City Therapeutics will join a slew of biotechs that have entered the public markets this year, with a $200 million raise that landed above expectations. In addition, the biotech is conducting new drug-enabling studies for CITY-RBP4 in Stargardt disease, an inherited eye condition that causes vision loss in children and young adults. This program was to receive a $20 million boost from the IPO, according to the prospectus. Another $40 million will support clinical tests of CITY-TFR2 in anemia associated with myelofibrosis, while the remaining $70 million will help fund platform development and preclinical activities. City Therapeutics, the RNA interference biotech developing therapies for thromboembolic diseases, has pulled off an upsized IPO, raising $200 million that will be split across its pipeline. City will join a bustling group of biotechs that have joined the Nasdaq this year, including Aktis Oncology, Generate:Biomedicines and ADARx Pharmaceuticals. City’s $200 million raise falls in the middle of the pack, with a record high achieved this year by Parabilis Medicines’ $670 million IPO in June.

A single intravitreal injection improved acuity in Stargardt disease; a randomized trial in geographic atrophy is planned. A single intravitreal injection improved acuity in Stargardt disease; a randomized trial in geographic atrophy is planned. ... age-related macular degeneration (AMD) slow lesion growth but do not recover lost vision. Data presented at the 130th Annual Meeting of the American Academy of Ophthalmology (AAO) in New Orleans from October 9-12, 2026, suggest that an optogenetic gene therapy may eventually address that gap.1 The human data so far come from small, open-label cohorts with an inherited disease, not from older adults with AMD. Boyer said he had expected that the therapy's effect on remaining viable cells might obscure any acuity benefit, and that this did not happen.1 Whether that holds in eyes with GA—which typically retain more functioning photoreceptors at the lesion margins—is still unknown. Neither has been shown to improve visual acuity.5,6 And no approved therapy currently restores function to the retina, where photoreceptors have already been lost. Rather than using gene replacement, son-vec uses optogenetics. Through this treatment approach, an adeno-associated viral vector delivers a gene encoding a multi-characteristic opsin to ON bipolar cells, which often survive after photoreceptors degenerate; and the bipolar cells then respond directly to light.1,7 Because this approach does not target a specific causative mutation, it is described as mutation agnostic. The presentation highlights 2 lines of research.1 The first: an open-label, multicenter phase 2 study in which patients with Stargardt disease and visual acuity of 20/200 or worse each received a single injection of son-vec. According to the data, patients whose atrophy was limited to the macula gained an average of 12 Early Treatment Diabetic Retinopathy Study (ETDRS) letters. The therapy was well tolerated, with no serious adverse events (AEs) reported.1

BERKELEY, Calif.--(BUSINESS WIRE)--Ray ... therapies for those living with severe retinal degenerations, today announced it has been awarded an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to advance RTx-021 for the treatment of Stargardt disease. The funding will support the ongoing Phase 1/2 clinical trial and manufacturing ... BERKELEY, Calif.--(BUSINESS WIRE)--Ray Therapeutics (RayTx), an industry-leading clinical-stage biopharmaceutical company pioneering differentiated vision restoration therapies for those living with severe retinal degenerations, today announced it has been awarded an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to advance RTx-021 for the treatment of Stargardt disease. The funding will support the ongoing Phase 1/2 clinical trial and manufacturing activities to prepare the program for late-stage development. Stargardt disease is the most common form of juvenile macular degeneration, and about 40,000 people in the United States are living with the condition. It is caused by mutations that lead to photoreceptor loss over time, resulting in progressive loss of central vision. ... RayTx is a clinical-stage biopharmaceutical company advancing optogenetic gene therapies to restore vision in people with retinal degenerative diseases. The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state’s biotechnology economy, and expand access to transformative therapies. It is clear that Stargardt’s patients need life-changing treatments that can help them maintain their independence and navigate daily life with confidence,” said Paul Webb, PhD, Senior Science Officer for Clinical Development of CIRM. “CIRM’s mission is to help bring promising science to the patients who need it. We’re hopeful that this approach will advance a potential vision-restoring therapy for people in California and around the world who currently have no approved treatment options.”

Gildeuretinol has received Breakthrough Therapy, Rare Pediatric Disease, Fast Track and Orphan Drug designations for Stargardt disease from the FDA. The EMA has designated gildeuretinol as an orphan medicinal product to treat non-syndromic inherited retinal dystrophies due to defects in the ... Gildeuretinol has received Breakthrough Therapy, Rare Pediatric Disease, Fast Track and Orphan Drug designations for Stargardt disease from the FDA. The EMA has designated gildeuretinol as an orphan medicinal product to treat non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, which includes Stargardt disease. Alkeus Pharmaceuticals has dosed the first participant in the phase 3 NORTHSTAR trial of oral gildeuretinol for Stargardt disease, a condition with no approved treatments. NORTHSTAR is a randomized, placebo-controlled, double-masked, 24-month trial designed to evaluate the efficacy, safety, and pharmacokinetics of oral gildeuretinol in patients with advanced Stargardt disease who present with atrophic lesions at baseline. The study aims to enroll approximately 230 participants aged 8 to 45 years across an estimated 55 sites in more than 11 countries. The study design has been agreed upon by both the US Food and Drug Administration and the European Medicine Agency (EMA). Alkeus Pharmaceuticals in a press release1 announced dosing of the first participant in the NORTHSTAR study (NCT07419334), a pivotal phase 3 global trial evaluating oral gildeuretinol acetate (ALK-001) in patients with advanced Stargardt disease—a rare, progressive inherited retinal dystrophy for which no approved therapy currently exists in the US and Europe.

Tarsus Pharmaceuticals has acquired Alkeus for up to $800 million, gaining access to a promising therapy for Stargardt disease currently in late-stage testing. This acquisition highlights the potential value of innovative treatments in the rare eye disease space.