Kate Therapeutics Inc. recently presented data on a novel muscle- and heart-targeted, liver de-targeted development candidate for the treatment of Duchenne muscular dystrophy (DMD) – KT-809.

Precision Biosciences Inc. has received pre-IND regulatory feedback from the FDA and ex-U.S. agencies providing alignment and clarity on Precision's final IND/CTA-enabling preclinical plans and clinical strategy for PBGENE-HBV.

Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE).

Bloomsbury Genetic Therapies Ltd. announced that the FDA granted orphan drug designation for its investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency (OTCD).

Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel phase I clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia (AML) and severe aplastic anemia.

Researchers from Interius Biotherapeutics Inc. presented the development and preclinical evaluation of a novel gene therapy candidate, INT-2104, as potential candidate for the treatment of B-cell malignancies.

Alexion, Astrazeneca Rare Disease, part of Astrazeneca plc, has entered a definitive purchase and license agreement for a portfolio of preclinical gene therapy programs and enabling technologies from Pfizer Inc.

EG 427 has announced the final closing of a series A financing, bringing the total raised in the...

Regenxbio Inc. has announced it is developing a...

Sensorion SA has submitted a clinical trial application (CTA) for OTOF-GT to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA). Sensorion plans to submit the CTA in Europe in the coming weeks.