University of California, San Diego (UCSD), School of Medicine scientists used human brain organoids to reveal how a genetic mutation associated with a profound form of autism disrupts neural development. The scientists used gene therapy  tools to recover the gene’s function. Their study is published in Nature Communications. “Transcription Factor…

A CRISPR-directed gene-editing therapy, which targets a gene that enhances cancer cell survival in response to chemotherapy for non-small cell lung carcinoma (NSCLC), was recently developed by researchers at ChristianaCare’s Gene Editing Institute in Delaware. The scientists found disrupting NRF2 in NSCLC tumor cells resulted in collateral damage due to…

A new study reports that gene therapy can be combined with a novel surgical approach to deliver genes into the inner ear of monkeys, which could provide a promising pathway for translating this research to creating gene therapy to treat human hearing disorders. Inner ear gene therapy, using adeno-associated viral…

NICE has authorized the use of gene therapy atidarsagene autotemcel (Orchard Therapeutics’ Libmeldy) for some children with metachromatic leukodystrophy (MLD). At list price of $3.8 million, this one-off treatment is the most expensive drug NICE has ever evaluated. Following public consultation on NICE’s earlier draft guidance, which did not recommend…

The University of Pennsylvania’s Gene Therapy Program (GTP) has signed a collaboration agreement with contract manufacturer The Center for Breakthrough Medicines (CBM) worth an estimated $75-$100 million. This partnership is focused on moving therapeutics from concept to clinic by connecting some of Penn’s established gene therapy platforms with CBM’s manufacturing…

Scientists from the University of Virginia (UVA) School of Medicine have developed a gene therapy to treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. The gene therapy, developed by Stoke Therapeutics, is now in clinical trials. Because most Dravet syndrome cases are caused…

A team of scientists at Children’s National Hospital, Washington, DC, has adopted a new approach to develop a gene therapy for limb-girdle muscular dystrophy 2B (LGMD2B) that circumvents the need for targeting hard-to-reach degenerating muscles with large doses and packaging the large dysferlin gene into a carrier vector—the two primary…

The Phase Ib trial assessing Pfizer’s mini-dystrophin gene therapy candidate PF-06939926 in Duchenne muscular dystrophy (DMD) was placed on hold by the FDA after the company acknowledged the death of a young male participant in the open-label study. The study was designed to assess a single intravenous infusion of PF-06939926…

Roche Group company Spark Therapeutics announced it plans to invest $575 million in a new gene therapy innovation center on the Drexel University campus in the University City neighborhood of the Philadelphia. The 500,000-square-foot center will serve as a Roche global center of excellence for gene therapy manufacturing, facilitating collaborations…

An experimental gene therapy developed at Boston Children’s Hospital was able to reverse a common type of inherited hearing loss in a mouse model. This is a new type of gene therapy, as the gene causing the hearing loss, stereocilin (STRC), is too big to be incorporated into normal adeno-associated…

AviadoBio has come out of stealth mode to announce the raising of an $80M Series A round to develop gene therapies to target neurodegenerative conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The round was co-led by international VC’s New Enterprise Associates and Monograph Capital. Other participants…

Research led by Northwestern University shows that damage to mitochondria in the brain can cause late-onset Parkinson’s symptoms in mice and that it is possible to boost the effects of levodopa treatment in these animals using a form of gene therapy. Mitochondria provide energy to cells around the body and…

The FDA, NIH and fifteen private organizations and companies have launched the Bespoke Gene Therapy Consortium (BGTC) to help develop effective gene therapies for conditions that currently do not have treatment options. The consortium is the sixth initiative to come out of the Accelerating Medicines Partnership, which was launched in…

Mammoth Bioscience’s CRISPR systems technology will be used by Vertex Pharmaceuticals  to develop in vivo gene-editing therapies for two undisclosed genetic diseases, in a deal that has the potential to generate as much as $695 million for Mammoth, the two companies announced today. “We are focused on developing in vivo…

Boehringer Ingelheim (BI) is partnering for intellectual property and know-how from IP Group and Oxford Biomedica (OXB) to advance a potential gene therapy for cystic fibrosis (CF).  BI 3720931 is a novel, replication deficient lentiviral vector in an inhaled formulation. IP Group is acting on behalf of the three UK…