Novartis

Granite Bio is a biotechnology company focused on developing novel monoclonal antibodies aimed at treating inflammatory, autoimmune, and fibrotic disorders. The company specializes in creating therapeutic antibodies that deplete pathogenic cells and selectively target key biological processes associated with these diseases. By concentrating on these advanced treatments, Granite Bio contributes to the broader fields of biotechnology, pharmaceuticals, and diagnostics, ultimately seeking to improve patient outcomes in complex medical conditions.

Rhygaze is focused on developing a novel gene therapy aimed at restoring vision in patients affected by diseases that lead to blindness. The company's innovative technology specifically targets cone cells, which are crucial for light detection but may lose sensitivity due to various conditions. By delivering a light-sensor gene directly to these compromised cone cells, Rhygaze seeks to repair their ability to detect light, thereby offering potential vision restoration solutions for individuals suffering from blindness.

Kate Therapeutics is a biotechnology company dedicated to developing adeno-associated virus (AAV)-based gene therapies for genetically defined muscle and heart diseases. The company employs advanced technology platforms to enhance tissue-specific delivery and gene regulation, overcoming significant challenges associated with current gene therapies. By utilizing innovative capsids, Kate Therapeutics achieves superior muscle transduction and effectively de-targets the liver, allowing for more efficient tissue transduction at lower viral doses. This approach addresses key limitations in muscle gene delivery, aiming to improve treatment outcomes for patients with muscle and heart conditions.

LoQus23 is a DDF-formed company focussing on targeting DNA damage repair pathways to treat Huntington’s disease and other triplet repeat diseases.

Borealis Biosciences is a discovery-stage biotechnology company focused on developing next-generation RNA-based medicines specifically for kidney diseases. The company utilizes its expertise in molecular and cellular biology, chemistry, and analytical capabilities to identify target opportunities within different patient subsets affected by kidney conditions. By refining methods for delivering RNA medicines to specific cell types, Borealis Biosciences aims to tackle significant unmet medical needs in the treatment of kidney diseases.

Rhygaze is focused on developing a novel gene therapy aimed at restoring vision in patients affected by diseases that lead to blindness. The company's innovative technology specifically targets cone cells, which are crucial for light detection but may lose sensitivity due to various conditions. By delivering a light-sensor gene directly to these compromised cone cells, Rhygaze seeks to repair their ability to detect light, thereby offering potential vision restoration solutions for individuals suffering from blindness.

FundaMental Pharma is a neuroscience company focused on developing innovative treatments for neurological diseases at the preclinical stage. The company creates neuroprotective molecular spacers aimed at preventing neurodegeneration in the brain. Utilizing peptides and gene therapy, FundaMental Pharma specializes in diagnosing polymorphisms and is dedicated to developing a novel class of drugs that target untreatable neurological conditions. Through its research, the company seeks to provide pharmacological solutions for patients suffering from various challenging neurological disorders.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

Orionis Biosciences is a biotechnology company focused on early-stage drug discovery and development, particularly targeting oncology and immunotherapies. Headquartered in Waltham, Massachusetts, with additional research facilities in Ghent, Belgium, the company utilizes a range of innovative technologies to create conditionally active drug modalities aimed at treating diseases with significant unmet medical needs. By inducing molecular proximity and cooperativity, Orionis enhances drug potency and precision, allowing for novel target access. Their approach facilitates the development of a diverse pipeline of drug candidates, including therapies that engage both adaptive and innate immune systems. The company collaborates with strategic partners, including VIB in Belgium, and is supported by a team of seasoned entrepreneurs and scientists, as well as a transatlantic network of investors.

Splice Bio is a biotechnology company based in Barcelona, Spain, focused on developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012 and originally named Proteodesign, S.L., the company aims to address the needs of patients suffering from incurable genetic diseases. The intein platform, developed from research in the Muir Lab at Princeton University, seeks to overcome two significant limitations of traditional adeno-associated virus (AAV) therapies: it enhances the capacity for larger gene delivery and broadens the range of tissues that can be targeted for treatment.

