Sofinnova Partners

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing innovative therapeutic products for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts are dedicated to addressing disabling symptoms associated with conditions affecting the nervous system, such as Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma aims to enhance the quality of life and improve patient outcomes for those suffering from these challenging disorders.

nChroma Bio is a biotechnology company specializing in targeted genetic medicine. It operates an integrated product engine for safe, accurate, and targeted in vivo administration, aiming to overcome limitations of current genetic medicine techniques. The company focuses on liver-targeted therapies, initially for chronic hepatitis B and D, leveraging epigenetics to enable precise and specific gene regulation. This approach allows for therapies with programmable tissue specificity, potentially offering durable and targeted cures for the liver and other organs.

Meiogenix is a biotechnology company focused on developing innovative breeding technologies aimed at harnessing the untapped genetic diversity of various organisms. By modulating homologous recombination frequencies in eukaryotic cells, Meiogenix enhances natural biodiversity and facilitates the creation of new products. This approach is designed to help businesses tackle pressing global food and industrial challenges, ultimately contributing to sustainable solutions in these sectors.

Purespring Therapeutics is a biotechnology company specializing in the development of gene therapies for various kidney diseases. It focuses on targeting podocytes, specialized cells within the kidneys, using adeno-associated virus (AAV) vectors. The company aims to address unmet needs in renal conditions and has established an in-vivo functional screening platform to identify protective factors that could potentially benefit multiple kidney diseases.

Genespire is a biotechnology company dedicated to developing innovative gene therapies for patients suffering from genetic diseases, with a particular emphasis on primary immunodeficiencies and inherited metabolic disorders. The company specializes in creating therapies that utilize integration-defective lentiviral vectors combined with gene editing techniques. This approach allows for highly effective and adaptable targeted insertion of therapeutic genes into blood cells, providing patients with a range of tailored therapeutic options suited to their specific conditions. By focusing on transformative solutions, Genespire aims to significantly improve the lives of individuals affected by severe genetic disorders.

Micropep Technologies is an agricultural biotechnology company founded in 2016 and based in Toulouse, France. The company specializes in the development of non-GMO bioherbicides and biostimulants utilizing its innovative miPEP technology, which harnesses naturally-occurring micro-peptides to regulate gene expression and enhance plant growth. Micropep focuses on four key development programs: germination, flowering, growth, and weed control, aiming to improve agricultural yields while maintaining the integrity of plant DNA. By leveraging the potential of these biomimetic peptides, Micropep seeks to provide effective solutions for managing plant genetics and enhancing resistance to diseases, thereby supporting sustainable farming practices.

Cure51 is a company focused on advancing cancer treatment through the development of a clinical and molecular database. Its primary mission is to create a comprehensive database of cancer survivors, utilizing their unique biological characteristics to identify potential drug targets. By analyzing the biology of long-term cancer survivors, Cure51 aims to aid healthcare researchers in decoding genetic information derived from biopsies, blood tests, imaging, and other samples. This innovative approach seeks to accelerate drug discovery, ultimately contributing to the formulation of new and effective cancer therapies.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

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.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

AAVantgarde Bio is a biotechnology company focused on developing gene therapies specifically for inherited retinal disorders. The company utilizes proprietary Adeno-Associated Viral (AAV) vector platforms to overcome the limitations of traditional AAV vectors, particularly in terms of cargo capacity. This innovative approach allows for the delivery of large genes to both ocular and non-ocular tissues, facilitating effective in vivo treatment options for patients suffering from these conditions. Through its advanced technology, AAVantgarde Bio aims to enhance therapeutic outcomes and improve the quality of life for individuals affected by genetic eye diseases.

Alia Therapeutics is focused on the development of next-generation gene-editing medicines aimed at curing rare genetic diseases. The company employs innovative CRISPR-based genome editing tools that allow for the correction of inborn genetic errors directly within patients' cells. By utilizing unique therapeutic approaches, Alia Therapeutics seeks to address genetic disorders at their root cause, enabling healthcare professionals to modify genomes effectively. Additionally, the company emphasizes the importance of safe and efficient delivery methods for these gene-editing solutions to target cells, positioning itself at the forefront of advancements in the treatment of genetic diseases.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing innovative therapeutic products for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts are dedicated to addressing disabling symptoms associated with conditions affecting the nervous system, such as Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma aims to enhance the quality of life and improve patient outcomes for those suffering from these challenging disorders.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Micropep Technologies is an agricultural biotechnology company founded in 2016 and based in Toulouse, France. The company specializes in the development of non-GMO bioherbicides and biostimulants utilizing its innovative miPEP technology, which harnesses naturally-occurring micro-peptides to regulate gene expression and enhance plant growth. Micropep focuses on four key development programs: germination, flowering, growth, and weed control, aiming to improve agricultural yields while maintaining the integrity of plant DNA. By leveraging the potential of these biomimetic peptides, Micropep seeks to provide effective solutions for managing plant genetics and enhancing resistance to diseases, thereby supporting sustainable farming practices.

Meiogenix is a biotechnology company focused on developing innovative breeding technologies aimed at harnessing the untapped genetic diversity of various organisms. By modulating homologous recombination frequencies in eukaryotic cells, Meiogenix enhances natural biodiversity and facilitates the creation of new products. This approach is designed to help businesses tackle pressing global food and industrial challenges, ultimately contributing to sustainable solutions in these sectors.

