Sofinnova Investments

Atsena Therapeutics is a clinical-stage company focused on developing gene therapies for inherited retinal diseases. Utilizing an adeno-associated virus (AAV) platform, including a novel spreading capsid, Atsena's clinical programs aim to prevent blindness caused by genetic mutations.

BioAge Labs develops medicines to treat aging and age-related diseases using its proprietary BioAge platform, which combines systems biology and AI. Its pipeline includes BGE-117 for multiple aging-related conditions, BGE-175 for immune aging and respiratory infections, and azelaprag for obesity in the elderly.

Quanta Therapeutics is a biotechnology company focused on developing novel cancer therapeutic candidates. It specializes in allosteric modulation to directly inhibit RAS-driven cancer types by restoring conformational control of active RAS signaling at the cell membrane.

Quanta Therapeutics is a biotechnology company focused on developing novel cancer therapeutic candidates. It specializes in allosteric modulation to directly inhibit RAS-driven cancer types by restoring conformational control of active RAS signaling at the cell membrane.

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.

XyloCor Therapeutics, Inc. is a biopharmaceutical company specializing in the development of gene therapies for cardiovascular diseases, particularly advanced coronary artery disease. Founded in 2013 and based in Newtown Square, Pennsylvania, the company focuses on innovative treatments aimed at addressing significant unmet medical needs. Its lead product candidate, XC001, is currently in clinical development for patients suffering from refractory angina, targeting individuals who have exhausted other treatment options. XyloCor's approach centers on stimulating the formation of new coronary blood vessels to enhance blood supply to areas of the heart that are inadequately perfused, thereby improving patient outcomes in those affected by heart failure and ischemic conditions. The company collaborates with Weill Cornell Medical College to advance its gene therapy technologies.

Vera Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2016 and formerly known as Trucode Gene Repair, Inc. The company focuses on developing innovative biologic therapeutics with the potential to transform treatment for patients suffering from serious immunological diseases and genetic disorders. Its lead product candidate, atacicept, is a fusion protein administered as a subcutaneous injection once weekly, designed to inhibit factors that stimulate the production of autoantibodies associated with autoimmune diseases. Additionally, Vera Therapeutics is utilizing gene-editing technology aimed at addressing conditions such as sickle cell disease and cystic fibrosis, striving to enhance gene detection and regulation for improved patient outcomes.

Atsena Therapeutics is a clinical-stage company focused on developing gene therapies for inherited retinal diseases. Utilizing an adeno-associated virus (AAV) platform, including a novel spreading capsid, Atsena's clinical programs aim to prevent blindness caused by genetic mutations.

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.

Vera Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2016 and formerly known as Trucode Gene Repair, Inc. The company focuses on developing innovative biologic therapeutics with the potential to transform treatment for patients suffering from serious immunological diseases and genetic disorders. Its lead product candidate, atacicept, is a fusion protein administered as a subcutaneous injection once weekly, designed to inhibit factors that stimulate the production of autoantibodies associated with autoimmune diseases. Additionally, Vera Therapeutics is utilizing gene-editing technology aimed at addressing conditions such as sickle cell disease and cystic fibrosis, striving to enhance gene detection and regulation for improved patient outcomes.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

XyloCor Therapeutics, Inc. is a biopharmaceutical company specializing in the development of gene therapies for cardiovascular diseases, particularly advanced coronary artery disease. Founded in 2013 and based in Newtown Square, Pennsylvania, the company focuses on innovative treatments aimed at addressing significant unmet medical needs. Its lead product candidate, XC001, is currently in clinical development for patients suffering from refractory angina, targeting individuals who have exhausted other treatment options. XyloCor's approach centers on stimulating the formation of new coronary blood vessels to enhance blood supply to areas of the heart that are inadequately perfused, thereby improving patient outcomes in those affected by heart failure and ischemic conditions. The company collaborates with Weill Cornell Medical College to advance its gene therapy technologies.

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.

Quanta Therapeutics is a biotechnology company focused on developing novel cancer therapeutic candidates. It specializes in allosteric modulation to directly inhibit RAS-driven cancer types by restoring conformational control of active RAS signaling at the cell membrane.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Spark Therapeutics focuses on developing potentially curative, one-time gene therapy products to treat debilitating genetic diseases. Its pipeline includes LUXTURNA for inherited retinal disease, SPK-FIX for hemophilia B (in collaboration with Pfizer), and preclinical programs targeting neurodegenerative diseases and other retinal degenerative conditions.

Lysogene S.A. is a biotechnology company based in Neuilly-sur-Seine, France, focused on the research and clinical development of gene therapy for neurodegenerative disorders. Founded in 2009, the company is advancing several clinical programs, including LYS-SAF302, currently in Phase II/III trials for the treatment of mucopolysaccharidosis type IIIA, and LYS-GM101, which is in Phase I/II trials for GM1 gangliosidosis. Lysogene is also engaged in a collaborative research effort with the Weizmann Institute of Science to develop a novel AAV gene therapy approach targeting neuronopathic Gaucher disease, Parkinson's disease, and other conditions. The company aims to enhance the health outcomes of patients suffering from life-threatening, incurable disorders by utilizing AAV vectors that effectively deliver genetic material to the central nervous system.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

IVERIC bio is a biopharmaceutical company dedicated to discovering and developing innovative treatments for retinal diseases that have significant unmet medical needs. The company is actively working on therapeutic product candidates aimed at age-related retinal diseases, as well as gene therapy product candidates designed for orphan inherited retinal diseases. By focusing on these areas, IVERIC bio aims to address critical gaps in the current treatment landscape for patients suffering from various retinal conditions.

DiObex, Inc. operates as a biotechnology company in the United States. The company develops novel products for the treatment of metabolic disorders, including type-one and type-two diabetes, dyslipidemia, obesity, and diabetic nephropathy. Its products include DIO-901, a very low dose glucagon product that is developed for the reduction of nocturnal hypoglycemia in patients with type-one diabetes; and DIO-902, a cortisol synthesis inhibitor that is developed as a therapy for glucose and cholesterol control in patients with type-two diabetes. DiObex, Inc. was founded in 2003 and is based in San Francisco, California.

DiObex, Inc. operates as a biotechnology company in the United States. The company develops novel products for the treatment of metabolic disorders, including type-one and type-two diabetes, dyslipidemia, obesity, and diabetic nephropathy. Its products include DIO-901, a very low dose glucagon product that is developed for the reduction of nocturnal hypoglycemia in patients with type-one diabetes; and DIO-902, a cortisol synthesis inhibitor that is developed as a therapy for glucose and cholesterol control in patients with type-two diabetes. DiObex, Inc. was founded in 2003 and is based in San Francisco, California.