Pediatric Neuron Disease Gets Its First Gene Therapy
(MedPage Today) -- The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - March 19, 2024 Category: American Health Source Type: news

FDA Approves Lenmeldy (atidarsagene autotemcel) for Children with Metachromatic Leukodystrophy
TOKYO and LONDON and BOSTON, March 18, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the U.S. Food and... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - March 18, 2024 Category: Drugs & Pharmacology Source Type: news

Bluebird secures up to $175M in debt financing deal
The agreement includes an initial tranche totaling $75 million, and additional capital dependent on bluebird bio hitting certain commercial milestones on the rollout of its FDA-approved gene therapies. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 18, 2024 Category: Biotechnology Authors: Hannah Green Source Type: news

Science Continues To Outpace Commercialization Of Cell And Gene Therapies
The continued lack of robust revenue for approved cell and gene therapies is a reminder that being innovative doesn ’t necessarily translate into profitability. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - March 4, 2024 Category: Pharmaceuticals Authors: Joshua Cohen, Senior Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news

Novartis presents new data on safety and efficacy of Zolgensma
Novartis today presented new data that continue to support the clinical benefits of Zolgensma® (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease modifying therapy at the time of treatment. (Source: World Pharma News)
Source: World Pharma News - March 4, 2024 Category: Pharmaceuticals Tags: Featured Novartis Business and Industry Source Type: news

Xtalks Announces its Life Science Webinar Calendar for March 2024
Upcoming free, educational webinars from Xtalks will feature topics on biomarkers, cell and gene therapy, clinical trials, commercialization & HEOR, drug discovery & development, fundamental research, laboratory technology, medical device, pharmaceutical, pharma manufacturing & supply... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 1, 2024 Category: Pharmaceuticals Tags: SVY Source Type: news

UCLA Samueli to lead $4 million cell research project funded by Chan Zuckerberg Initiative
Key takeawaysThe grant will fund a three-year collaboration among researchers at UCLA, USC and Caltech to advance cell and tissue engineering technologies.The project, led by UCLA ’s Dino Di Carlo, will engineer, manipulate and analyze cell-to-cell interactions that underlie complex multicellular systems in the body.Di Carlo said he aims for the collaboration to develop into a long-term partnership across institutions to advance biotechnology in Los Angeles.The Chan Zuckerberg Initiativetoday announced a $4 million grant to support research led by the UCLA Samueli School of Engineering that will examine cellular behavio...
Source: UCLA Newsroom: Health Sciences - February 29, 2024 Category: Universities & Medical Training Source Type: news

Gene therapy company launched by parents, Nobel Prize winner doses 1st patient in ultra-rare disease
Children in the study will be followed for five years. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - February 28, 2024 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

Bio-Rad Launches Vericheck ddPCR Replication Competent Lentivirus and Replication Competent AAV Kits for Cell and Gene Therapy Production
Rapid, Sensitive, and Validated Detection of Viral Replication Genes Using Droplet Digital™ PCR During Cell and Gene Therapy Production (Source: The Scientist)
Source: The Scientist - February 27, 2024 Category: Science Tags: The Scientist The Marketplace Source Type: news

Asia-Pacific Cell and Gene Therapy Manufacturing QC Market Growth Report 2024: Market to Reach $2.233 Billion by 2033 - Focus on China, Japan, South Korea, Australia, India
DUBLIN, Feb. 27, 2024 /PRNewswire/ -- The "Asia-Pacific Cell and Gene Therapy Manufacturing QC Market: Analysis and Forecast, 2023-2033" report has been added to ResearchAndMarkets.com's offering. The comprehensive study showcases an anticipated compound annual growth rate (CAGR) of... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 27, 2024 Category: Pharmaceuticals Tags: MRR Source Type: news

Miltenyi Biotec launches India operations, to open Centre in Hyderabad
German biomedical discovery provider Miltenyi Biotec is launching operations in India, opening its first office and setting up a Centre of Excellence. The CoE will offer training in cell and gene therapy and contribute to the vibrant life sciences ecosystem by supporting local development and manufacturing in India. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - February 27, 2024 Category: Pharmaceuticals Source Type: news

Krystal Biotech shares up sharply after it logs revenue from newly approved product
It's a gene-therapy treatment of a rare skin disease called dystrophic epidermolysis bullosa (DEB). (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 26, 2024 Category: Biotechnology Authors: Paul J. Gough Source Type: news

New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle
The approval of two gene therapies to treat sickle cell disease has given hope to patients who suffer from the debilitating disease, which overwhelmingly affects Black people and people of color. Health officials now face a challenge in finding a way to provide equitable access to the costly…#michaelgoodwin #bluebirdbio #lyfgenia #juliekanter #adultsickleclinic #medicaid #katemcevoy #vertex #fda #merck (Source: Reuters: Health)
Source: Reuters: Health - February 24, 2024 Category: Consumer Health News Source Type: news

ClearPoint gets FDA nod for latest software, highlights study
The U.S. Food and Drug Administration (FDA) has cleared ClearPoint Neuro's ClearPoint 2.2 Software with Maestro Brain Modeling. The company is highlighting the first-in-human cases and the publication of a validation study for the software, published in NeuroImage. The ClearPoint system uses MRI guidance for peri-procedural segmentation of the cortical structures of the brain to identify both targets and safety zones for cell and gene therapy delivery, laser ablation, biopsy, and deep brain stimulation. The first clinical cases using ClearPoint 2.2 were completed successfully in the first quarter of 2024, and the company...
Source: AuntMinnie.com Headlines - February 22, 2024 Category: Radiology Authors: AuntMinnie.com staff writers Tags: Industry News Source Type: news

Iovance stock soars on FDA approval of its melanoma gene therapy
Iovance has its gene therapy manufacturing plant at the Philadelphia Navy Yard. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 20, 2024 Category: Biotechnology Authors: John George Source Type: news