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…

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…

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…

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…

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…

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…

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…

Tevard Biosciences has announced a four-year collaboration with Vertex Pharmaceuticals to develop new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) caused by nonsense mutations. The deal includes options to expand into additional muscular dystrophies and a second indication. “Given Vertex’s proven track record of developing novel therapies to…

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…

Jim Geraghty Jim Geraghty, a seasoned biotech executive and a veteran of the orphan drug movement since its inception, took some time to talk to Damian Doherty about his new book, Inside the Orphan Drug Revolution. Jim gives an historical perspective of the leading protagonists who…

This year marks the 10-year anniversary of when CRISPR-Cas9 gene editing was first revealed to the world. Much has happened since then, including wide rollout in many sectors, development of CRISPR-based therapeutics and the discovery that the technology can also be used to develop highly accurate and affordable diagnostics. The…

Deepak Asudani Deepak Asudani and his two children Ananya and Aarnav have published the first of a series of educational books on DNA and genetics aimed at schoolchildren who are curious to learn about what makes them unique whilst highlighting the similarities we share. Damian Doherty…