pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The disease is also difficult to diagnose and often takes around six years before doctors correctly identify it.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

AUGUST 13, 2020

The FDA controversially approved Vyondys 52 late last year after a previous rejection and an appeal from Sarepta. Viltepso was evaluated in two clinical studies with a total of 32 patients, all of whom were male and had genetically confirmed DMD. The post NS Pharma takes on Sarepta as FDA approves DMD drug appeared first on.

FDA Approval 52

FDA Approval Drugs Gene Trials 52

FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Gene Therapy 119

Gene Therapy Gene FDA Approval Production 119

Drug Discovery World

OCTOBER 18, 2023

What’s more, they are expensive, not readily available, and in many countries, there are a host of stringent regulations, as well as ethical and legal considerations around their use. Mouse models, on the other hand, are cost-effective and readily available, making them a favourite for many researchers. She received a Ph.D.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

STAT News

MARCH 6, 2023

But they said the sum covers only the direct costs of designing and manufacturing the vaccines ― which are tailored to the unique genetic makeup of each patient’s tumor ― and that the FDA has approved the breakdown of expenses.

Vaccination 98

Vaccination Vaccine Drugs Clinical Trials 98

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

pharmaphorum

DECEMBER 21, 2021

However, despite the work still left to be done, there are now a growing number of treatments in the pipeline and 2020 proved to be a remarkable year for orphan drug approvals, with the US FDA approving more orphan designated treatments than non-orphan drugs. Headway being made.

Development 98

Development Genome Genomics Drugs 98