral (AAV) vector technologies and therapeutics aimed at treating rare diseases. Utilizing a proprietary platform that combines structural insights with accelerated evolution, StrideBio creates novel AAV capsids designed to evade neutralizing antibodies. This approach allows for enhanced gene addition, gene silencing, and gene editing capabilities, addressing the limitations of first-generation gene therapies. By engineering unique and differentiated vectors, StrideBio aims to improve treatment outcomes for patients facing devastating conditions.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Reneo Pharmaceuticals is a clinical-stage pharmaceutical company based in San Diego, California, that focuses on developing therapies for patients with rare genetic mitochondrial diseases. These conditions are linked to defects in energy metabolism, leading to issues such as muscle weakness and wasting. The company is developing REN001, an oral drug designed to modulate genes involved in mitochondrial function, aiming to enhance adenosine triphosphate (ATP) production, which is essential for cellular energy. By improving mitochondrial activity, Reneo seeks to enhance the daily functioning and quality of life for patients suffering from these disorders. Founded in 2014, Reneo Pharmaceuticals is committed to addressing the needs of individuals with orphan metabolic diseases.

Escape Bio is a clinical-stage biopharmaceutical company that develops small molecule drugs intended to treat neurodegenerative diseases. The company is developing therapeutics for the novel, precisely therapies for genetic neurodegenerative diseases. Escape Bio's pipeline includes candidates targeting known genetic drivers of multiple diseases, including ESB1609, a small molecule S1P5 receptor agonist for the treatment of CNS lysosomal storage disorders and ESB5070, a small molecule kinase inhibitor for Parkinson’s disease patients who have an LRRK2 G2019S variant and an Alzheimer's disease program targeting ApoE4. E-scape Bio was founded in 2015 and is headquartered in San Francisco, California, United States.

Mission Bio, Inc. is a biotechnology company that specializes in tools for studying cellular heterogeneity in human health and life sciences. The company has developed the Tapestri Platform, the first single-cell multi-omics platform, which allows for the simultaneous analysis of genotype and phenotype from the same cell. This platform utilizes proprietary microfluidic droplet technology to enable precise detection of genomic variability at single-cell resolution, facilitating research in various applications, including oncology, blood cancers, solid tumors, and genome editing. Additionally, Mission Bio offers Tapestri Designer, a cloud-based tool for creating customized single-cell DNA panels, further enhancing its capabilities in targeted molecular analysis. Founded in 2011 and based in South San Francisco, California, the company was previously known as Torrent Bio, Inc. before rebranding in 2013.

Freeline Therapeutics is a clinical-stage biotechnology company focused on developing innovative adeno-associated virus (AAV) vector-mediated gene therapies aimed at treating inherited systemic diseases. The company employs engineered AAV vectors and capsids, alongside novel promoters and transgenes, to introduce functional copies of therapeutic genes into human liver cells. This process allows for the production of essential proteins in the bloodstream, addressing deficiencies caused by genetic disorders. Freeline's key product candidates include FLT180a for hemophilia B, FLT190 for Fabry disease, and FLT201 for Gaucher disease Type 1. Their ongoing development programs encompass various clinical trials to advance these gene therapies and provide transformative solutions for patients suffering from debilitating conditions.

Glycomine, Inc. is a biotech company focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation, which can severely impact patients' health. Established in 2014 and located in San Carlos, California, Glycomine aims to address the significant unmet medical need for effective treatments in the realm of rare diseases, where approximately 95% lack any FDA-approved therapies. The company specializes in creating replacement therapies that utilize a combination of substrates, enzymes, and proteins, delivered through bio-nano materials that target specific cells within clinically relevant organs. By innovating in this area, Glycomine seeks to provide new therapeutic options for patients suffering from disorders of metabolism and protein misfolding.

Oxford BioMedica is a biopharmaceutical company based in Oxford, United Kingdom, specializing in gene and cell therapy. It is recognized for its expertise in lentiviral vector technology, which allows for the integration of therapeutic genes into non-dividing cells, including neurons and retinal cells. The company operates in two main segments: Platform and Product. The Platform segment focuses on bioprocessing and development services for third parties, while the Product segment is dedicated to the clinical and preclinical development of gene and cell therapies targeting a range of diseases, particularly those affecting the ocular and central nervous system. Oxford BioMedica's product pipeline includes several candidates, such as OXB-302 for haematological tumors and OXB-203 for wet age-related macular degeneration, among others. The company has established partnerships with major pharmaceutical firms, including Novartis and Bristol Myers Squibb, to enhance its research and development efforts. Founded in 1995, Oxford BioMedica aims to transform treatment options for diseases with limited or no existing therapies.

