News

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Using mesenchymal stem cells to treat adults with recessive dystrophic epidermolysis bullosa (RDEB) appears to be safe and may ease symptoms such as itching for at least two months, a Phase 1/2 clinical trial shows. The results were described in the study “A Phase I/II open-label…

Cannabinol (CBN), a rare type of cannabinoid, is the active component of InMed Pharmaceuticals‘ INM-755 treatment candidate for epidermolysis bullosa (EB). CBN is also the active component of InMed’s INM-088, being developed as a therapy to reduce pressure in the eyeball (intraocular) and confer neuroprotection for…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

Krystal Biotech is opening its second gene therapy facility, called Astra, to support the production and potential commercialization of B-VEC, the company’s gene therapy for people with dystrophic epidermolysis bullosa (DEB). The new cGMP (current good manufacturing practices) facility is based in Findlay Township, Pennsylvania. It…

Aegle Therapeutics has raised $4 million to fund the first clinical trial evaluating AGLE-102, a therapy candidate for people with dystrophic epidermolysis bullosa (DEB) based on the company’s extracellular vesicle therapy. The Phase 1/2 trial (NCT04173650) is anticipated to begin in the first half of…

Abeona Therapeutics has begun its Phase 3 VIITAL trial evaluating EB-101, a gene-corrected cell therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the company recently announced. An institutional review board at Stanford University approved the start of the pivotal…

Castle Creek Pharmaceutical recently acquired Fibrocell Science and is now leading the development of treatments for epidermolysis bullosa (EB), including the investigational gene therapy FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The companies had previously established a partnership to develop FCX-007. “Our organizations have a shared…

The Epidermolysis Bullosa iPS Cell Consortium has received $1.6 million in funding to develop stem cell-based therapies for people with inherited skin diseases such as epidermolysis bullosa (EB). A grant from the National Institutes of Health (NIH) 21st Century Cures Act provided $800,000, which was quickly matched by…