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First gene therapy to treat rare blood disease nears European approval

“The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. “Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European

CRISPR Drapes Invisibility Cloak over Stem Cells

“An international research team has used CRISPR-Cas9 gene editing to render induced pluripotent stem cells (iPSCs) invisible to the immune system, a bioengineering sleight of hand that initial in vivo tests suggest could open the door to generating stem cells for universal transplantation. The new approach, developed by scientists at the University of California, San

CMS unveils CAR-T proposal, with emphasis on patient outcomes

“Two CAR-T therapies — Novartis’ Kymriah (tisagenlecleucel) and Gilead Sciences’ Yescarta (axicabtagene ciloleucel) — are already on the market, yet their high price tags and potentially one-time administration has put pressure on CMS to figure out a way to cover these kinds of treatments. The agency took a few steps toward that goal over the

REGENXBIO Receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy

“REGENXBIO announced the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to RGX-181. RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene. In addition to the Rare

ARM Extends Its Reach

Adding Education to Advocacy at the Alliance for Regenerative Medicine “In the beginning of the new millennium, a new therapeutic approach also dawned — actually a number of new approaches gathered collectively under the umbrella term “regenerative medicine (RM).” The most notable case in the first years of the 2000s concerned Christopher Reeve, the first

Bridging The Public Knowledge Gap Around Cell And Gene Medicine

By: Morrie Ruffin, Executive Director, ARM Foundation for Cell and Gene Medicine in Cell & Gene Cell and gene therapies hold the potential to transform medicine; however, if patients are to gain access to these therapies, we must increase public awareness and understanding of how these therapies can benefit them. According to a review of

ARM Foundation for Cell and Gene Medicine Announces Board of Directors Appointments

Experts in regenerative medicine bring exceptional leadership to ARM Foundation to help increase understanding and acceptance of cell and gene medicine. Washington, D.C., August 14, 2018 – The ARM Foundation for Cell & Gene Medicine (ARM Foundation), an independent 501(c)(3) non-profit organization, today announced five appointments to its Board of Directors: Amy DuRoss of Vineti;

ARM Foundation for Cell and Gene Medicine releases Health Economic Impact Landscape Analysis led by IQVIA

Health Economic Impact Landscape Analysis is the first step to developing macroeconomic value model for regenerative medicine advanced therapies. Washington, D.C., August 6, 2018 – The ARM Foundation for Cell and Gene Medicine (ARM Foundation) today released its first-of-its-kind Health Economic Impact Landscape Analysis of regenerative medicine advanced therapy The analysis includes a comprehensive review

ARM Foundation for Cell and Gene Medicine launched

The Alliance for Regenerative Medicine (ARM) launched an independent, non-profit foundation dedicated to researching, creating and disseminating information about gene and cell therapies. The ARM Foundation for Cell and Gene Medicine has commissioned a study of the macroeconomic impacts of regenerative medicines, and market research on public awareness and acceptance of gene medicines, Stewart Parker,

New foundation will increase awareness about cell and gene therapy, but caution is recommended

The Alliance for Regenerative Medicine (ARM) announced the launch of the ARM Foundation for Cell and Gene Medicine, which will be dedicated to increasing public awareness and understanding of the clinical and societal benefits of cell and gene medicine — including gene therapy, gene editing, cell therapy, tissue-engineering, and organ regeneration — and supporting its

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