News

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…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to APR-TD011, a spray therapy candidate for the treatment of epidermolysis bullosa (EB). This designation is intended to encourage the development of therapies for rare and serious diseases, which are expected to outperform existing alternatives. Among its…

After its recent clinical hold by regulators, the Phase 3 VIITAL trial has been given the green light to investigate EB-101, a gene-corrected cell therapy for people with recessive dystrophic epidermolysis bullosa (RDEB). Abeona Therapeutics, the company developing EB-101, plans to start VIITAL in early 2020. RDEB…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

Scientists have discovered a new mutation in the COL7A1 gene — which provides instructions for making a type of collagen protein — in a Chinese family with pretibial dystrophic epidermolysis bullosa (DEB), a study revealed. The study, “A novel mutation of COL7A1 in a Chinese DEB‐Pt family…