Regulatory Education for Industry (REdI) Annual Conference 2024: Biologics Track
The Biologics Track will focus on the developmental and regulatory topics relevant to cellular and gene therapies, tissue-engineered products, blood products, vaccines, and other biological products. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - April 8, 2024 Category: Drugs & Pharmacology Authors: FDA Source Type: news
OCGN Stock Alert: Why Is Ocugen Up 20% Today?
Biotechnology firm Ocugen (NASDAQ:OCGN) — which specializes in developing gene therapies to address blindness diseases — saw its shares move higher on an encouraging clinical development. An advisory committee responsible for overseeing safety matters approved Ocugen to use a medium dosage of its…#ocugen #dsmb #peterchang #dsmbchair #ocgn #amd #joshenomoto (Source: Reuters: Health)
Source: Reuters: Health - April 5, 2024 Category: Consumer Health News Source Type: news
Heart-1 Gene Therapy Trial Pauses Enrollment Heart-1 Gene Therapy Trial Pauses Enrollment
A phase 1 gene editing trial in patients with heterozygous familial hypercholesterolemia and atherosclerotic CVD was paused after abnormal lab results were seen.Medscape Medical News (Source: Medscape Cardiology Headlines)
Source: Medscape Cardiology Headlines - April 4, 2024 Category: Cardiology Tags: Cardiology Source Type: news
Gene Therapy, DNA's Past, RNA's Future: The Golden Era
Gene therapy made significant breakthroughs between 2000 and the 2010s, marking a golden era in medical history. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - April 2, 2024 Category: Pharmaceuticals Authors: William A. Haseltine, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
Groundbreaking gene therapy offers ‘glimmer of hope’ for children with rare diseases
Within the first few weeks after Lori and Lee Greenwood ’s daughter Noa was born, her parents knew that something was wrong. A lot of different things, actually. The infant cried inconsolably. She wouldn’t follow her parents with her eyes, leading to concerns about her vision. She struggled to gain weight and missed milestone after milestone. She was slow to even respond to her parents’ presence.
After weeks and months of having doctors dismiss it as the normal fits and fuss of a newborn, the… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 1, 2024 Category: Pharmaceuticals Authors: UMass Chan Medical School Source Type: news
Seattle Children's receives $50 million gift for pediatric cancer research
The gift from the William Lawrence and Blanche Hughes Foundation will be used to develop treatments for childhood cancers using cell and gene therapies. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 28, 2024 Category: Pharmaceuticals Authors: Rick Morgan Source Type: news
Gene therapy from RTP biotech improves vision in clinical study
After spending most of their lives nearly totally blind, a few patients can see objects for the first time after receiving a gene therapy being developed by a biotech in Research Triangle Park. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 28, 2024 Category: Biotechnology Authors: Zac Ezzone Source Type: news
How Hypertrophic Cardiomyopathy Progresses in Adults
Hypertrophic cardiomyopathy is the most common form of genetic heart disease worldwide. Researchers have estimated that up to 1 in 200 people have the condition, which is characterized by an abnormal thickening of the walls of the heart. This thickening can make it difficult for the heart to pump blood.
Hypertrophic cardiomyopathy can be a deadly disease, and there was a time when it was largely untreatable. But the last 20 years have witnessed a sea change in the condition’s management—a change that has led to an estimated 10-fold decrease in deaths.
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Source: TIME: Health - March 27, 2024 Category: Consumer Health News Authors: Markham Heid Tags: Uncategorized freelance healthscienceclimate Source Type: news
Gene Therapy, DNA's Past, RNA's Future: Early History
The period between the 1920s and 1950s was a time of significant progress and exciting discoveries in genetics and biomedicine (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - March 27, 2024 Category: Pharmaceuticals Authors: William A. Haseltine, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
Why Alzheimer's Drug Approval Delay? $3 Billion in DMD Gene Therapy; ADHD Prevalence
(MedPage Today) -- Why is donanemab, an investigational anti-amyloid agent for early Alzheimer's disease, facing an approval delay? (The Guardian)
Early use of erenumab (Aimovig) showed sustained efficacy compared with oral migraine preventives... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - March 26, 2024 Category: Neurology Source Type: news
Gene therapy maker bluebird to revise financials after accounting errors
(Source: Reuters: Health)
Source: Reuters: Health - March 26, 2024 Category: Consumer Health News Source Type: news
Auburn installs Siemens Magnetom 7T MRI scanner
Auburn University has installed Siemens' Magnetom 7T MRI scanner in its College of Engineering.
The scanner features dedicated radiofrequency sodium imaging coils and parallel transmit technology and will be used for brain and knee imaging, with a clinical focus on better understanding epilepsy, multiple sclerosis, and other disorders.Siemens' Magnetom 7T MRI scanner.PR Newswire
Projects currently underway that will use the scanner include research exploring post-traumatic stress disorder biomarkers in senior adults via spectroscopy, comparing brain connectivity between healthy populations with mental illness with a focu...
Source: AuntMinnie.com Headlines - March 26, 2024 Category: Radiology Authors: AuntMinnie.com staff writers Tags: Industry News Source Type: news
ANSI/PDA Standard on Cryopreservation of Cells for Cell and Gene Therapies Receives Complete Recognition Status by U.S. FDA CBER
The 2021 standard is included in a cohort of standards relevant to regenerative medicine therapies recognized either completely or partially by the Center for Biologics in 2023. BETHESDA, Md., March 25, 2024 /PRNewswire-PRWeb/ -- PDA today proudly announced at its 2024 Annual Meeting in... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 25, 2024 Category: Pharmaceuticals Source Type: news
Intrathecal Gene Therapy Shows Promise in Giant Axonal Neuropathy
MONDAY, March 25, 2024 -- Intrathecal gene transfer with scAAV9/JeT-GAN may result in some benefit for children with giant axonal neuropathy, according to a study published in the March 21 issue of the New England Journal of Medicine.
Diana X.... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - March 25, 2024 Category: Pharmaceuticals Source Type: news
First Gene Therapy for Children With Metachromatic Leukodystrophy Approved by FDA
FRIDAY, March 22, 2024 -- The U.S. Food and Drug Administration has approved Lenmeldy (atidarsagene autotemcel) as the first gene therapy for the treatment of children with metachromatic leukodystrophy (MLD).
Lenmeldy is a one-time, individualized... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - March 22, 2024 Category: Pharmaceuticals Source Type: news
