Pfizer saw positive topline results of its investigational gene therapy for adults with hemophilia A in a Phase III study (AFFINE). The therapy, giroctocogene fitelparvovec, reduced patients’ bleeds for at least 15 months and was generally well tolerated. It comprises a bio-engineered AAV6 capsid and a modified B-domain deleted human…

A new gene therapy treatment for Duchenne muscular dystrophy show promise of arresting the decline of the muscles of those affected by this inherited genetic disease, and perhaps, in the future, repairing those muscles. The laboratory results not only show promise to treat patients with this severely debilitating and incurable…

William Chou, MD, strives to bring more hope to patients with neurodegenerative diseases. And Passage Bio, where Chou is president and CEO, may now have the green light to test their lead gene therapy asset more broadly than the initial focus of frontotemporal dementia (FTD) caused by mutations in one…

Lexeo Therapeutics this week announced positive interim data from early trials of its gene therapy LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. LX2006 was well tolerated with no treatment-related serious adverse events, and provided clinically meaningful improvements in cardiac biomarkers were observed with increasing improvement over time.  FA…

Updated interim data from a Phase I/II trial of uniQure’s “one shot” gene therapy in Huntington’s disease were promising, suggesting AMT-130 is effective and safe. Huntington’s is one of the most common rare diseases. It is caused by the expansion of CAG trinucleotide in exon 1 of a multifunctional gene…

Harith Rajagopalan, MD, PhD, founded Fractyl Health with the vision of eliminating obesity, and it looks like his idea for how to do that has gained some preclinical steam. The metabolic therapeutics company has new data from Rejuva—its adeno-associated virus (AAV)-based glucagon-like peptide-1 (GLP-1) pancreatic gene therapy program designed to…

Sarepta is shining, as its Duchenne Muscular Dystrophy (DMD) gene therapy Elevidys has been granted the much wider label the biotech was aiming for—making it available to four-year-olds and older. This news comes just about a week after Pfizer, which was fast on Sarepta’s heels with their own gene therapy…

Scientists at the University of Bristol have demonstrated that restoring a protein that declines with age can prevent age-related and immune-mediated retinal degeneration. The researchers identified rare genetic mutations in the IRAK3 gene, which produces the IRAK-M protein and has been associated with age-related macular degeneration (AMD), a leading cause…

A trial conducted by researchers in the U.S. and China has shown that a new gene therapy for children born with autosomal deafness caused by the OTOF gene restored hearing function when both ears were treated. The five children in the study, reported today in Nature Medicine, also showed better…

Over the last thirty years, protein-based injections and DNA therapeutics accounted for the majority of newly introduced treatments. However, these medicines remain out of reach for many around the world due to high pricing, with some therapies ranging from thousands to several millions of dollars to purchase. [caption id=”attachment_147257″ align=”alignright”…

Researchers have identified a compact version of a gene-editing protein that is small enough to fit into adeno-associated viruses (AAVs) and can be modified to deliver highly efficient, targeted therapy to cells. The relatively small version of the CRISPR-associated protein Cas12a occurs naturally in the Erysipelotrichia class of bacteria and is…

Verve Therapeutics, a biotech based in Cambridge, MA, has progressed its second base edited gene therapy, aimed at significantly lowering low-density lipoprotein (LDL) cholesterol, to the clinic in a Phase I trial. The company is using base editing, a more precise gene editing technique than CRISPR-Cas9, to create novel therapies…

Broken String Biosciences and the Francis Crick Institute are partnering to develop novel applications for the start-up’s proprietary DNA break-mapping platform, INDUCE-seq, to investigate the impact of genomic instability in the development of amyotrophic lateral sclerosis (ALS). The partnership will examine DNA break-mapping technology and advance understanding of genomic instability…

By Larissa Warneck-Silvestrin July 24th of 2006. It’s a date that you never forget.” On that fateful Monday, Sharon King and her husband were told by a geneticist that their seven-year-old daughter, Taylor, had a rare genetic disease. They asked the geneticist what they could do and where to seek…

Sickle cell disease (SCD) will be the first focus of the U.S.’s new Cell and Gene Therapy (CGT) Access Model, which aims to increase access to cell and gene therapies for vulnerable populations. Gene therapies are some of the most expensive treatments in the world. The Centers for Medicare &…