Gyroscope is a clinical-stage gene therapy company that specializes in developing treatments for retinal diseases, particularly focusing on Age-related Macular Degeneration (AMD), a major cause of blindness. The company leverages advancements in understanding the complement system's role in eye diseases and uses gene therapy as a therapeutic approach. Gyroscope's lead investigational therapy, GT005, is designed to be administered as a one-time treatment under the retina, aiming to restore balance to an overactive complement system by enhancing the production of the Complement Factor I protein. Currently, GT005 is undergoing evaluation in multiple clinical trials, including a Phase I/II trial named FOCUS and two Phase II trials called EXPLORE and HORIZON. Founded in 2016 in Stevenage, Herefordshire, by Andrew Lotery, David Kavanagh, and Peter Lachmann, Gyroscope seeks to provide innovative solutions for patients suffering from retinal diseases.

LoQus23 is a DDF-formed company focussing on targeting DNA damage repair pathways to treat Huntington’s disease and other triplet repeat diseases.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

Vedere Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on developing gene therapy products aimed at restoring functional vision for patients who have experienced vision loss due to Inherited Retinal Degenerations (IRDs) and other causes of vision impairment, both genetic and non-genetic. Unlike many ocular gene therapies that primarily slow the progression of vision loss, Vedere Bio seeks to restore lost vision, regardless of the patient's genetic background or stage of their condition. As of September 2020, Vedere Bio operates as a subsidiary of Novartis AG.

Novellus is a biotechnology company working to develop engineered cellular medicines. The company uses its non-immunogenic mRNA molecules to reprogram and gene-edit cells safely, and with high efficiency. They engineer precision medicines for patient segments and select from its technology toolbox for in vivo, ex vivo, allogeneic and autologous delivery. Novellus platform enables the high-level expression of gene-editing and cell-reprogramming proteins for high-specificity gene repair and both autologous and allogeneic cell therapies. The company is advancing its lead program, a breakthrough therapy for a rare dermatologic disease, through pre-clinical studies. Novellus was founded in 2015 and is headquartered in Cambridge, Massachusetts, United States.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

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.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Twentyeight-Seven Therapeutics is a developer of microRNAs designed to target regulatory proteins.The company's modulating RNA which are short non-coding RNAs that inhibit target gene expression by suppressing mRNA translation and promoting mRNA decay and develop small molecules that can modulate levels of miRNAs by targeting proteins that interact with these miRNAs, enabling users to target miRNAs are directly involved in cancer initiation, progression, and metastasis.

Coda Biotherapeutics, Inc. is a biotechnology company focused on developing innovative therapies to modulate neuronal activity through its chemogenetic platform. Established in 2014 and based in San Francisco, the company employs gene therapy techniques to modify specific neuronal populations, enabling them to express engineered ligand-gated ion channels that are responsive to a proprietary small molecule. This interaction allows for precise, dose-dependent control of neuronal activity, facilitating the treatment of conditions such as neuropathic pain, epilepsy, and movement disorders. Coda's approach utilizes viral vectors to deliver these engineered neurotransmitter receptors directly to targeted neurons, enhancing the efficiency of treatment for severe nervous system disorders compared to traditional therapies.

Twentyeight-Seven Therapeutics is a developer of microRNAs designed to target regulatory proteins.The company's modulating RNA which are short non-coding RNAs that inhibit target gene expression by suppressing mRNA translation and promoting mRNA decay and develop small molecules that can modulate levels of miRNAs by targeting proteins that interact with these miRNAs, enabling users to target miRNAs are directly involved in cancer initiation, progression, and metastasis.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

AveXis, now known as Novartis Gene Therapies, is a biotechnology company focused on developing and commercializing gene therapies aimed at treating rare and life-threatening neurological genetic disorders. Acquired by Novartis in 2018, the company specializes in motor neuron cell-targeted gene replacement therapies for conditions such as spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. By addressing these severe diseases, which often result in progressive muscle weakness, paralysis, and death, AveXis plays a crucial role in enhancing treatment options for affected patients and their families.

Homology Medicines, Inc., established in 2015 and headquartered in Bedford, Massachusetts, is a biotechnology company focused on developing genetic medicines for patients with rare genetic diseases. The company's proprietary platform employs human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies or nuclease-free gene editing treatments in vivo, targeting various disease-relevant tissues such as the liver, central nervous system, and eye. Homology Medicines has two lead product candidates: HMI-102, currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, and HMI-103, intended for PKU treatment in pediatric patients. Additionally, the company is developing HMI-202 to treat metachromatic leukodystrophy. Homology Medicines aims to cure the underlying causes of these diseases by leveraging its robust intellectual property portfolio and experienced management team with a proven track record in rare disease therapeutics.

