Remove Cardiology Remove Gene Remove Gene Therapy
article thumbnail

Rocket agrees to acquire biotech firm Renovacor

Pharmaceutical Technology

The most advanced programme of the company is adeno-associated virus (AAV)-based gene therapy, REN-001. It leverages gene transfer technology to target the monogenic cause of BAG3-linked dilated cardiomyopathy (DCM), a severe kind of heart failure. .

article thumbnail

First hybrid gene therapy shows early promise in treating long QT syndrome

Scienmag

— In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition. ROCHESTER, Minn.

Insiders

Sign Up for our Newsletter

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

article thumbnail

STAT+: Pharmalittle: Pfizer scaling back early-stage research into rare diseases; BioNTech and U.K. partner on cancer trials

STAT News

… Pfizer told employees it plans to pull back on early-stage research into treatments for rare diseases, including the development of new viral-based gene therapies , Barron’s reports. Among the assets on the chopping block is a gene therapy manufacturing facility in Durham, N.C.,

article thumbnail

STAT+: Verve Therapeutics begins human tests of first ‘base editor,’ aiming at heart disease

STAT News

Somewhere in New Zealand, the first patient ever has been dosed with a kind of gene-editing treatment known as a base editor, a newer way of utilizing CRISPR for gene editing. The company studying the treatment, Verve Therapeutics, announced the news Tuesday. Continue to STAT+ to read the full story…

article thumbnail

“Can You Hear Me?” – The (Hopefully) Coming Transition from Virtual Meetings

Eye on FDA

A meeting scheduled for May 8 of the Cellular, Tissue and Gene Therapies Advisory Committee was slated to be a hybrid event – with members of the committee participating virtually and FDA staff appearing onsite at the agency campus, but was altered to become an all-virtual event via Adobe Connect.

article thumbnail

Aurlumyn (Iloprost) Gets FDA Nod as First Severe Frostbite Treatment

XTalks

Related: Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease The active ingredient in Aurlumyn is iloprost, a vasodilator that opens up blood vessels and stops blood from clotting. Blisters, which can affect all layers of skin, typically form 24 hours after exposure and tissue turns black if it starts dying.

article thumbnail

Bayer delivers on medical innovation fueling transformation of pharma business

The Pharma Data

Our leadership in cardiology, radiology and women’s health is recognized worldwide, and we are expanding our presence in oncology, working tirelessly to bring forward new approaches that can change the treatment paradigm for patients,” said Stefan Oelrich, Member of the Board of Management, Bayer AG and President of Bayer’s Pharmaceuticals Division.