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

CRISPR Clinical Trials: A 2024 Update - Innovative Genomics Institute

The treatment was generally well-tolerated with an acceptable safety profile. In a US-based phase 1 trial of 16 participants, 15 responded to ...

Ultragenyx and GeneTx Provide Program Update on GTX-102 for ...

“We are seeing encouraging early clinical activity across multiple domains and multiple measurements without any evidence of drug-related safety ...

Novel Gene Therapy Safe for Retinitis Pigmentosa - Medscape

STOCKHOLM — Trial results are demonstrating the safety and effectiveness of a novel gene modifier therapy for treating retinitis pigmentosa ...

Alzheimer Gene Therapy LX1001 Demonstrates Dose-Dependent ...

LX1001, an AAV gene therapy, was considered safe and well tolerated, with no events of amyloid-related imaging abnormalities (ARIA) ...

The STEER research study - Novartis

Efficacy and safety have not been established for this new use. There ... Adeno-associated virus (AAV) as a vector for gene therapy. BioDrugs. 2017;31 ...

Gene therapy clinical trials worldwide to 2023—an update - Ginn

Interim data presented for two higher doses of 0.7 and 1.0 mg/kg of NTLA ... Safety and efficacy of gene replacement therapy for X-linked ...

Taking Stock of Retinal Gene Therapy: Looking Back and Moving ...

The safety and efficacy results in one early-onset severe blinding disease may lead to the first gene therapy drug approval in the United States ...

ST-920 gene therapy lowers Fabry disease severity in trial

“We remain encouraged by the emerging safety and efficacy data ... Such antibodies are associated with reduced ERT efficacy. After gene ...

Oral Presentations - ASGCT Annual Meeting

Methods: REKLAIM is a Phase I/II dose-escalating intravenous gene therapy to evaluate safety and efficacy of ... Interim Safety and Efficacy of Anti-. CD70 ...

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

Durable Effect in Interim Wet. AMD Trial Results. A dose-dependent ... RGX-314 is a novel adeno-associated viral vector carrying a gene that encodes a.

Gene therapy with bidridistrogene xeboparvovec for limb-girdle ...

Here we report interim results from a first-in-human, open-label, nonrandomized, phase 1/2 trial evaluating the safety and efficacy of bidridistrogene ...

Safety of AADC Gene Therapy for Moderately Advanced Parkinson ...

All 4 non-vector–related SAEs (atrial fibrillation and pulmonary embolism in 1 participant and 2 events of small bowel obstruction in another participant) ...

A randomised, double-blind, placebo-controlled, multicentre study of ...

A randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BIOBYPASS gene therapy in patients with severe coronary artery ...

EyeDNA Therapeutics Announces Positive 24-month Data ...

“The highly encouraging safety and efficacy data observed in patients two years after treatment with HORA-PDE6b continue to support our view ...

Package Insert - ZOLGENSMA - FDA

ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of ... The safety and efficacy of ZOLGENSMA in patients ...

Press Releases - Atsena Therapeutics

Atsena Therapeutics Announces Positive Clinical Data from the First Cohort of Phase I/II Trial Evaluating ATSN-201 Gene Therapy for the Treatment of X-linked ...

Beacon Therapeutics treats first patient in global registration trial for ...

“We are delighted to start the final phase of this important trial of gene therapy for X-linked retinitis pigmentosa,” he said in the news ...

Recombinant Adeno-Associated Virus Gene Therapy in Light of ...

The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies in Europe and the United States are landmark ...

New Data at Cure SMA Highlight Potential Benefit of SPINRAZA ...

... safety of SPINRAZA treatment after gene therapy.” Interim Clinical ... treatment of SMA; and risks and uncertainties associated with ...

The effect of human gene therapy for RPE65-associated Leber's ...

RPE65-associated LCA (RPE65-LCA) is an inherited retinal degeneration caused by the mutations of RPE65 gene and gene therapy has been ...