[2024/10/11] Gene and Cell Therapy- weekly digest from PackGene
FeaturedNewsArticlesPackGene's NewsletterReceive the latest news and insights to your inbox.About PackGenePackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span...
Ultragenyx Provides Update on Stage 1 Cohorts in Phase 1/2/3 Cyprus2+ Study for Wilson Disease with AAV9 Gene Therapy
October 3, 2024 NOVATO, Calif. — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced promising results from the ongoing Phase 1/2/3 Cyprus2+ study evaluating UX701 gene therapy for Wilson disease. Early-stage data from Stage 1 of the trial revealed significant...
Diverse Genomes Make Medicine More Equitable
Africa has a high burden of chronic kidney disease, with prevalence rates much higher than in developed countries. Many patients don’t even realize they are living with the disease. But when Segun Fatumo, PhD, head of non-communicable diseases genomics at MRC Uganda,...
World-First CAR-T Therapy Using Donor Cells Sends Autoimmune Diseases into Remission
In a groundbreaking advancement, three individuals suffering from severe autoimmune diseases have achieved remission after receiving a revolutionary therapy using donor-derived immune cells. This pioneering treatment, which utilized bioengineered immune cells known as...
Novel gene therapy shows promise in targeting a mutation linked to epilepsy
Clearance of mutant protein & restoration of normal neuronal activity by Gapmer ASO. Credit: NUS MedicineResearchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) are working on a therapy that holds potential in treating...
Purespring nabs $105M to take gene therapy into the clinic as IgAN field matures
Purespring Therapeutics has reeled in £80 million ($105 million) to test two gene therapies in humans. The Sofinnova Partners-led Series B, disclosed Wednesday morning, will carry the London startup into an open-label Phase 1/2 next year for a kidney disease that has...
A Philadelphia biotech designed a first-of-its-kind therapy for rare cancer: Now, it waits for patients
Credit: Pixabay/CC0 Public DomainWithin the next couple of weeks, the first blood samples from patients will arrive at the loading dock of Adaptimmune, a biotechnology company headquartered on the northeastern edge of the Navy Yard. Their arrival will open a new...
Jim Wilson secures $100M for global approach aimed at reviving gene therapy field
A gene therapy pioneer thinks he has a solution to the field’s struggle to commercialize breakthrough cures: Go global. Jim Wilson’s Philadelphia-based company GemmaBio on Tuesday announced up to $100 million in funding to manufacture and test six gene therapies in...
2024 Nobel Prize in Medicine: Discovering the Secrets of microRNA in Gene Regulation
Victor Ambros discovered that the lin-4 gene produces a microRNA that negatively regulates the lin-14 gene, while Gary Ruvkun cloned the lin-14 gene and together they found that the lin-4 microRNA binds to a matching sequence in lin-14's mRNA to inhibit its function....
U.S. duo wins Nobel Prize in medicine for discovery of microRNA and gene regulation breakthrough
Victor Ambros and Gary Ruvkun won the Nobel Prize in Medicine.US scientists Victor Ambros and Gary Ruvkun won the Nobel Prize in Medicine on Monday for their discovery of microRNA and its role in how genes are regulated, solving a decades-old mystery, the Nobel...
Harnessing natural killer T cells to advance cancer immunotherapy for solid tumors
CAR-NKT cells target tumor cells in vitro. a, Representative flow cytometry plots illustrating the purity of NKT cells isolated from the spleen of iVα14-Jα18 transgenic mice, n = 12 with similar results. b,c, Representative flow cytometry histograms (b) and summary...
Ambros, Ruvkun Win the Nobel Prize in Physiology or Medicine for microRNAs
The 2024 Nobel Prize in Physiology or Medicine has been awarded jointly to Victor Ambros, PhD, and Gary Ruvkun, PhD, for the discovery of microRNA and its role in post-transcriptional gene regulation. Ambros and Ruvkun’s seminal discovery in 1993 revealed a new, and...
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