OCU400

Nanoscope's Optogenetic Gene Therapy Shows Promise in Retinitis Pigmentosa Clinical Trials

2 months ago

• Nanoscope Therapeutics' MCO-010 gene therapy demonstrated clinically meaningful vision improvements in 16 out of 18 patients with severe retinitis pigmentosa in a Phase 2 trial, compared to only four of nine in the placebo group. • The mutation-agnostic therapy uses a synthetic opsin (MCO protein) delivered via intravitreal injection to restore light-detecting function in damaged retinal cells, potentially benefiting patients regardless of their specific genetic mutation. • Following treatment, patients showed significant improvements in vision-guided mobility and object recognition, with some experiencing "transformative" results while maintaining a favorable safety profile with no serious adverse events reported.

Phase 3 Trial Shows Promising Results for Novel Encapsulated Cell Therapy in MacTel Treatment

3 months ago

• Phase 3 clinical trials of NT-501, an encapsulated cell therapy delivering CNTF, demonstrated significant reduction in disease progression for macular telangiectasia type 2 patients, with up to 52% reduction in ellipsoid zone loss. • The innovative implantable device, developed by Neurotech, maintains long-term viability with CNTF production documented for up to 14.5 years, offering a potential alternative to frequent intravitreal injections. • FDA review of the therapy is currently underway with a PDUFA date set for March 18, 2025, marking a potential breakthrough in MacTel treatment.

ViGeneron's VG901 Receives FDA Rare Pediatric Disease Designation and Advances to Dose Escalation in Phase 1b Trial for Retinitis Pigmentosa

4 months ago

• The FDA granted Rare Pediatric Disease Designation to ViGeneron's VG901, a gene therapy for retinitis pigmentosa caused by CNGA1 gene mutations, highlighting the urgent need for new treatments. • VG901 leverages ViGeneron's vgAAV capsid to deliver the functional CNGA1 gene to retinal photoreceptor cells via intravitreal injection, offering a less invasive approach. • The Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in the ongoing Phase 1b clinical trial, indicating promising early safety results for VG901. • ViGeneron's VG801 receives FDA IND clearance for mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal diseases associated with mutations in the ABCA4 gene.

Gameto's Fertilo Receives FDA Clearance for Phase 3 iPSC Fertility Trial

5 months ago

• Gameto's Fertilo, an iPSC-based therapy, has received FDA IND clearance for a Phase III trial, marking a significant advancement in reproductive medicine. • The Fertilo technology uses engineered ovarian support cells to mature eggs outside the body, reducing hormone injections by 80% and shortening treatment cycles. • The Phase III trial will assess the safety and efficacy of Fertilo, focusing on embryo development, pregnancy rates, and maternal health across 15 US sites. • Fertilo achieved the world's first live birth using its technology in December 2024 and has secured regulatory clearance in multiple countries.

Ocugen's OCU410 ArMaDa Trial Proceeds to Phase 2 for Geographic Atrophy

5 months ago

• The Data and Safety Monitoring Board (DSMB) has approved the continuation of Phase 2 in Ocugen's OCU410 ArMaDa clinical trial for geographic atrophy (GA). • Initial Phase 2 data indicates OCU410 is safe and well-tolerated, with no serious adverse events reported among the 15 subjects assessed. • OCU410, a novel modifier gene therapy, is administered via a single subretinal injection, potentially reducing the treatment burden compared to current therapies. • The ArMaDa trial is ongoing across 13 U.S. centers, with dosing expected to complete in early 2025 and further efficacy updates to be provided.

Ocugen's OCU400 Shows Promise in Retinitis Pigmentosa Treatment with Positive Clinical Data and Regulatory Milestones

5 months ago

• Ocugen's OCU400 gene therapy demonstrated a statistically significant improvement in low-luminance visual acuity in patients with retinitis pigmentosa (RP). • The European Medicines Agency (EMA) has granted OCU400 an Advanced Therapy Medicinal Product (ATMP) classification, expediting its regulatory review process. • Phase 1/2 trial results showed that 100% of treated patients experienced improved or preserved visual function over two years, regardless of the underlying genetic mutation. • Ocugen's Phase 3 liMeliGhT clinical trial is ongoing, with plans for BLA/MAA submissions in the U.S. and Europe anticipated in the first half of 2026.

Ocugen's Gene-Agnostic Therapy OCU400 Shows Promise in Retinitis Pigmentosa Phase III Trial

6 months ago

• Ocugen's OCU400, a gene-agnostic modifier gene therapy, is currently in Phase III trials for retinitis pigmentosa (RP), targeting a broad range of genetic mutations. • The Phase III study involves 150 patients randomized to receive OCU400 or remain as untreated controls, with the primary endpoint being improvement in mobility. • OCU400 has received orphan drug designation from both the FDA and EMA for RP and LCA, along with RMAT designation from the FDA, highlighting its potential clinical benefit. • Ocugen anticipates completing Phase III enrollment by early next year and aims to submit BLA and marketing authorization applications in 2026.

