Jenna Payesko

The agency said the gene therapy should remain on the market while it assesses the situation and does not impact their evaluation of data from the human clinical trials.

Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.

The investigational therapy has the potential to be a first-in-class neuronal progenitor cell therapeutic with anti-apoptotic activity that improves cerebral blood flow and neurological outcome in stroke.

A phase 1/2 clinical trial is expected to be initiated in the second half of 2019.

Abeona Therapeutics is currently planning a phase 1/2 clinical trial to evaluate ABO-202 in Batten disease.

The planned phase 1/2 trial of the recombinant AAV5 vector treatment, the first one-time administered AAV gene therapy to enter clinical testing for Huntington disease, is expected to begin dosing patients in the second half of 2019.

The trial results indicate that EBV-specific adoptive T cell therapy is well tolerated and further back this approach in efficacy trials.

The designation is backed by positive interim data from ASPIRO, which has demonstrated significant improvements in neuromuscular and respiratory function at week 24.

Clinical data demonstrate that CD34+ cell therapy improves exercise capacity, angina frequency and reduces mortality in no-option refractory angina.

The investigational new drug application allows the initiation of phase 1/2 clinical trial to assess the safety of BIVV003 in adults.

A novel subretinal implantation of a stem cell-based bioengineered patch may restore vision or prevent further vision loss.

This is the first and only FDA-approved gene therapy for treatment for an inherited disease.

This is the first gene therapy approved in the US targeting mutations in a specific gene.