Editors' (re)View: A new RNA biotech; Gene therapy butterflies

Editor’s note: Welcome to Editors' (re)View, our editors’ takes on things going on in the pharma world that deserve some extra consideration.

A new RNA biotech in town

Earlier this week we reported ReNAgade Therapeutics’ launch, which was backed by the largest series A funding round of 2023. 

The Massachusetts-based biotech was established in 2021, with a mission to “overcome the limitations of RNA medicines” by applying its proprietary 'deliver, code, edit and insert' technologies. 

Notably, the company is led by a team of industry veterans with extensive experience. Amit D. Munshi, former CEO of Arena Pharmaceuticals, brings his expertise to the company. Pete Smith, who has worked at Alnylam and Moderna, joins as the chief scientific officer. Ciaran Lawlor, previously associated with Moderna and Boston Consulting Group, serves as the chief operating officer. Brian Shuster, with a background in companies like Bristol Myers Squibb, Johnson & Johnson, and JPMorgan, assumes the role of chief business officer.

With a bold entrance into the playing field, ReNAgade makes promises of delivering RNA medicines to previously inaccessible tissues and cells and expanding the potential market for addressing various diseases. The company has established a joint venture with Orna Therapeutics, integrating ReNAgade's delivery platform with Orna's circular RNA technology. 

— Andrea Corona

Gene therapy butterflies

A few years ago, I watched a documentary about a life-threatening skin disorder called dystrophic epidermolysis bullosa (DEB). People who suffer from this rare disease are sometimes referred to as 'butterfly children,' because their skin is as fragile as a butterfly's wings.

Children with DEB face severe pain, open wounds and a grueling daily bandaging process. Sometimes their fingers and toes need special dressing because the digits can actually join together as the skin heals. The documentary stuck with me for years because of what these kids have to endure on a daily basis, so hearing that the FDA approved a new treatment was incredible news.

DEB is caused by mutations in the COL7A1 gene, resulting in lack of production of functional type VII collagen (COL7) protein. The new gel, Krystal Biotech's Vyjuvek, is a genetically modified herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds. In a 31 subject trial, 65% of the Vyjuvek-treated wounds completely closed versus 26% of the placebo-treated wounds.

Aside from the relief it offers suffering patients, Vyjuvek racked up some big firsts: It's the first-ever FDA approved therapy for those battling DEB, the first-ever approved topical gene therapy, and the first-ever approved redosable gene therapy.

The approval is not only a huge milestone for patients and families affected by DEB, but it also ushers in a whole new paradigm to treat genetic diseases.

—Karen Langhauser