Ocugen's Gene Therapy Programs Advance in Clinical Trials, Cash Runway Extended into Q1 2026

6 months ago

• Ocugen's OCU400 Phase 3 liMeliGhT trial for retinitis pigmentosa is on track to complete enrollment in the first half of 2025, potentially reaching a broader patient population. • The OCU410 Phase 1/2 ArMaDa clinical trial is actively recruiting patients for Phase 2, evaluating its safety and efficacy in treating geographic atrophy. • OCU410ST GARDian clinical trial's DSMB approved enrollment for the second phase of the Phase 1/2 clinical trial, with preliminary data to be presented soon. • Recent debt and equity financings have extended Ocugen's cash runway into the first quarter of 2026, supporting the advancement of its ophthalmology pipeline.

Ocugen Advances Ophthalmology Pipeline with Positive Clinical and Regulatory Milestones

6 months ago

• Ocugen received Health Canada approval to initiate the Phase 3 OCU400 liMeliGhT trial and FDA approval for an Expanded Access Program for OCU400 in Retinitis Pigmentosa. • Phase 1 trial initiated for OCU200 in Diabetic Macular Edema, addressing a significant unmet need with potential for non-responders to current therapies. • OCU410 and OCU410ST trials progressing, with preliminary safety and efficacy data for geographic atrophy and Stargardt disease to be presented soon. • Ocugen secured $30 million in debt financing, extending the company's financial runway into the first quarter of 2026, supporting ongoing clinical programs.

Ocugen's Gene Therapy Programs Advance in Clinical Trials, Bolstered by New Financing

6 months ago

• Ocugen's OCU400 Phase 3 liMeliGhT trial for retinitis pigmentosa is progressing as planned, with enrollment expected to complete in the first half of 2025. • The OCU410 Phase 1/2 ArMaDa clinical trial is actively recruiting patients for Phase 2, evaluating its potential in treating geographic atrophy. • OCU410ST GARDian clinical trial's DSMB has approved the enrollment for the second phase, focusing on Stargardt disease. • Ocugen secured $30 million in debt financing, extending the company's cash runway into the first quarter of 2026 to support its ophthalmology pipeline.

Ocugen's OCU410ST Gene Therapy Advances to Phase 2 for Stargardt Disease

7 months ago

• Ocugen's OCU410ST, a gene therapy for Stargardt disease, has been cleared to advance to Phase 2 of the GARDian clinical trial after a positive safety review. • The Data and Safety Monitoring Board (DSMB) found OCU410ST safe and well-tolerated in Phase 1, with no serious adverse events reported among treated patients. • Phase 2 will utilize the high and medium doses of OCU410ST, with hopes of addressing the unmet medical need for the estimated 100,000 Stargardt patients in the US and Europe. • OCU410ST delivers the _RORA_ gene via an AAV5 vector, potentially modifying key disease pathways like lipofuscin formation and oxidative stress.

Ocugen's OCU410ST Advances to Phase 2 for Stargardt Disease Following Positive Safety Data

7 months ago

• The Data and Safety Monitoring Board (DSMB) has approved Ocugen's OCU410ST to advance to Phase 2 clinical trials for Stargardt disease. • Phase 1 results showed OCU410ST was well-tolerated with no serious adverse events reported among the nine patients enrolled. • OCU410ST is a novel modifier gene therapy using an AAV delivery platform to deliver the RORA gene to address Stargardt disease. • Stargardt disease, affecting approximately 100,000 individuals in the U.S. and Europe, currently lacks FDA-approved treatments.

Ocugen's OCU400 Gene Therapy Shows Promise in Retinitis Pigmentosa Phase 1/2 Trial

8 months ago

• OCU400, a gene-agnostic therapy, demonstrated a favorable safety profile in a Phase 1/2 trial for retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). • Approximately 60% of RP patients in the Phase 1/2 study showed improvement in mobility, assessed via luminance-dependent navigation, after OCU400 treatment. • Ocugen's OCU400 has received orphan drug designation from the FDA and EMA for RP and LCA, and is currently in Phase 3 trials. • The Phase 3 trial aims to enroll 150 patients with RP, evaluating OCU400's efficacy across a broad range of genetic mutations.

FDA Approves Expanded Access Program for Ocugen's OCU400 in Retinitis Pigmentosa

10 months ago

• The FDA has approved Ocugen's expanded access program (EAP) for OCU400, a modifier gene therapy, for adults with retinitis pigmentosa (RP). • The EAP will allow eligible RP patients with unmet medical needs to access OCU400 outside of the ongoing Phase 3 liMeliGhT clinical trial. • OCU400 targets multiple gene mutations associated with RP by resetting dysfunctional gene networks in retinal cells to restore cellular homeostasis. • Ocugen anticipates potential BLA and MAA approval for OCU400 in 2026, with Phase 3 trial enrollment expanded to include diverse RP gene mutations.

Drug Development Updates