Novartis

Granite Bio is a biotechnology company focused on developing novel monoclonal antibody therapies targeting inflammatory, autoimmune, and fibrotic disorders.

Rhygaze is a biotechnology company focused on developing innovative gene therapies aimed at restoring vision in individuals affected by blindness-related diseases. The company’s therapy utilizes advanced technology to deliver a light-sensor gene directly to cone cells that have lost their light sensitivity. By repairing these cells' ability to detect light, Rhygaze seeks to enable effective vision restoration for patients. Through its specialized approach, the company aims to make significant contributions to the healthcare industry and improve the quality of life for those facing vision loss.

Kate Therapeutics is a biotechnology company specializing in developing adeno-associated virus (AAV)-based gene therapies for genetically defined muscle and heart diseases. It employs innovative technology platforms to enhance tissue-specific delivery and gene regulation, addressing key limitations of existing gene therapies.

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 biotechnology company focused on developing next-generation RNA-based therapies for kidney diseases. It specializes in molecular and cellular biology, vivarium space, chemistry, and analytical capabilities to enhance the delivery of RNA medicines to specific cell types.

Rhygaze is a biotechnology company focused on developing innovative gene therapies aimed at restoring vision in individuals affected by blindness-related diseases. The company’s therapy utilizes advanced technology to deliver a light-sensor gene directly to cone cells that have lost their light sensitivity. By repairing these cells' ability to detect light, Rhygaze seeks to enable effective vision restoration for patients. Through its specialized approach, the company aims to make significant contributions to the healthcare industry and improve the quality of life for those facing vision loss.

FundaMental Pharma is a neuroscience company developing novel drugs targeting neurodegeneration. Its portfolio focuses on preclinical stage therapies designed to uncouple the physiological function of glutamate-gated NMDA receptors from their toxic effects, aiming to stop neurodegeneration and improve brain health.

Fore Biotherapeutics is a precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Orionis Biosciences is a biotechnology company focused on early-stage drug discovery and development. It specializes in innovative medicines for diseases with high unmet medical needs, initially concentrating on oncology and immunotherapies. The company operates research facilities in Waltham, MA, USA, and Ghent, Belgium, led by an experienced team of entrepreneurs and scientists.

Splice Bio is a biotechnology company based in Barcelona, Spain, that specializes in developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012, the company focuses on addressing the needs of patients suffering from incurable genetic diseases. Splice Bio's technology, originally developed in the Muir Lab at Princeton University, enhances the capabilities of traditional adeno-associated viruses (AAVs) by allowing for larger gene cargo delivery and targeting a broader range of tissues. This advancement aims to overcome existing limitations in gene therapy, making it a promising solution for treating complex genetic disorders. The company was formerly known as Proteodesign, S.L. before rebranding to Splice Bio in September 2020.

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 that specializes in developing engineered regulatory T cells, known as 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, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

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 precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

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 that specializes in developing engineered regulatory T cells, known as 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, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic 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.

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 is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.

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 based in Paris, France, focused on the research, development, and commercialization of gene therapy treatments for inherited liver disorders and orphan diseases. Founded in 2016, the company utilizes a liver-tropic adeno-associated virus (AAV) to deliver therapeutic genes directly to hepatocytes, aiming to correct genetic disorders at the source. Vivet is particularly engaged in developing innovative solutions for conditions such as Wilson disease, Progressive Familial Intrahepatic Cholestasis (PFIC), and Citrullinemia Type I (CTLN1). To enhance its gene therapy approaches, Vivet collaborates with the Fundación para la Investigación Médica Aplicada at the Centro de Investigación Médica Aplicada, focusing on optimizing AAV vectors for targeted liver delivery and ensuring long-term expression of therapeutic genes.

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 develops cellular engineering solutions based on CRISPR technologies, offering a gene editing platform with applications in human therapeutics, disease modeling, genomics research, and agricultural biotechnology.

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 is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

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 is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

Caribou Biosciences develops cellular engineering solutions based on CRISPR technologies, offering a gene editing platform with applications in human therapeutics, disease modeling, genomics research, and agricultural biotechnology.

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 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.

GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

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.

Proteostasis Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

Okairos AG is a biopharmaceutical company headquartered in Basel, Switzerland, with operations in Rome and Naples, Italy. The company specializes in the discovery and development of genetic T-cell vaccines, focusing on conditions such as hepatitis C virus. Okairos also excels in gene delivery technologies, providing expertise in scalable viral-vector manufacturing processes. This enables other biotech firms to transition genetic vaccines and medicinal products from preclinical stages to commercial readiness, utilizing advanced good manufacturing practice (GMP) facilities. Previously a subsidiary of Istituto Di Ricerche Di Biologia Molecolare P. Angeletti Spa, Okairos is dedicated to advancing innovative therapeutic solutions in the healthcare sector.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

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 precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.