AAV Gene Therapy Revolutionizes Treatment for LCA1, Improving Vision Significantly
Innovative AAV Gene Therapy Dramatically Restores Vision in LCA1 Researchers have made a significant breakthrough in treating Leber congenital amaurosis type 1 (LCA1), a severe inherited retinal disease that causes early childhood blindness, by using an innovative...
Cellular Origins & 3P Innovation Partner to Streamline Cell and Gene Therapy Manufacturing
Cellular Origins, a TTP Company focused on enabling scalable, cost-effective, and efficient manufacture of cell and gene therapies (CGTs), has partnered with 3P innovation, an engineering company and supplier of automated fill-finish equipment, to integrate 3P...
Voyager Therapeutics Expands Gene Therapy Portfolio with Novartis License for Next-Generation Capsid
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company focused on neurogenetic medicines, has entered into a licensing agreement with Novartis AG (NYSE: NVS) for a novel capsid developed using Voyager’s TRACER™ capsid discovery platform. This license will...
BridgeBio ends development of gene therapy for adrenal gland disorder
BridgeBio said Tuesday night it won’t move forward with a gene therapy for a group of genetic disorders that impair adrenal glands and cause disruptions in producing cortisol. The California biotech made the decision because Phase 1/2 data were “not yet...
Gene Therapy for Inherited Deafness Approaches a Threshold
A hearing test for a newborn baby is routine. And if it reveals a hearing problem, the choice that is presented to those hoping to improve the baby’s hearing is also routine. There are usually two options: a cochlear implant or a hearing aid. But thanks to the efforts...
Validating CRISPR Preclinical Models with Multiomics
The power of CRISPR became definitively clear when the first CRISPR-based gene therapy, Casgevy (exa-cel), won regulatory approvals for the treatment of sickle cell disease. But CRISPR’s applications are not limited to therapeutics. CRISPR has also been used to create...
GenAssist Ltd Announced the First DMD Patient Dosed with its Base Editing Drug
SUZHOU, China, Sept. 9, 2024 /PRNewswire/ -- On September 06, 2024, GenAssist Ltd (GenAssist), announced the first DMD patient dosed with its base editing drug, GEN6050X injection, in an investigator-initiated trial (IIT). "This is the first-in-human trial for DMD...
NeoImmuneTech Presents Promising Interim Results of CAR-T Combination with its NT-I7 Asset at ESMO 2024
Adequate cell expansion and persistence remains a key challenge that limits the efficacy of chimeric antigen receptor T (CAR-T) cell therapies New clinical results presented at ESMO 2024 show NT-I7 treatment following CAR-T cell administration is safe and well...
Gene therapy offers promise for treating glaucoma and age-related macular degeneration
Credit: International Journal of Molecular Sciences (2024). DOI: 10.3390/ijms25168876Scientists from Trinity College Dublin have developed a highly promising gene therapy to treat glaucoma, a debilitating eye condition that can lead to complete vision loss, and which...
Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington’s disease
Products used in the paper Details Operation AAV vector packaging The AAV-mini-cmv-spCas9(AAV9-Cas9, AAV-Cas9), AAV-controlgRNA- RFP (AAV9-Ctrl-gRNA-RFP, AAV9-Ctrl-gRNA, Ctrl-gRNA) and AAV9-HTT-gRNA-RFP-20Q (AAV9-HTT-gRNA-20Q, HTT-gRNA-20Q) donor vectors were sent to...
Eli Lilly Joins Forces with AI Startup Genetic Leap in $409M Deal Centered Around RNA-Targeted Drug Discovery
Eli Lilly and Co. (LLY.N), a major global pharmaceutical company, has formed a research partnership with Genetic Leap, a startup that uses artificial intelligence (AI) and machine learning for drug discovery. The deal is valued at $409 million and focuses on...
Skyline Therapeutics Receives FDA Orphan Drug Designation for Gene Therapy for Retinitis Pigmentosa
BOSTON & SHANGHAI, September 2, 2024 – Skyline Therapeutics, a gene therapy company focused on innovative treatments for rare and severe diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108....
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