article thumbnail

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

article thumbnail

‘Life-changing’ topical gene therapy gel heals EB wounds

Drug Discovery World

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . This was a life-changing event for Vincenzo,” said Peter Marinkovich, Associate Professor of Dermatology and Director of Stanford Medicine’s Blistering Disease Clinic.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

First Topical Gene Therapy + Bausch + Lomb’s First Prescription Med for Dry Eye Ep. 112

XTalks

In this episode, Ayesha talked about the FDA approval of the first topical gene therapy for the treatment of a rare skin condition. Krystal Biotech’s Vyjuvek is a topical gene therapy that has been approved for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).

article thumbnail

Regenerative medicine: new focus on VEGF target?

Pharmaceutical Technology

Staggeringly, none of the 61 drugs approved for Parkinson’s within the last 20 years were gene therapies. There is a distinct shift in drug targets between early- and late-stage therapies, with drugs moving away from COL7A1 and CD19 to the VEGF target. However, gene therapies remain key molecule types for these targets.

Medicine 246
article thumbnail

Regenerative medicine switches to VEGF as top drug target

Drug Discovery World

Two key regenerative medicines in late stages are beremagene geperpavec, a gene therapy by Krystal Biotech for the treatment of epidermolysis bullosa, a rare dermatological condition, and Yescarta, a gene-modified cell therapy by Gilead Sciences indicated for nodal marginal zone B-cell lymphoma.

article thumbnail

SLAS2024: Day One Highlights

Drug Discovery World

Williams discussed the advantages of the company’s ‘disease agnostic’ small molecule degraders, and its success so far in translating results from preclinical to clinical tests in dermatology, inflammation and oncology indications. Product launches More than 400 exhibitors are at the event this year, many of whom are launching new products.

article thumbnail

Filsuvez Topical Gel Gets FDA Approval for Epidermolysis Bullosa

XTalks

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector. Regarding the price of Filsuvez, it has not yet been publicly disclosed by the company.