- Category: Pipeline
- Tags: inherited retinal disease
Pipeline
FDA grants Orphan Drug designation to BlueRock's RP cell therapy
Marks second designation for OpCT-001; includes tax credits for qualified clinical testing and, if approved, 7 years of U.S. market exclusivity.
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PolyActiva and RareSight to advance long-acting therapies for pediatric IRDs
Collaboration leverages PolyActiva’s PREZIA drug delivery platform to develop NCE-enabling pharmacologic drugs for early-onset vision loss.
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FDA clears Opus Genetics' IND for BEST1 IRD gene therapy
Company plans to initiate phase 1/2 clinical trial on OPGx-BEST1, a potential first-ever treatment for this patient base.
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Kiora's KIO-104 receives US patent for expanded protection of inflammatory retinal diseases
The investigational small molecule’s intellectual property and potential market exclusivity are now protected through 2043.
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Boehringer partners with Re-Vana on extended-release ophthalmic therapeutics
Collaboration involves photo-crosslinked, biodegradable implants designed to enable continuous, 3-to-12-month drug delivery to treat retinal diseases.
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FDA OKs AAVantgarde's IND for Stargardt gene therapy trial
Clearance enables initiation of FIH study on AAVB-039, an AAV8 that delivers the full-length ABCA4 gene to address the disease’s root cause—enabling treatment for all types.
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Nona Biosciences and Kodak Sciences to advance antibody therapies for ophthalmic diseases
Kodiak to further develop retinal disease asserts via Nona’s Harbour Mice platform, designed to generate fully human monoclonal antibodies at half the size of regular IgG.
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Alkeus receives two rare disease FDA designations for Stargardt candidate
New statuses join gildeuretinol’s (ALK-001) previous Fast Track and Orphan Drug designations; gives company potential eligibility for priority review voucher.
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FDA grants IND clearance for BlueRock's primary photoreceptor disease cell therapy
New status paves the way for a phase 1/2a trial to evaluate the safety and efficacy of an induced pluripotent cell-derived candidate.
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Beacon Therapeutics to evaluate patented AAV gene therapy for retinal diseases
Licensing agreement involves potential development and commercialization of Abeona Therapeutics’ patented capsid, a pan-retinal-administered vector.
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Neurotech Pharma earns BLA priority review for MacTel cell therapy
Investigational ocular implant is designed to slow progression of gradual central vision deterioration in rare neurodegenerative retinal disease.
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SalioGen Therapeutics's gene coding therapy targets Stargardt disease
One-time, non-viral subretinal injection uses novel Gene Coding technology for gene integration.
Pipeline
PulseSight Therapeutics launches with focus on non-viral gene therapy for retinal diseases
Ophthalmic biotech company features a proprietary tech platform for treating wet AMD and dry AMD.
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Théa acquires exclusive rights to Kiora's IRD therapy
Commercialization agreement involves clinical development of KIO-301 for retinitis pigmentosa.
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Mireca Medicines granted $1M for inherited retinal disease advancements
Company receives Fighting Foundation Blindness’s Translational Research Acceleration Program Award for its lead investigational asset: MM238.
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FDA grants ODD to HuidaGene’s IRD gene therapy
Multinational trial will assess HG004 for adult and pediatric patients with IRDs linked to RPE65 mutations.
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Eyebiotech Limited introduces first retinal disease candidate
Restoret, the company’s lead asset, is designed to target retinal diseases characterized by vascular leakage via intravitreal injection.
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