Oxford BioMedica is a biopharmaceutical company based in Oxford, United Kingdom, specializing in gene and cell therapy. It is recognized for its expertise in lentiviral vector technology, which allows for the integration of therapeutic genes into non-dividing cells, including neurons and retinal cells. The company operates in two main segments: Platform and Product. The Platform segment focuses on bioprocessing and development services for third parties, while the Product segment is dedicated to the clinical and preclinical development of gene and cell therapies targeting a range of diseases, particularly those affecting the ocular and central nervous system. Oxford BioMedica's product pipeline includes several candidates, such as OXB-302 for haematological tumors and OXB-203 for wet age-related macular degeneration, among others. The company has established partnerships with major pharmaceutical firms, including Novartis and Bristol Myers Squibb, to enhance its research and development efforts. Founded in 1995, Oxford BioMedica aims to transform treatment options for diseases with limited or no existing therapies.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

Therachon is a biotechnology company dedicated to developing treatments for rare genetic diseases that currently lack available therapies. Based in Basel, Switzerland, with research facilities in Nice, France, Therachon focuses on creating innovative therapeutics for conditions such as achondroplasia, the most prevalent form of short-limbed dwarfism, through its lead candidate, TA-46. The company also addresses other rare gastrointestinal and musculoskeletal disorders, aiming to provide effective solutions for severe medical conditions like short bowel syndrome.

Escape Bio is a clinical-stage biopharmaceutical company that develops small molecule drugs intended to treat neurodegenerative diseases. The company is developing therapeutics for the novel, precisely therapies for genetic neurodegenerative diseases. Escape Bio's pipeline includes candidates targeting known genetic drivers of multiple diseases, including ESB1609, a small molecule S1P5 receptor agonist for the treatment of CNS lysosomal storage disorders and ESB5070, a small molecule kinase inhibitor for Parkinson’s disease patients who have an LRRK2 G2019S variant and an Alzheimer's disease program targeting ApoE4. E-scape Bio was founded in 2015 and is headquartered in San Francisco, California, United States.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

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 focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

Lysogene is a pioneer in the basic research and clinical development of gene therapy for neurodegenerative disorders . Their mission is to radically improve the health of patients suffering from incurable life threatening conditions. They do this by developing AAV vectors that have demonstrated their effectiveness in safely delivering genetic material to the central nervous system.

Alios BioPharma is focused on the discovery and development of innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company employs a range of proprietary technologies, including small molecule activators that stimulate innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein engineering of interferons. These complementary platforms enable the development of various therapeutic products targeting serious viral infections, such as chronic hepatitis B and C, HIV, respiratory viruses like pandemic influenza, and emerging viral diseases such as SARS. Alios BioPharma’s unique chemical library of nucleoside analogs and advanced virology-based screening systems further enhance its ability to create effective antiviral agents.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatment options 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. Through its focused research and development efforts, IVERIC bio seeks to address critical gaps in the treatment landscape for patients suffering from these conditions.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatment options 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. Through its focused research and development efforts, IVERIC bio seeks to address critical gaps in the treatment landscape for patients suffering from these conditions.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.

Chr. Hansen is a global bioscience company that develops natural ingredient solutions for the food, nutritional, pharmaceutical, and agricultural industries. It develops and produces cultures, enzymes, probiotics, and natural colors for a rich variety of foods, confectionery, beverages, dietary supplements, and even animal feed. All solutions are based on strong research and development competencies coupled with significant technology investments. Chr. Hansen has more than 2,500 employees in 30 countries and main production facilities in Denmark, France, USA, and Germany. It was founded in 1874 and is listed on Nasdaq Copenhagen.

Alios BioPharma is focused on the discovery and development of innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company employs a range of proprietary technologies, including small molecule activators that stimulate innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein engineering of interferons. These complementary platforms enable the development of various therapeutic products targeting serious viral infections, such as chronic hepatitis B and C, HIV, respiratory viruses like pandemic influenza, and emerging viral diseases such as SARS. Alios BioPharma’s unique chemical library of nucleoside analogs and advanced virology-based screening systems further enhance its ability to create effective antiviral agents.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatment options 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. Through its focused research and development efforts, IVERIC bio seeks to address critical gaps in the treatment landscape for patients suffering from these conditions.

Alios BioPharma is focused on the discovery and development of innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company employs a range of proprietary technologies, including small molecule activators that stimulate innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein engineering of interferons. These complementary platforms enable the development of various therapeutic products targeting serious viral infections, such as chronic hepatitis B and C, HIV, respiratory viruses like pandemic influenza, and emerging viral diseases such as SARS. Alios BioPharma’s unique chemical library of nucleoside analogs and advanced virology-based screening systems further enhance its ability to create effective antiviral agents.

Alios BioPharma is focused on the discovery and development of innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company employs a range of proprietary technologies, including small molecule activators that stimulate innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein engineering of interferons. These complementary platforms enable the development of various therapeutic products targeting serious viral infections, such as chronic hepatitis B and C, HIV, respiratory viruses like pandemic influenza, and emerging viral diseases such as SARS. Alios BioPharma’s unique chemical library of nucleoside analogs and advanced virology-based screening systems further enhance its ability to create effective antiviral agents.

EpiTherapeutics is dedicated to developing innovative cancer therapies through the exploration of epigenetics, a field advanced by Professor Kristian Helin and his team at the Biotech Research & Innovation Centre at the University of Copenhagen. The company focuses on small-molecule therapies, specifically targeting enzymes that regulate transcription in cancerous cells. EpiTherapeutics has established a library of selective small molecule inhibitors aimed at epigenetic regulation, particularly histone demethylases. Its lead pre-clinical compounds are currently being investigated for their potential effectiveness in treating various forms of cancer.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatment options 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. Through its focused research and development efforts, IVERIC bio seeks to address critical gaps in the treatment landscape for patients suffering from these conditions.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.