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.

Vivet Therapeutics SAS is a biotechnology company focused on researching, developing, and commercializing gene therapy treatments for inherited liver disorders and orphan diseases. Founded in 2016 and based in Paris, France, the company employs a liver-targeting adeno-associated virus (AAV) vector to deliver therapeutic genes directly into hepatocytes, addressing genetic disorders at their source. Vivet is particularly known for its work on diseases such as Wilson disease, Progressive Familial Intrahepatic Cholestasis, and Citrullinemia Type I. In collaboration with the Fundacion para la Investigacion Medica Aplicada and the Centro de Investigación Medica Aplicada at the University of Navarra, Vivet aims to enhance gene delivery technologies and ensure long-term expression of therapeutic genes, thereby advancing the field of gene therapy for liver-related conditions.

Bionano Genomics, Inc. is a biotechnology company focused on genome mapping and analysis. It develops and markets the Saphyr system, an advanced platform designed for ultra-sensitive and specific detection of structural variations in DNA, such as insertions, deletions, and translocations, which are often linked to genetic disorders and diseases, including cancer. The Saphyr system employs optical genome mapping technology, allowing for direct visualization and analysis of long DNA molecules without relying on sequencing. This capability provides researchers and clinicians with critical insights into the structural variations within genomes, enhancing the understanding of disease mechanisms and supporting the development of personalized treatment strategies. Bionano Genomics also offers essential reagents and data solutions, including Bionano prep kits, labeling kits, and software tools for comprehensive experiment management. Its products are primarily sold to academic and governmental research laboratories, as well as pharmaceutical, biotechnology, and contract research organizations across multiple regions, including North America, Europe, and Asia. Founded in 2003 and headquartered in San Diego, California, the company was previously known as BioNano Genomics, Inc. until its name change in July 2018.

Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, that specializes in cellular engineering and genome editing solutions using CRISPR technology. Incorporated in 2011, the company has developed a CRISPR-Cas gene editing platform that allows precise modifications to DNA, which is applicable across various fields including human and animal therapeutics, agricultural biotechnology, and industrial biotech. Caribou's innovative approach aims to create transformative therapies, particularly in the development of allogeneic CAR-T and CAR-NK cell therapies, targeting serious diseases. The company focuses on advancing its pipeline of next-generation genome-edited cell therapies while also contributing to basic and applied biological research.

Bionano Genomics, Inc. is a biotechnology company focused on genome mapping and analysis. It develops and markets the Saphyr system, an advanced platform designed for ultra-sensitive and specific detection of structural variations in DNA, such as insertions, deletions, and translocations, which are often linked to genetic disorders and diseases, including cancer. The Saphyr system employs optical genome mapping technology, allowing for direct visualization and analysis of long DNA molecules without relying on sequencing. This capability provides researchers and clinicians with critical insights into the structural variations within genomes, enhancing the understanding of disease mechanisms and supporting the development of personalized treatment strategies. Bionano Genomics also offers essential reagents and data solutions, including Bionano prep kits, labeling kits, and software tools for comprehensive experiment management. Its products are primarily sold to academic and governmental research laboratories, as well as pharmaceutical, biotechnology, and contract research organizations across multiple regions, including North America, Europe, and Asia. Founded in 2003 and headquartered in San Diego, California, the company was previously known as BioNano Genomics, Inc. until its name change in July 2018.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, that specializes in cellular engineering and genome editing solutions using CRISPR technology. Incorporated in 2011, the company has developed a CRISPR-Cas gene editing platform that allows precise modifications to DNA, which is applicable across various fields including human and animal therapeutics, agricultural biotechnology, and industrial biotech. Caribou's innovative approach aims to create transformative therapies, particularly in the development of allogeneic CAR-T and CAR-NK cell therapies, targeting serious diseases. The company focuses on advancing its pipeline of next-generation genome-edited cell therapies while also contributing to basic and applied biological research.

