Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, failed on the primary endpoint in a pivotal Phase III study, the company announced yesterday. The endpoint is a measure of motor function, called the North Star Ambulatory Assessment.  Despite this setback, the company said the results of the trial were…

On Friday, an FDA advisory committee voted 8-6 that the benefits of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy (DMD) outweighs the treatment’s risks. If it is approved, SRP-9001 (delandistrogene moxeparvovec), which is being developed with Roche, will be the first gene therapy approved for this disease. The vote…

In a striking collaboration, four companies shared laboratory and clinical data to find what was causing certain serious adverse events (SAEs) in Duchenne Muscular Dystrophy (DMD) gene therapy trials. The companies, all frontrunners in this field, are Pfizer, Sarepta, Solid Biosciences, and Genethon. Based on this work, they propose these…

Researchers based at Harvard and the Broad Institute have adapted currently used gene therapy vectors to make them more suitable for targeting muscular conditions such as Duchenne muscular dystrophy and also to make them less toxic to patients. One of the most common methods for delivering gene therapy today is…

After a 30-year wait, the last decade has seen a number of therapies for treating Duchenne muscular dystrophy (DMD) get FDA approval, either by the normal or accelerated pathway, with many more in development. DMD is a recessive, degenerative muscle disorder that almost exclusively affects males or people with one…

By Donna Snyder One of the quandaries that researchers face when considering a clinical study in children is balancing the need to protect this vulnerable patient population with the need to conduct pediatric research that can improve their health and well-being. The ethical framework for pediatric research was developed to…

Michelle Werner, CEO, Alltrna The mRNA molecule has played a transformational role in recent years in helping contain the spread of SARS-CoV-2, but a less known molecular sibling has been largely overlooked in the past by biotech entrepreneurs and industry. The molecule is known as tRNA (transfer…

On the heels of success for its new heart drug (acoramidis) in Phase III, BridgeBio Pharma has raised up to $1.2B from Blue Owl Capital and Canada Pension Plan Investment Board (CPP Investments). BridgeBio expects acoramidis will launch soon and become the “potential backbone of therapy for transthyretin amyloid cardiomyopathy…

Precision medicines are gaining increasing traction in healthcare. Here are five companies driving the field with recent approvals in RNA therapeutics, cell and gene therapies, and more. Medicines have traditionally been given to patients in a one-size-fits-all approach, with many patients failing to benefit and experiencing side effects. This challenge…

New research by the EveryLife Foundation for Rare Diseases, in one of the first studies of healthcare utilization and costs for patients with a rare disease, has found that early rare disease diagnosis could save as much as $500,000 per patient. The report, “The Cost of Delayed Diagnosis in Rare…

Kate Therapeutics (KateTx) today emerged from stealth mode with a $51 million Series A round and a deal with Astellas Pharma to develop a gene therapy for X-linked myotubular myopathy (XLMTM). “KateTx is applying novel capsid and cargo technology platforms to enable skeletal and cardiac muscle targeting and liver de-targeting. We believe…

A little over a decade ago, a paper was published in Science1  that made science fiction a reality. Emmanuel Charpentier, Jennifer Doudna, and colleagues reported that they had identified a means of harnessing an element of a bacterial immune system to carry out genome editing in a way that was…

Cell and gene therapy have surprisingly long histories. Cell therapy has existed in the form of bone marrow transplant, used to treat cancers and other conditions, since the late 1950’s, although it only came to prominence with the approval of the first chimeric antigen receptor (CAR) T-cell cancer therapies (Kymriah…

Results aren’t usually publicly shared, but behind the scenes, real-world data (RWD) and evidence (RWE) are having a big effect on rare disease drug development and clinical management. That impact is particularly evident in Duchenne Muscular Dystrophy (DMD) right now. Sarepta Therapeutics, for example, has three commercial DMD products already.…

Pfizer today announced very positive results from a Phase III study of fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult men with moderately severe to severe hemophilia B. This caps off a breakthrough year for hemophilia B treatment, as the first gene therapy for this blood clotting…