Retinal Degeneration Fund

Perceive Pharma operates as a pharmaceutical company. Perceive Pharma develops innovative small molecule neuroprotective treatments.

NVasc is an early-stage ophthalmology company with a focus on treating blindness. It specializes in treating the leading causes of blindness through choroidal and retinal revascularization. The company has acquired the research rights to growth factors (NVB001 and NVB002) that have been shown to encourage the growth of non-leaking new vessels in the retina and choroid in mouse models of ischemic eye diseases. The company is planning to manufacture NVB001 which can be used in toxicokinetic studies and in early clinical trials in humans.

Amber Bio is a biotechnology company pioneering new gene editing modalities using multi-kilobase edits to reach previously undruggable patient populations.

Perceive Biotherapeutics is a biotechnology company focused on developing innovative ophthalmic therapeutics aimed at treating various eye diseases. The company leverages novel protective biologies to create a diverse pipeline of therapeutic assets that target significant causes of irreversible blindness. By utilizing multiple therapeutic modalities, Perceive Biotherapeutics seeks to provide medical professionals with effective solutions for improving eye health and preventing vision loss.

SparingVision SAS is a biotechnology company based in Paris, France, dedicated to discovering and developing gene therapy-based treatments for inherited retinal diseases that can lead to blindness. Founded in 2016, the company is focused on creating innovative therapies for conditions such as retinitis pigmentosa, the most prevalent form of inherited retinal degeneration, which currently lacks comprehensive treatment options. SparingVision is advancing its lead candidate, SPVN06, which aims to address the needs of the nearly 2 million individuals affected by this rare genetic disorder globally. By targeting the underlying causes of these diseases, SparingVision seeks to improve the quality of life for patients suffering from genetic retinal disorders.

Nacuity Pharmaceuticals, based in Fort Worth, Texas, is a biotechnology company focused on developing innovative treatments for retinitis pigmentosa and other ophthalmological disorders. Established in 2016, the company is dedicated to addressing ocular oxidative stress and aims to provide effective therapies for patients affected by various retinal diseases. Nacuity has garnered significant financial and technical backing from the Foundation Fighting Blindness, which has invested nearly $700 million over the past 46 years to support research into preventing and treating blinding retinal diseases. The Foundation is a key partner in advancing Nacuity’s development program, particularly through its commitment to fund a substantial portion of the company's Phase 1 and Phase 2 clinical trials via a milestone-based matching grant.

SalioGen Therapeutics is a biotechnology company focused on developing gene therapies for inherited disorders. Utilizing its proprietary Exact DNA Integration Technology (EDIT), the company employs mammal-derived enzymes, known as Saliogase, to facilitate genome engineering. This innovative approach enables the delivery of any size gene through non-viral methods, offering significant advantages in manufacturing and application. SalioGen's technology aims to provide durable, safe, and affordable treatments, potentially transforming the landscape of care for patients with genetic conditions and enhancing the efficiency of cell therapy and biologics manufacturing in the healthcare sector.

Opus Genetics is a gene therapy company focused on addressing challenges related to the manufacturing of treatments for ultra-rare retinal degenerative diseases. By utilizing adeno-associated virus-based gene therapies, Opus aims to provide effective solutions for individuals suffering from blinding conditions. The company's patient-first approach emphasizes the importance of accessibility to these specialized treatments, ensuring that advancements in gene therapy reach those in need efficiently.

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.

Atsena Therapeutics is a clinical-stage gene therapy company focused on developing treatments for inherited retinal diseases that can lead to blindness. Their clinical pipeline includes therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Atsena employs an adeno-associated virus (AAV) technology platform, including a novel spreading capsid, to specifically target these retinal disorders. The company aims to prevent vision loss caused by genetic mutations through innovative ocular gene therapy solutions. With a commitment to advancing treatments for some of the most challenging forms of inherited retinal disease, Atsena Therapeutics seeks to improve the lives of patients affected by these conditions.

Stargazer Pharmaceuticals Inc. is a biopharmaceutical company based in Boston, Massachusetts, founded in 2018. The company specializes in developing and manufacturing therapies for rare eye diseases, with a particular focus on Stargardt’s disease, a form of juvenile retinal degeneration. Currently operating in stealth mode, Stargazer Pharmaceuticals aims to provide novel treatment options for conditions that lack existing therapies, thus enabling healthcare professionals to better address the needs of patients with these rare diseases.