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.

Commit Biologics is a biotechnology research company based in Aarhus, Denmark, established in 2021. The company specializes in developing an innovative immunotherapy platform aimed at treating various types of cancer. This platform utilizes bispecific domain antibodies to effectively target and eliminate tumor cells. Furthermore, it incorporates modular technology that enhances its ability to engage the body's innate immune response, thereby activating the complement component of the immune system. This approach not only focuses on cancer treatment but also has potential applications in addressing autoimmune diseases.

Coave Therapeutics specializes in developing gene therapies for rare ocular and central nervous system (CNS) diseases. Their primary focus is creating treatments for rare retinal disorders using a recombinant adeno-associated virus vector, which delivers a functional copy of the PDE6B gene directly between photoreceptors and the retinal pigment epithelium to provide effective visual restoration.

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 innovative medicines aimed at addressing aging-related diseases. The company specializes in regulating cellular homeostasis, which involves restoring balance within cells to eliminate toxic accumulations. By targeting the cellular systems responsible for maintaining cellular health, Booster Therapeutics aims to create drug modalities that can prevent and treat various diseases associated with aging.

Commit Biologics is a biotechnology research company based in Aarhus, Denmark, established in 2021. The company specializes in developing an innovative immunotherapy platform aimed at treating various types of cancer. This platform utilizes bispecific domain antibodies to effectively target and eliminate tumor cells. Furthermore, it incorporates modular technology that enhances its ability to engage the body's innate immune response, thereby activating the complement component of the immune system. This approach not only focuses on cancer treatment but also has potential applications in addressing autoimmune diseases.

Asgard Therapeutics is a private biotechnology company focused on developing innovative cancer immunotherapies through direct cell reprogramming technologies. Founded as a spin-off from Lund University, the company employs its proprietary TrojanDC technology to initiate immune responses by leveraging the biological properties of professional antigen-presenting cells. With a commitment to creating personalized and efficient gene therapy products, Asgard Therapeutics aims to expand its pipeline of off-the-shelf cancer treatments, particularly for patients with cancers that are resistant to traditional therapies. By activating tailored anti-cancer immune defenses, the company seeks to offer new therapeutic options for individuals in need of effective cancer treatment alternatives.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Elo Life Systems is a biotechnology company dedicated to enhancing human health and wellness through innovative food solutions. The company aims to bridge the gap between food, agriculture, and health by employing precision gene editing and breeding technologies, genomics, and data analytics. Through partnerships with stakeholders across the food systems value chain, Elo focuses on improving agricultural productivity, meeting nutritional demands, and addressing food security challenges. By replacing sugar and artificial sweeteners with healthier alternatives, Elo Life Systems empowers individuals to enjoy their favorite foods while promoting healthier lifestyles.

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 and wellness through innovative food solutions. The company aims to bridge the gap between food, agriculture, and health by employing precision gene editing and breeding technologies, genomics, and data analytics. Through partnerships with stakeholders across the food systems value chain, Elo focuses on improving agricultural productivity, meeting nutritional demands, and addressing food security challenges. By replacing sugar and artificial sweeteners with healthier alternatives, Elo Life Systems empowers individuals to enjoy their favorite foods while promoting healthier lifestyles.

Metagenomi, Inc. is a genetic medicines company focused on developing innovative gene editing systems to address genetic diseases. Incorporated in 2016 and headquartered in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to discover novel genome editing technologies. By sourcing cellular machinery from previously uncharacterized organisms, the company creates a proprietary toolbox that includes programmable nucleases, base editors, and advanced integration systems like prime editing and CRISPR-associated transposases. These systems are designed to be ultra-small, efficient, and highly specific, minimizing the risk of immune responses. Metagenomi aims to revolutionize gene editing techniques and deliver curative therapeutics to patients worldwide.

iECURE is a biotechnology company dedicated to developing innovative gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. Its approach employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted interventions in diseases characterized by metabolic "loss of function" disorders. By focusing on conditions with high unmet medical needs, iECURE aims to offer potential cures for devastating illnesses that currently lack effective treatment options. The company's commitment to advancing gene editing technology positions it as a key player in the field of genetic medicine.