Bionano Genomics, Inc. is a biotechnology company focused on genome mapping and analysis. It develops and markets the Saphyr system, an advanced platform designed for ultra-sensitive and specific detection of structural variations in DNA, such as insertions, deletions, and translocations, which are often linked to genetic disorders and diseases, including cancer. The Saphyr system employs optical genome mapping technology, allowing for direct visualization and analysis of long DNA molecules without relying on sequencing. This capability provides researchers and clinicians with critical insights into the structural variations within genomes, enhancing the understanding of disease mechanisms and supporting the development of personalized treatment strategies. Bionano Genomics also offers essential reagents and data solutions, including Bionano prep kits, labeling kits, and software tools for comprehensive experiment management. Its products are primarily sold to academic and governmental research laboratories, as well as pharmaceutical, biotechnology, and contract research organizations across multiple regions, including North America, Europe, and Asia. Founded in 2003 and headquartered in San Diego, California, the company was previously known as BioNano Genomics, Inc. until its name change in July 2018.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

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.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Genoptix, Inc. is a specialized provider of oncology diagnostics services, focusing on delivering comprehensive testing solutions for hematologic malignancies and solid tumors. The company offers a variety of services, including customizable clinical trial support, custom assay development, and companion diagnostics. Its diagnostic capabilities encompass chromogenic and fluorescent immunohistochemistry, cytogenetics, flow cytometry, and a range of molecular testing platforms. Genoptix performs specialty assays to aid in patient diagnosis, treatment stratification, and disease monitoring, while also providing professional services for study data interpretation and endpoint adjudication. The company collaborates with community oncologists and pathologists to enhance molecular profiling in oncology and has established a strategic alliance with Bionano Genomics. Founded in 1999 and headquartered in Carlsbad, California, Genoptix is committed to advancing cancer diagnostics and improving patient outcomes through innovative testing solutions.

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.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

Neovacs is a biotechnology company founded in 1993 as a spin-off from the Pierre & Marie Curie University in Paris by Professor Daniel Zagury, a prominent immunologist. The company specializes in the development of therapeutic vaccines known as "Kinoids," which are designed to treat a range of conditions including autoimmune diseases, allergies, and cancer. Neovacs holds an extensive patent portfolio and is recognized for its innovative approach to vaccine development, particularly in creating vaccines that target human cytokines and immunosuppressive viral proteins. Unlike traditional monoclonal antibody treatments that provide exogenous therapies, Neovacs' therapeutic vaccines promote a robust natural polyclonal antibody response in patients, positioning the company as a pioneer in the field of immunotherapy.

Okairos AG is a biopharmaceutical company that specializes in the discovery and development of genetic T-cell vaccines, with a significant focus on combating the hepatitis C virus. The company is also involved in providing health and allied services. Headquartered in Basel, Switzerland, Okairos operates in Rome and Naples, Italy. It has expertise in gene delivery technologies and scalable viral-vector manufacturing processes, which facilitate the advancement of vaccines and advanced therapies. By utilizing state-of-the-art GMP facilities, Okairos supports biotech companies in transitioning their genetic vaccines and medicinal products from preclinical stages to commercial readiness.

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.

AGY Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative treatments for central nervous system (CNS) diseases. The company targets neurodegenerative diseases, CNS injuries, stroke, cognitive disorders, and schizophrenia, with a diverse pipeline that includes both clinical and preclinical programs. AGY has developed a proprietary platform for functional gene discovery, which aids in identifying and validating therapeutic targets that can modulate disease progression. Key programs in their pipeline include treatments for stroke, cognitive impairment, and schizophrenia, as well as various antibody and receptor therapies. Founded in 1998 and based in South San Francisco, California, AGY Therapeutics aims to address significant unmet medical needs in the CNS therapeutic landscape.

AGY Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative treatments for central nervous system (CNS) diseases. The company targets neurodegenerative diseases, CNS injuries, stroke, cognitive disorders, and schizophrenia, with a diverse pipeline that includes both clinical and preclinical programs. AGY has developed a proprietary platform for functional gene discovery, which aids in identifying and validating therapeutic targets that can modulate disease progression. Key programs in their pipeline include treatments for stroke, cognitive impairment, and schizophrenia, as well as various antibody and receptor therapies. Founded in 1998 and based in South San Francisco, California, AGY Therapeutics aims to address significant unmet medical needs in the CNS therapeutic landscape.

SyStemix develops cellular and cell-based gene therapies for cancer, AIDS, and genetic diseases based on the use of isolated, expanded, and gene-modified hematopoietic stem cells. The company is based in Palo Alto, California. SyStemix, Inc. was acquired by Novartis AG. In February 1997, SyStemix completed a merger with Novartis Biotech Holding Corp., an indirect wholly owned subsidiary of Novartis, Inc. Novartis shuttered Systemix in 2000.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.