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…

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…

An insect odorant receptor will be added to Cardea Bio’s Biosignal Processing Unit (BPU) to detect infectious diseases like SARS-CoV-2 in the air in a collaboration with the Bill and Melinda Gates Foundation. The San Diego health tech company was founded in 2013 and has specialized in making and refining…

Polygenic risk scores (PRS)—which quantify inherited risk by integrating multiple sites of DNA variation—promise a more personalized approach to medicine. For many years, academic groups have been grappling with the mechanics of creating PRS, relying on large-scale GWAS analyses from large patient cohorts like the UK Biobank and others to…

Looking for an alternative to the problematic adeno-associated virus (AAV) as a delivery vehicle for gene therapies, Sarepta Therapeutics has signed a $57 million deal with GenEdit, which has developed a polymer nanoparticle platform for tissue-selective delivery called NanoGalaxy. The companies will collaborate to use NanoGalaxy and Sarepta’s gene editing technology…