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).

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

Pharmaceutical Technology

JULY 29, 2022

In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

XTalks

NOVEMBER 10, 2023

“As we strive to help patients with limited or no treatment options, developing innovative treatments in rare diseases is an inspiring challenge and one we have taken on for 70-plus years as a leader in hematology.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

pharmaphorum

SEPTEMBER 28, 2022

. “We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease,” said Nia Tatsis, Vertex’ chief regulatory and quality officer, in a statement.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective?

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy 111

Gene Therapy Gene Drugs FDA Approval 111

XTalks

JANUARY 6, 2025

For example, antisense oligonucleotide (ASO)-based therapies have gained traction, with 100 Phase I clinical trials initiated and around 25 percent of these advancing to Phase II or Phase III trials in recent years. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111

Pharmaceutical Technology

APRIL 24, 2023

Covid-19 vaccines from Moderna and Pfizer /BioNTech, which use lipid nanoparticles, became the only two FDA-approved vaccines for almost all ages. However, after a period of slow movement in the field, the FDA approved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147