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
Genprex Announces Closing of $6.5 Million Registered Direct Offering Priced At-the-Market Under Nasdaq Rules
AUSTIN, Texas, March 22, 2024 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the closing on March 21, 2024 of its... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 22, 2024 Category: Pharmaceuticals Tags: OFR Source Type: news
A Journey Into the Brain
With the help of directed evolution, scientists inch closer to developing viral vectors that can cross the human blood-brain barrier to deliver gene therapy. (Source: The Scientist)
Source: The Scientist - March 22, 2024 Category: Science Tags: News News & Opinion Source Type: news
The World ’s Most Expensive Drug Is Now a $4.25 Million Gene Therapy
A new gene therapy for an ultra-rare disease will have a wholesale cost of $4.25 million, making it the world’s most expensive drug.
The one-time treatment, Lenmeldy, won U.S. regulatory approval on Monday to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD.
MLD is a fatal disease in which infants sometimes start to lose the ability to walk and talk. Orchard Therapeutics said the drug’s price “reflects its clinical, economic and societal value” in a statement Wednesday.
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Source: TIME: Health - March 21, 2024 Category: Consumer Health News Authors: Gerry Smith/Bloomberg Tags: Uncategorized healthscienceclimate wire Source Type: news
Experimental gene therapy for giant axonal neuropathy shows promise in NIH clinical trial
An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH). Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age. Fourteen children with GAN, ages 6 to 14 years, were treated with gene transfer therapy at the NIH Clinical Center and then followed for about six years to assess safety. (Source: World Pharma News)
Source: World Pharma News - March 21, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Spinal Delivery of Gene Therapy Promising for Neurodegenerative Disease
(MedPage Today) -- Gene therapy delivered for the first time via lumbar puncture showed promise of benefit in slowing progression of giant axonal neuropathy (GAN) in a phase I clinical trial.
The slope of change in motor function went from -7... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - March 21, 2024 Category: Neurology Source Type: news
Experimental gene therapy for giant axonal neuropathy shows promise in NIH clinical trial
Treatment for rare childhood disease was well tolerated and slowed loss of motor function. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - March 20, 2024 Category: American Health Source Type: news
Orchard ’s newly approved gene therapy is most expensive drug in history
The company has put forward a record-setting price for a drug to treat a rare and fatal disease for which there has previously been no treatment options beyond supportive and end-of-life care. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 20, 2024 Category: Biotechnology Authors: Hannah Green Source Type: news
Rare genetic disease therapy becomes world’s most expensive drug at $4.25mn
Lenmeldy gene therapy treats MLD, a condition that attacks the central nervous system of young children (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - March 20, 2024 Category: Pharmaceuticals Source Type: news
Stem Cell Editing Repairs Severe Immunodeficiency
Scientists hoping to treat immunodeficiencies using gene therapy have found a way to edit stem cells in mice without disrupting gene regulation. (Source: The Scientist)
Source: The Scientist - March 20, 2024 Category: Science Tags: News News & Opinion Source Type: news
TSHA Stock Earnings: Taysha Gene Therapies Beats EPS, Beats Revenue for Q4 2023
Taysha Gene Therapies (NASDAQ: ) just reported results for the fourth quarter of 2023. InvestorPlace Earnings is a project that leverages data from TradeSmith to automate coverage of quarterly earnings reports. InvestorPlace Earnings distills key takeaways including earnings per share and revenue,…#tradesmith (Source: Reuters: Health)
Source: Reuters: Health - March 20, 2024 Category: Consumer Health News Source Type: news
FDA OKs Gene Therapy for Metachromatic Leukodystrophy FDA OKs Gene Therapy for Metachromatic Leukodystrophy
Atidarsagene autotemcel (Lenmeldy) is approved for presymptomatic late infantile, presymptomatic early juvenile, or early symptomatic early juvenile MLD.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - March 19, 2024 Category: Neurology Tags: Neurology & Neurosurgery Source Type: news
PTC Therapeutics Announces the Submission of a BLA to the U.S. FDA for Upstaza (eladocagene exuparvovec) for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
SOUTH PLAINFIELD, N.J., March 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - March 19, 2024 Category: Drugs & Pharmacology Source Type: news
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 Orchard ’s gene therapy for children with rare genetic disease
This decision marks the first FDA-approved treatment for children with a rare genetic disease called metachromatic leukodystrophy (MLD) and Orchard's entry into the U.S. drug market. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 18, 2024 Category: Pharmaceuticals Authors: Hannah Green Source Type: news