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.

Micropep Technologies is an agricultural biotechnology company founded in 2016 and based in Toulouse, France. The company specializes in the development of non-GMO bioherbicides and biostimulants utilizing its innovative miPEP technology, which harnesses naturally-occurring micro-peptides to regulate gene expression and enhance plant growth. Micropep focuses on four key development programs: germination, flowering, growth, and weed control, aiming to improve agricultural yields while maintaining the integrity of plant DNA. By leveraging the potential of these biomimetic peptides, Micropep seeks to provide effective solutions for managing plant genetics and enhancing resistance to diseases, thereby supporting sustainable farming practices.

AAVantgarde Bio is a biotechnology company focused on developing gene therapies specifically for inherited retinal disorders. The company utilizes proprietary Adeno-Associated Viral (AAV) vector platforms to overcome the limitations of traditional AAV vectors, particularly in terms of cargo capacity. This innovative approach allows for the delivery of large genes to both ocular and non-ocular tissues, facilitating effective in vivo treatment options for patients suffering from these conditions. Through its advanced technology, AAVantgarde Bio aims to enhance therapeutic outcomes and improve the quality of life for individuals affected by genetic eye diseases.

Alia Therapeutics is focused on the development of next-generation gene-editing medicines aimed at curing rare genetic diseases. The company employs innovative CRISPR-based genome editing tools that allow for the correction of inborn genetic errors directly within patients' cells. By utilizing unique therapeutic approaches, Alia Therapeutics seeks to address genetic disorders at their root cause, enabling healthcare professionals to modify genomes effectively. Additionally, the company emphasizes the importance of safe and efficient delivery methods for these gene-editing solutions to target cells, positioning itself at the forefront of advancements in the treatment of genetic diseases.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing innovative therapeutic products for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts are dedicated to addressing disabling symptoms associated with conditions affecting the nervous system, such as Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma aims to enhance the quality of life and improve patient outcomes for those suffering from these challenging disorders.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

Genespire is a biotechnology company dedicated to developing innovative gene therapies for patients suffering from genetic diseases, with a particular emphasis on primary immunodeficiencies and inherited metabolic disorders. The company specializes in creating therapies that utilize integration-defective lentiviral vectors combined with gene editing techniques. This approach allows for highly effective and adaptable targeted insertion of therapeutic genes into blood cells, providing patients with a range of tailored therapeutic options suited to their specific conditions. By focusing on transformative solutions, Genespire aims to significantly improve the lives of individuals affected by severe genetic disorders.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

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.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

Micropep Technologies is an agricultural biotechnology company founded in 2016 and based in Toulouse, France. The company specializes in the development of non-GMO bioherbicides and biostimulants utilizing its innovative miPEP technology, which harnesses naturally-occurring micro-peptides to regulate gene expression and enhance plant growth. Micropep focuses on four key development programs: germination, flowering, growth, and weed control, aiming to improve agricultural yields while maintaining the integrity of plant DNA. By leveraging the potential of these biomimetic peptides, Micropep seeks to provide effective solutions for managing plant genetics and enhancing resistance to diseases, thereby supporting sustainable farming practices.

ProQR Therapeutics N.V. is a biopharmaceutical company based in Leiden, the Netherlands, focused on the discovery and development of RNA-based therapeutics for genetic disorders. Founded in 2012, the company targets severe rare diseases, including Leber's congenital amaurosis type 10 and Usher syndrome type 2. ProQR's leading product candidates include sepofarsen, currently in a phase II/III clinical trial for Leber's congenital amaurosis 10, QR-421a in a phase 1/2 trial for Usher syndrome type 2 and non-syndromic retinitis pigmentosa, QR-1123 in a phase 1/2 trial for autosomal dominant retinitis pigmentosa, and QR-504a, which is undergoing its first clinical trial for Fuchs endothelial corneal dystrophy. The company has established license agreements with several prominent medical institutions, including General Hospital Corporation and Radboud University Medical Center.

ProQR Therapeutics N.V. is a biopharmaceutical company based in Leiden, the Netherlands, focused on the discovery and development of RNA-based therapeutics for genetic disorders. Founded in 2012, the company targets severe rare diseases, including Leber's congenital amaurosis type 10 and Usher syndrome type 2. ProQR's leading product candidates include sepofarsen, currently in a phase II/III clinical trial for Leber's congenital amaurosis 10, QR-421a in a phase 1/2 trial for Usher syndrome type 2 and non-syndromic retinitis pigmentosa, QR-1123 in a phase 1/2 trial for autosomal dominant retinitis pigmentosa, and QR-504a, which is undergoing its first clinical trial for Fuchs endothelial corneal dystrophy. The company has established license agreements with several prominent medical institutions, including General Hospital Corporation and Radboud University Medical Center.

HAYA Therapeutics is a company focused on developing RNA-based therapeutics aimed at treating heart failure and other fibrotic diseases. The company specializes in biopharmaceutical therapies that target specific regulators of fibrosis, particularly long noncoding RNA, which plays a critical role in myocardial fibrosis. By honing in on heart-specific mechanisms, HAYA Therapeutics seeks to enhance the efficacy and safety of treatments for heart-related illnesses and other serious conditions associated with aging, including cancer. Through its innovative approach, the company aims to improve patient outcomes in the management of heart failure and related health challenges.

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.