XTalks

MAY 26, 2023

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.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

Drug Discovery World

DECEMBER 19, 2022

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.

Gene Therapy 52

Gene Therapy Gene Dermatology Clinical Trials 52

Pharmaceutical Technology

MARCH 27, 2023

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

Medicine Gene Therapy Gene Dermatology 246

Drug Discovery World

FEBRUARY 5, 2024

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.

Gene Therapy 64

Gene Therapy Life Science Production Dermatology 64

pharmaphorum

DECEMBER 15, 2020

Eli Lilly has acquired Prevail, a biotech focusing on gene therapies for neurodegenerative diseases including Parkinson’s, in a deal potentially worth more than $1 billion. The CVR pays out if one of Prevail’s gene therapies is approved in one of several developed countries before December 31, 2024.

Gene Therapy 67

Gene Therapy Gene Dermatology Trials 67

The Pharma Data

SEPTEMBER 15, 2020

20vPnC showed a safety and tolerability profile that was similar to Prevnar 13 ® Pneumococcal 13-valent Conjugate Vaccine [Diphtheria CRM197 Protein]. Based on the acceptable safety profile and the favorable immune response data, including the 4th dose response data, Pfizer received Breakthrough Therapy Designation.

Gene Therapy 52

Gene Therapy Vaccination Vaccine Trials 52

XTalks

AUGUST 2, 2023

Johnson & Johnson’s Top 5 Best-Selling Drugs of 2022: 1) Stelara (ustekinumab) Stelara is an immunosuppressant biologic therapy that blocks the IL-12 and IL-23 proteins that play a role in plaque psoriasis and Crohn’s disease. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A.

Sales 98

Sales Manufacturing FDA Approval Life Science 98