MedGenome is a company focused on advancing global health through its sequencing genomics-based diagnostics and research platform. This platform utilizes genomics technologies, bioinformatics, computing, and big data analytics to explore the genetic underpinnings of various diseases, including cancer, metabolic disorders, eye disorders, and rare diseases. MedGenome offers high-end genetic diagnostic tests across multiple categories such as immuno-oncology, infectious diseases, reproductive health, and rare diseases. By leveraging the genetic diversity of large populations, the company aims to provide valuable insights that support the healthcare industry in understanding diseases at both genetic and molecular levels.

Engimmune Therapeutics is a biotechnology company focused on developing innovative T cell receptor (TCR) therapies aimed at treating solid tumors. The company leverages advanced life sciences technologies, including genome editing, functional high-throughput screening, deep sequencing, and machine learning, to create highly potent and specific immunotherapies. By addressing critical efficacy and safety challenges in cancer treatment, Engimmune Therapeutics is dedicated to providing effective solutions for patients suffering from serious diseases with significant unmet medical needs.

Metagenomi, Inc. is a genetic medicines company focused on developing innovative gene editing systems to address genetic diseases. Incorporated in 2016 and headquartered in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to discover novel genome editing technologies. By sourcing cellular machinery from previously uncharacterized organisms, the company creates a proprietary toolbox that includes programmable nucleases, base editors, and advanced integration systems like prime editing and CRISPR-associated transposases. These systems are designed to be ultra-small, efficient, and highly specific, minimizing the risk of immune responses. Metagenomi aims to revolutionize gene editing techniques and deliver curative therapeutics to patients worldwide.

Asgard Therapeutics is a private biotechnology company focused on developing innovative cancer immunotherapies through direct cell reprogramming technologies. Founded as a spin-off from Lund University, the company employs its proprietary TrojanDC technology to initiate immune responses by leveraging the biological properties of professional antigen-presenting cells. With a commitment to creating personalized and efficient gene therapy products, Asgard Therapeutics aims to expand its pipeline of off-the-shelf cancer treatments, particularly for patients with cancers that are resistant to traditional therapies. By activating tailored anti-cancer immune defenses, the company seeks to offer new therapeutic options for individuals in need of effective cancer treatment alternatives.

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 genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

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, Inc. is a biotechnology company focused on advancing the study of cellular heterogeneity through its innovative Tapestri Platform, which is the first single-cell multi-omics platform. This platform allows for the simultaneous analysis of genotype and phenotype from individual cells, enabling precise detection of variations in disease progression and treatment responses. Utilizing proprietary microfluidic droplet technology, Mission Bio provides targeted solutions for high-impact applications in fields such as oncology, hematology, and genome editing. The company also offers Tapestri Designer, a cloud-based tool for creating custom single-cell DNA panels, enhancing its capabilities in research and clinical settings. Founded in 2011 and based in South San Francisco, California, Mission Bio was previously known as Torrent Bio, Inc. before rebranding in 2013.

Freeline Therapeutics, established in 2015 and based in Stevenage, UK, is a clinical-stage biotechnology company specializing in the development of systemic adeno-associated virus (AAV) vector-mediated gene therapies. The company focuses on treating inherited, debilitating diseases by delivering functional copies of therapeutic genes into human liver cells, enabling the production of functional proteins in the bloodstream. Freeline's pipeline includes FLT180a for hemophilia B, FLT190 for Fabry disease, and FLT201 for Gaucher disease Type 1, all in various stages of clinical trials. Additionally, the company offers expertise in CMC process development, analytics, and AAV technologies.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

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, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

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, 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.

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, 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 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, 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.

GlycoEra is a biotechnology research company focused on developing innovative therapeutics that target significant unmet medical needs in inflammatory and autoimmune diseases, as well as in immune-oncology. The company has established a proprietary platform that enables the creation of first-in-class biologics by assembling customized and highly homogeneous human glycans onto glycoproteins in a site-specific manner. This approach facilitates a deeper understanding of the role of glycans in human health and disease, positioning GlycoEra to contribute meaningfully to the advancement of novel treatment options in the healthcare sector.