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The most advanced programme of the company is adeno-associated virus (AAV)-based genetherapy, REN-001. It leverages gene transfer technology to target the monogenic cause of BAG3-linked dilated cardiomyopathy (DCM), a severe kind of heart failure. .
— In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid genetherapy in long QT syndrome, a potentially lethal heart rhythm condition. ROCHESTER, Minn.
… Pfizer told employees it plans to pull back on early-stage research into treatments for rare diseases, including the development of new viral-based genetherapies , Barron’s reports. Among the assets on the chopping block is a genetherapy manufacturing facility in Durham, N.C.,
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…
A meeting scheduled for May 8 of the Cellular, Tissue and GeneTherapies 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.
Related: Vyjuvek Gets FDA Nod as First Topical GeneTherapy 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.
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.
Scientific blogs discussing new advancements in oncology, cardiology, or rare diseases. For example, a LinkedIn article on the future of genetherapy can position a brand as an authority in genetic medicine, attracting biotech investors, researchers, and prescribing physicians.
Pfizer’s Rare Disease late-stage pipeline currently includes three genetherapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. and -50.4%
Patients’ genetic profile strongly influences size of effect in early-phase trial People with extremely high levels of triglycerides (a type of fat in the blood) and a specific genetic profile saw a substantial reduction in triglycerides after taking the human monoclonal antibody evinacumab compared with those taking a placebo, in a study presented (..)
Meanwhile, BioMarin’s genetherapy Roctavian is approved by the FDA for the treatment of hemophilia A. Onureg (azacitidine), a leukemia therapy, also saw large growth of 70 percent to $124 million in 2022, and the multiple sclerosis drug Zeposia (ozanimod) generated $250 million in 2022, an 87 percent increase.
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