Touting what’s been so far the “cleanest safety profile that’s been presented with genetic medicine in the eye,” deemed “a game changer for this field,” 4D Molecular Therapeutics Inc. CEO David Kirn said phase III planning will begin in later this year to test gene therapy 4D-150 in wet age-related macular degeneration (AMD).
Therini Bio Inc. has closed a US$36 million series A financing round that will support its work on developing fibrin-targeted therapies for diseases driven by chronic inflammation, including Alzheimer’s disease, multiple sclerosis and retinal diseases such as diabetic macular edema.
Ocugen Inc.’s OCU-410ST (AAV5-hRORA) has been awarded orphan drug designation by the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19) and cone-rod dystrophy 3 (CORD3) diseases.
In research presented this week at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, investigators from Life Biosciences Inc. reported using epigenetic reprogramming in a nonarteritic anterior ischemic optic neuropathy (NAION) model in nonhuman primates (NHPs).
Stoke Therapeutics Inc. has received clinical trial application (CTA) clearance from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study (OSPREY) of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA).
Nearly everyone with diabetes for >20 years will develop diabetic retinopathy or uveitis, which are collectively among the most common causes of premature blindness. Melanocortin is a short peptide that not only controls pigmentation, but that is most importantly expressed constitutively in the intraocular space where it serves essential functions suppressing inflammation in the retina, ultimately preventing blindness.
Complement Therapeutics GmbH raised €72 million (US$79.4 million) in a series A round to move into the clinic a novel gene therapy for treating geographic atrophy secondary to dry age-related macular degeneration. It’s the largest series A round completed in Europe so far this year.
PYC Therapeutics Ltd.’s lead candidate, VP-001, is entering the clinic, and the RNA therapy could be the first potential treatment for retinitis pigmentosa type 11, which causes blindness that begins in childhood and ultimately leads to legal blindness by middle age.
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