Some eyes carry genetic protection against degeneration. Perceive Biotherapeutics is trying to turn that advantage into a lasting treatment - and its next test is whether the biology can slow geographic atrophy.
Neurotech Pharmaceuticals is a Cumberland, Rhode Island biotechnology company that develops long-acting therapies for chronic retinal diseases using its Encapsulated Cell Technology (ECT) platform - a tiny implant of genetically engineered cells that continuously secretes a therapeutic protein inside the eye. In March 2025 its lead product ENCELTO (revakinagene taroretcel-lwey, formerly NT-501/Renexus) became the first and only FDA-approved treatment for macular telangiectasia type 2 (MacTel), delivering ciliary neurotrophic factor (CNTF) to slow the loss of photoreceptors.
ProJenX is a New York City clinical-stage biotechnology company developing prosetin, a first-in-class, oral, brain-penetrant MAP4K inhibitor for amyotrophic lateral sclerosis (ALS) and other neurodegenerative brain diseases. Spun out of a two-decade collaboration between the nonprofit Project ALS and Columbia University researchers, ProJenX is built on the discovery that inhibiting MAP4 kinases protects motor neurons across multiple patient-derived stem cell models of ALS. The company is running the PRO-101 Phase 1b trial in people living with ALS.
Noveome Biotherapeutics is a Pittsburgh clinical-stage biopharmaceutical company built around ST266, a multi-targeted secretome of hundreds of biologically active proteins harvested from a novel population of amnion-derived cells. Rather than transplanting cells, Noveome delivers the healing signals those cells secrete - aiming to modulate inflammation, protect nerves, and accelerate tissue repair. Its lead program treats necrotizing enterocolitis, a devastating gut disease in premature infants, with additional pipeline work spanning ophthalmology, neurology, and dermatology.
Nura Bio is a clinical-stage biopharmaceutical company in South San Francisco developing brain-penetrant, small-molecule neuroprotective medicines. Its work centers on SARM1, an injury-activated NAD hydrolase that triggers axon degeneration, an early and common event across many neurological diseases. The lead candidate, NB-4746, is an oral, brain-penetrant SARM1 inhibitor that completed Phase 1 in healthy volunteers and shows protection in preclinical models of ALS, MS, traumatic brain injury and chemotherapy-induced peripheral neuropathy. The company has raised more than $140 million in Series A financing led by The Column Group.