Two New Studies Point to the Promise of Gene Therapy for High Cholesterol
MONDAY, Nov. 13, 2023 (Healthday News) -- Two new gene-editing treatments that target dangerously high levels of cholesterol in people with a genetic predisposition to the condition were found safe and effective in new, groundbreaking research.While... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - November 13, 2023 Category: General Medicine Source Type: news

Daily statins taken by millions could be replaced by remarkable new gene therapy that cuts 'bad' cholesterol by up to 50% with a SINGLE dose, scientists claim as they hail 'brave new world' of heart treatment
Daily statins taken by millions to ward off heart attacks and strokes could be replaced 'within the decade' by a gene-editing jab, scientists claim. (Source: the Mail online | Health)
Source: the Mail online | Health - November 12, 2023 Category: Consumer Health News Source Type: news

EU Approval of AAV5 DetectCDx(TM) Provides Access to Companion Diagnostic for Hemophilia A Gene Therapy
SALT LAKE CITY, Nov. 10, 2023 -- (Healthcare Sales & Marketing Network) -- ARUP Laboratories today announced that it has gained the Conformité Européenne (CE) mark for AAV5 DetectCDx™ single-site use under the European Union's In Vitro Diagn... Diagnostics, Regulatory ARUP Laboratories, BioMarin Pharmaceutical, AAV5 DetectCDx, hemophilia A (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 10, 2023 Category: Pharmaceuticals Source Type: news

Family's gene therapy startup wins historic OK for clinical trial targeting daughter's rare disease
Less than 100 people have been identified with life-threatening NGLY1 deficiency. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - November 8, 2023 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

The Wonderfully Shrunken Cas13
Scientists removed unnecessary sections of the Cas13 enzyme, creating a mini-enzyme that works and fits with other CRISPR elements into a single gene therapy vector. (Source: The Scientist)
Source: The Scientist - November 7, 2023 Category: Science Tags: News News & Opinion Source Type: news

Platform Manufacturing Processes for pDNA, mRNA, LNP and Fill/Finish in Clinical mRNA Therapies, Upcoming Webinar Hosted by Xtalks
In this free webinar, gain insights into the rapidly evolving landscapes of mRNA and cell/gene therapies and the common challenges they share. Attendees will learn how innovative solutions are driving the development, scaling and commercialization of these therapies. The featured speaker... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 7, 2023 Category: Pharmaceuticals Tags: FVT Source Type: news

DMD Gene Therapy Safe, Effective at 4 Years DMD Gene Therapy Safe, Effective at 4 Years
Four years after a single dose of SRP-9001 gene therapy, four children with Duchenne muscular dystrophy show sustained stabilization of motor function that would otherwise show expected decline.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 6, 2023 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news

Gene Therapy Controls Diabetic Eye Disease for at Least a Year
(MedPage Today) -- SAN FRANCISCO -- A form of gene therapy stabilized or improved diabetic retinopathy (DR) for up to a year, particularly in patients with nonproliferative (NP) DR, a small clinical study showed. Overall, about 40% of patients... (Source: MedPage Today Endocrinology)
Source: MedPage Today Endocrinology - November 6, 2023 Category: Endocrinology Source Type: news

For A Change There ’s Optimism About A Gene Therapy’s Commercialization—Vyjuvek For Wound Care
Cantor Fitzgerald said it expects a “very strong product launch” of a gene therapy made by Krystal Biotech, the topical gel Vyjuvek which treats a rare skin disease. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 6, 2023 Category: Pharmaceuticals Authors: Joshua Cohen, Senior Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news

QuickSTAT recognized as the best cell and gene therapy logistics provider in the Asia-pacific region by ACGTEA
Award honors exceptional performance from providers that facilitate the advancement of new therapies, R&D and manufacturing. NEW YORK, Nov. 5, 2023 /PRNewswire-PRWeb/ -- For the second consecutive year, QuickSTAT has been recognized by the Asia-Pacific Cell & Gene Excellence Awards... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 5, 2023 Category: Pharmaceuticals Tags: AWD Source Type: news

Gene Therapy Market size to grow by USD 4.61 billion from 2022 to 2027, North America to account for 39% of market growth- Technavio
NEW YORK, Nov. 3, 2023 /PRNewswire/ -- The gene therapy market size is expected to grow by USD 4.61 billion from 2022 to 2027. In addition, the momentum of the market will progress at a CAGR of 19.91% during the forecast period, according to Technavio Research. The market has been... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 3, 2023 Category: Pharmaceuticals Tags: MRR Source Type: news

The Eyes Have It: Gene Therapy Offers Hope For Glaucoma Treatment
According to the World Health Organization (WHO), glaucoma is the second leading cause of blindness globally. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 3, 2023 Category: Pharmaceuticals Authors: William A. Haseltine, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news

15th Annual SCOPE: Summit for Clinical Ops Executives Puts Spotlight on the Cell & amp; Gene Therapies Supply Chain & amp; Medical Device Trials, February 11-14, 2024, Orlando, FL
Over 3,300 executives will convene to explore solutions for various aspects of planning and managing clinical trials. NEEDHAM, Mass., Nov. 2, 2023 /PRNewswire-PRWeb/ -- In response to the rapid expansion of the cell and gene therapy (CGT) market valued at $7.28 billion in 2022 and a... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 2, 2023 Category: Pharmaceuticals Tags: TDS Source Type: news

Two Sickle Cell Disease Gene Therapies May Soon Be Approved By FDA
FDA will soon decide approval of exa-cel and lovo-cel for sickle cell disease. These may be one-time treatment options for SCD patients burdened by unmet needs. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 2, 2023 Category: Pharmaceuticals Authors: Joshua Cohen, Senior Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news

FDA Advisors Say New Gene Therapy for Sickle Cell Disease Is Safe
WEDNESDAY, Nov. 1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December.The FDA had already decided that the therapy,... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - November 1, 2023 Category: Pharmaceuticals Source Type: news