In rats, islets delivered through the liver’s portal vein had taken hold one month later, supporting the delivery route planned for clinical testing.
Developers will have access to immune profiling and detailed donor assessments, intended to help them evaluate the starting material used to make donor-derived cell therapies.
The approval was based on a trial comparing 17 treated children with 27 untreated peers, with follow-up extending to nearly eight years and shipments expected within 30 to 60 days.
The designation was based on early U.S. trial data in spinal cord injury patients, where this non-replicating HSV-1 gene therapy is being tested as a single-course treatment aimed at reducing bladder overactivity without impairing normal function.
Kolon TissueGene’s phase 3 trial found its osteoarthritis therapy did not improve knee pain or function versus placebo, leaving the biotech to await results from a second late-stage study before deciding the program’s future.
The California agency is hoping to accelerate the cell & gene therapy industry amid uncertainty about federal funding.
REGENXBIO said the FDA will consider existing longer-term data for NAVSUNLI under the accelerated approval pathway without requiring new studies, allowing the company to target a BLA resubmission in Q3 2026.
The company is working on iPSC-derived, AAV, and small molecule therapies for neurodegenerative, orthopedic, and blood-based disorders, with the help of OpenAI's models which seem to be accelerating life science research.
The partnership hopes to speed up the scalable development of SC451 for type 1 diabetes by standardizing treatment protocols, improving clinical delivery and post-care, and preparing the therapy for broader use across care sites.
The partnership combines Dark Horse's regulatory and development consulting with KunTuo's on-the-ground clinical trial execution to help companies launch studies faster and generate data to support regulatory submissions.
The funding will support Latus Bio’s lead gene therapy programs in Huntington’s and CLN2 disease while advancing its low-dose AAV delivery platform designed to make gene therapies safer, more scalable, and accessible to broader patient populations.
Regeneron said it will reduce Medicaid and future U.S. drug prices in line with other developed countries, enable direct discounted purchases of Praluent through TrumpRx.gov, and continue investing in domestic R&D and manufacturing under its agreement with the U.S. government.
The recommendation is backed by Phase III data showing that Novartis’ one-time gene therapy improved motor function in people aged two and older with SMA and could lessen reliance on ongoing chronic treatment.
The partnership aims to streamline production and quality testing of gene-edited hematopoietic stem cell therapies for HIV and rare diseases.
The new open source gene editing services are ready to help iPSC developers create off-the-shelf allogeneic cell therapies at scale.
Dr. Marty Makary and Dr. Vinay Prasad publish new article in New England Journal of Medicine, proposing a new pathway. Here are the details.
NurExone's SCI candidate showed 100% gait recovery in rat models, and they're gearing up for human trials hopefully in 2026. Enjoy the interview!
As some countries move to deregulate regenerative medicine, others are looking to batten the hatches. Where the USA fits on that spectrum is anybody's guess right now.
What can the world learn from Japan’s regenerative medicine laws? We sat down with the non-profit that helped design them to find out.
The agreement gives Adicet access to MaxCyte’s technology for developing scalable, non-viral gene-edited cell therapies.
The new grants will support diverse early-stage research projects, aiming to advance regenerative medicine tools and future therapies.
Researchers are testing a novel CRISPR-based therapy for FSHD, with participants monitored for safety and long-term outcomes.
The companies will collaborate to leverage gene editing and GMP iPSC manufacturing to hopefully deliver new therapies.
Klotho Neurosciences’ gene therapy is advancing plans for ALS clinical trials next year.
Researchers are exploring whether SIRT1 gene therapy can protect human retinal cells from damage linked to optic neuropathies.