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...
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...
Eli Lilly to Invest $4.5 Billion in New Drug Manufacturing and Research Facility
Eli Lilly is making a major investment to enhance its drug manufacturing capabilities, announcing plans to build a $4.5 billion facility in Lebanon, Indiana. The new center, named the Lilly Medicine Foundry, will serve a dual purpose: developing innovative...
Avidity Biosciences Announces FDA Removed Partial Clinical Hold on Delpacibart Etedesiran (del-desiran/AOC 1001)
SAN DIEGO, Oct. 3, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug...
Quell Therapeutics Taps eXmoor Pharma to Manufacture CAR-Tregs
U.K. developer Quell Therapeutics hired eXmoor Pharma to make supplies of its pipeline of novel immunosuppressive cell therapies—called CAR-Tregs—at its new cell and gene therapy facility. Quell recently teamed with fellow U.K.-based eXmoor in a “strategic...
Study reveals key role of TRIO gene in epileptic encephalopathies
Defective migration of MGE-derived cortical INs in Trio–/– embryos. Credit: Molecular Psychiatry (2024). DOI: 10.1038/s41380-024-02742-yWhat are the molecular and cellular mechanisms by which some babies develop epileptic encephalopathies and autism spectrum disorder?...
Pfizer Withdraws Sickle Cell Disease Treatment Worldwide Amid Safety Concerns
On September 25, 2024, Pfizer Inc. announced the voluntary withdrawal of all lots of its sickle cell disease (SCD) treatment from global markets. Pfizer is halting the distribution and discontinuing all clinical trials and expanded access programs involving the...
FDA Grants Special Therapy Designation to BridgeBio for Achondroplasia Treatment
September 17, 2024 - BridgeBio Pharma, Inc. BridgeBio Pharma announced that the U.S. Food and Drug Administration (FDA) has granted a special therapy designation to its oral treatment under development for children with achondroplasia, the most common form of...
EydisBio Receives FDA Orphan Drug Designation for TAK1 Inhibitor for the Treatment of Systemic Sclerosis
DURHAM, N.C., Sept. 25, 2024 /PRNewswire/ -- EydisBio, Inc. is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EYD-001, its highly selective and potent, orally bioavailable TAK1 inhibitor for the treatment of...
NanoMosaic Transforms Cell and Gene Therapy Analytics with Advanced Quantification and Genome Mapping Capabilities. World-Renowned Expert Dr. Guangping Gao to Join Nanomosaic Scientific Advisory Board
WALTHAM, Mass., Sept. 24, 2024 /PRNewswire/ -- Nanomosaic, the multiplex and multi-omic marker analysis company, today announced major technical and commercial milestones for its Tessie system and assays optimized for gene therapy applications, evidencing the need for...
Vicebio and its molecular clamp vaccine raise big to tackle respiratory viruses
Today, Vicebio, a UK-based vaccine company completed a $100 million series B round led by TCGX with investments from Goldman Sachs Alternatives, Avoro Ventures, venBio, and participation from UniQuest and founding investor Medicxi. With its molecular clamp technology...
IVIEW Therapeutics Completes Patient Recruitment for Phase 1/2 Trial of IVW-1001, Exploring New Innovations in Dry Eye Disease and Gene Therapy
Cranbury, N.J., September 17, 2024 – IVIEW Therapeutics Inc., a clinical-stage biotechnology company known for advancing innovative therapies, including gene therapy, has successfully completed patient recruitment for its Phase 1/2 clinical trial evaluating IVW-1001....
Popular Services
AAV Packaging Service
AAV Analytical Service
Vector Design