Interim safety and efficacy of gene therapy for RLBP1|associated ...

Interim safety and efficacy of gene therapy for RLBP1-associated ...

Gene therapy holds promise for treatment of inherited retinal dystrophies, a group of rare genetic disorders characterized by severe loss of ...

(PDF) Interim safety and efficacy of gene therapy for RLBP1 ...

Dark adaptation kinetics, the primary efficacy endpoint, improved significantly in all dose-cohorts. Treatment with AAV8-RLBP1 resulted in the ...

Gene therapy effective in hereditary blindness - | Karolinska Institutet

... RLBP1 gene, the gene that is damaged in Bothnia dystrophy. Image ... “Interim safety and efficacy of gene therapy for RLBP1-associated ...

Interim safety and efficacy of gene therapy for RLBP1-associated ...

Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial. Anders Kvanta 1. , Nalini Rangaswamy2, Karen ...

Update on Viral Gene Therapy Clinical Trials for Retinal Diseases

The interim safety evaluation presented ... Further studies are needed to investigate the safety and efficacy of this RP-related gene augmentation therapy.

Study Demonstrates Gene Therapy's Potential to Treat Hereditary ...

A study shows that gene therapy can improve vision in Bothnia dystrophy patients. By using a viral vector to deliver a functional RLBP1 gene ...

Neurogene Reports Positive Interim Efficacy Data from First Four ...

Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett ...

Beacon Therapeutics' XLRP Gene Therapy AGTC-501 Continues to ...

There were no SAEs deemed related to AGTC-501 itself. Beacon additionally pointed out that according to Kaplan-Meier survival curves, ...

Safety and Efficacy of Adeno-Associated Viral Gene Therapy in ...

Overall, AAV therapy vectors were associated with a cumulative incidence of at least one SAE of 8% (95% confidence intervals [CIs] of 5% to 12%). SAEs were ...

Dr. Anand Presented Interim Study Results of New Gene Therapy for ...

Interim Data on ATSN-201 Shows Promise for XLRS - HCPLive

At AAO 2024, Kay describes encouraging interim safety and efficacy results from the ATSN-201 gene therapy trial in patients with X-linked ...

Voretigene neparvovec for inherited retinal dystrophy due to RPE65 ...

Voretigene neparvovec (VN) is the first gene therapy approach approved for their treatment. To date, real life experience has demonstrated functional ...

Interim safety and efficacy of gene therapy for RLBP1-associated ...

Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial · Searching Remote Databases, Please Wait.

Beacon Therapeutics Announces Positive 12-Month Data from ...

Presentation – Subretinal AGTC-501 Gene Therapy for XLRP: 12-Month Interim Safety & Efficacy Results of the Phase 2 SKYLINE Trial · Presenter ...

Experimental gene therapy discovered in-house enters clinical testing

"RLBP1 RP has no approved treatment, making it a disorder with very high unmet medical need," says Kali Stasi, a Translational Medicine Expert ...

Gene Therapy Approach Demonstrated Safety With Durable Effect in ...

Subretinal dosing has been completed in all 42 participants across the five cohorts, and at the time of the interim analysis there was 5- to 6-month follow-up ...

Efficacy and Safety of Retinal Gene Therapy Using Adeno ...

Choroideremia is a good candidate for gene therapy owing to the small size of the CHM gene, enabling packaging within an adeno-associated virus ...

Neurogene Presents Favorable Safety Data from Phase 1/2 Trial of ...

“Gene therapy has the potential to address the underlying cause of Rett syndrome with a one-time treatment, and these interim safety data from ...

Gene Therapy Improves Functional Measures in XLRP - CGTLive®

Patients with X-linked retinitis pigmentosa (XLRP) treated with AGTC-501 gene therapy experienced improvements in visual function including retinal sensitivity.

Gene Therapies in Clinical Development to Treat Retinal Disorders

The phase I/II HORIZON trial (NCT03316560) began in April 2018 with 29 male adults receiving a single dose of AGTC-501 in one eye. The interim ...