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.

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. Mechanistically, ALK-001 is engineered to reduce vitamin A dimer formation without visual-cycle modulation and carries multiple expedited FDA designations plus EMA orphan status; >400 patients treated to date.SHOW MORE 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).

Stargardt Disease. American Academy of Ophthalmology. Macula. American Academy of Ophthalmology. Vision Loss, Central. MedlinePlus. Stargardt macular degeneration. National Cancer Institute. Choroid. MedlinePlus. Choroideremia. National Organization of Rare Disorders. Choroideremia. U.S. Food & Drug Administration. FDA approves novel gene therapy to treat patients with a rare form of inherited ... Stargardt Disease. American Academy of Ophthalmology. Macula. American Academy of Ophthalmology. Vision Loss, Central. MedlinePlus. Stargardt macular degeneration. National Cancer Institute. Choroid. MedlinePlus. Choroideremia. National Organization of Rare Disorders. Choroideremia. U.S. Food & Drug Administration. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. This gene is associated with several IRDs, including retinitis pigmentosa. Other therapies in development include medications that prevent the death of cells in the eyes and prosthetics that are implanted into the eye to restore vision. Sources: PreventBlindness.org. Eye Diseases & Con... + 13 · PreventBlindness.org. Eye Diseases & Conditions: Inherited Retinal Diseases. Why family medical history matters to the health of your eyes.How your genetics can affect your vision ... Get a look at the healthcare providers you may work with if you are living with an inherited retinal disease (IRD).The specialists and therapies that treat IRDs Keep in mind that this is a simplified explanation of this condition—there are more than 60 different genes that can be involved in the development of RP, there are numerous subtypes of RP, the disease can follow different patterns, and it can occur as a result of multiple other genetic disorders. It’s estimated that RP affects one out of every 3,000 or 4,000 people. This inherited retinal disease is the most common form of juvenile macular degeneration and symptoms typically begin in late childhood or young adulthood.

City has secured nearly $100 million to advance its next-generation RNAi drugs, including a clotting disorder treatment currently in early human testing. An experimental therapy for Stargardt disease is also on the horizon.

A new study identifies key factors influencing vision-related quality of life in patients with recessive Stargardt disease. The findings could inform future therapeutic strategies and patient support initiatives.