Gene Therapy

ClinicalAugust 3, 2026

Gene Therapy for Sickle Cell Disease Shows Promising Safety and Efficacy in 36 Patients

An expanded access protocol for a gene therapy targeting BCL11A in sickle cell disease has shown increased fetal hemoglobin levels without unexpected safety issues in 36 patients.

ClinicalAugust 1, 2026

AAVrh10-PCCA Gene Therapy Trial for Propionic Acidemia Initiated

A clinical trial is underway to assess the safety and efficacy of AAVrh10-PCCA gene therapy for propionic acidemia.

CompetitiveJuly 27, 2026

Solid Biosciences Initiates SGT-003 Gene Therapy Study for Duchenne Muscular Dystrophy

Solid Biosciences has launched a multicenter, open-label study to evaluate SGT-003 in Duchenne muscular dystrophy across five age cohorts.

ClinicalJuly 24, 2026

Phase 1/2 Study of TSHA-105 in SLC13A5 Citrate Transporter Disorder Initiated

A Phase 1/2 open-label study is underway to evaluate TSHA-105 for SLC13A5 Citrate Transporter Disorder.

RegulatoryJuly 22, 2026

Long-Term Safety Follow-Up for Seattle Children's Gene Therapy Products Initiated

Seattle Children's Hospital is conducting a long-term follow-up study to assess the safety of its gene therapy products over a period of up to 15 years.

RegulatoryJuly 15, 2026

Long-term Follow-Up Study on Gene Therapy Adverse Events in Pediatric Oncology

A long-term follow-up study will evaluate delayed adverse events in pediatric patients who received gene therapy.

ClinicalJuly 13, 2026

Elpida Therapeutics initiates MELPIDA trial for SPG50 using AAV9 gene therapy

Elpida Therapeutics is conducting a trial for MELPIDA, a gene therapy targeting SPG50, focusing on safety and tolerability.

RegulatoryJuly 2, 2026

FDA Approves Casgevy for Young Children with Sickle Cell Disease

The FDA has granted supplemental approval for Casgevy for young children with sickle cell disease and transfusion-dependent β thalassemia.

CompetitiveJuly 1, 2026

Phase 1/2 Trial of AFTX-201 in BAG3-Associated Dilated Cardiomyopathy Initiated

Affinia Therapeutics has launched Phase 1/2 trial for AFTX-201 targeting BAG3-associated dilated cardiomyopathy.

ClinicalJuly 1, 2026

AbbVie Initiates Study on Surabgene Lomparvovec for Neovascular AMD

AbbVie is conducting a study to evaluate the safety and efficacy of Surabgene Lomparvovec in adults with neovascular age-related macular degeneration.

ClinicalJune 30, 2026

Phase I/II Trial of Lentiviral Gene Transfer for XSCID in Children Over Two Years

A Phase I/II trial is underway to evaluate a lentiviral gene transfer treatment for X-linked severe combined immunodeficiency (XSCID) in children older than two years.

ClinicalJune 27, 2026

AbbVie Initiates Phase 2b/3 Trial of Surabgene Lomparvovec for Diabetic Retinopathy

AbbVie is conducting Phase 2b/3 clinical trial to evaluate surabgene lomparvovec as a gene therapy for diabetic retinopathy.

ClinicalJune 26, 2026

Study on Fibroblast-Derived Cell Lines for Gene Therapy in Immune Disorders

NIAID is conducting a study to collect skin cell samples from patients with immune disorders to explore personalized gene therapy.

CompetitiveJune 25, 2026

Vironexis Biotherapeutics initiates Phase 1/2 trial for VNX-202 in HER2 positive cancers

Vironexis Biotherapeutics has launched Phase 1/2 trial to evaluate VNX-202 in HER2 positive cancer patients.

ClinicalJune 24, 2026

AAVB-039 shows promise in Stargardt Disease with ongoing safety and efficacy study

The 039-101 study is evaluating the safety and tolerability of AAVB-039 in Stargardt disease patients.

CompetitiveJune 24, 2026

Phase I Trial of Lentiviral Gene Therapy for Hemophilia A Initiated

A Phase I trial is underway to evaluate the safety and efficacy of lentiviral gene therapy for hemophilia A.

RegulatoryJune 23, 2026

FDA Draft Guidance Aims to Accelerate Gene Therapy Development

The FDA has released draft guidance intended to streamline the development process for gene therapies.

ClinicalJune 20, 2026

Gene Transfer Study for Giant Axonal Neuropathy Initiated by NINDS

A clinical trial has been initiated to evaluate the safety and potential efficacy of scAAV9/JeT-GAN for treating Giant Axonal Neuropathy.

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