Voyager Therapeutics hasn’t yet disclosed which anti-amyloid antibody it’s vectorizing, only telling analysts that it’s been exploring “several options.” (Dr_Microbe/ Getty images)
Both presence of the same APOE4 gene variant that predisposes people to Alzheimer’s and the size of the initial dose have been linked to the development of ARIA. There is “biologic rationale” to suggest that gene therapy wouldn’t confer the same degree of risk, Voyager Chief Scientific Officer Todd Carter, Ph.D., said on the earnings call.
“In a gene therapy approach, the anti-amyloid antibodies are steadily secreted by cells in the central nervous system, and thus we’d be avoiding high antibody concentrations that necessarily follow intravenous antibody infusions,” he said. “Moreover, the antibody would first engage the beta amyloid deposited in and around beta amyloid plaques rather than around blood vessels, thus avoiding ARIA.”
Still, some risk does remain, Sandrock acknowledged on the call. The company is considering several mitigation strategies to handle ARIA if it does arise, including the use of a small molecule to control gene expression.
Voyager hasn’t yet disclosed which anti-amyloid antibody it’s vectorizing, instead telling an analyst on the call that the company has been exploring “several options.”
Source: https://www.fiercebiotech.com/research/voyager-therapeutics-adds-anti-amyloid-gene-therapy-alzheimers-pipeline
Check out our AAV CDMO service to expedite your gene therapy research
PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
Related News
MeiraGTx’s AAV Gene Therapy Shows Promising Results for Parkinson’s
MeiraGTx has reported encouraging results from a midphase trial of its investigational gene therapy, AAV-GAD, for treating Parkinson’s disease. The company is now in discussions with regulators to advance the program to Phase III clinical trials.Significant...
[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...
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...
Related Services
Plasmids GMP Services
Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.
READ MORE
AAV GMP Services
Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
READ MORE
Technology Platforms
PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.
READ MORE