Guts & Glory: Both of Cynata’s iPSC Trials Flop, GVH Phase 2 and KOA Phase 3
Sad day for the industry, here's a summary of the CEO/Executive Chairman's readouts of the trials.
Regulatory
FDA Selects Seven Participants for PreCheck Pilot Program to Advance US Drug Manufacturing
The program is intended to accelerate US drug manufacturing scale-ups and address unmet patient needs. Two companies familiar to our regenerative readers, Cellares and Fujifilm Biotechnologies, made the list!
Orca Bio Nabs FDA Approval of TREGZI, its Cell Therapy for Allogeneic Transplant in Adults with Blood Disorders
Following Phase 3, TREGZI, known as Orca-T, had higher graft-versus-host disease-free survival at one year in patients receiving conventional blood stem cell transplants.
Steminent Filing for Japanese Approval of its Mesenchymal Stem Cell Therapy for Spinocerebellar Ataxia
The company is hoping to be the 2nd Taiwanese cell therapy approved abroad.
UniXell Biotechnology Nabs IND Clearance for its iPSC Parkinsons Therapy, UX-DA003
The Chinese company also received an IND in China a few weeks ago. Supported by good preclinical data, they'll advance clinical trials in both countries soon.
FDA Unveils its Plan for Operation TrialBlazer, Hoping to Streamline Drug Development Timelines
The FDA outlined a broad set of new pilot programs, guidance updates, and support resources to streamline drug development from first-in-human studies through pivotal trials while reducing unnecessary regulatory burden and improving...
FDA Reverses Decision on Regenxbio’s Gene Therapy for Hunter’s Syndrome, Supporting BLA Resubmission for Accelerated Approval
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.
Georgia Joins Utah, Florida, and Other States in “Legalizing” Birth Tissue-Derived Stem Cell Therapies
Georgia’s House Bill 1275 allows physicians and others to provide non-FDA-approved stem cell therapies derived from ethically sourced birth tissues, while imposing facility standards, disclosure rules, and patient consent requirements.
Cynata gains EU endorsement for Graft-vs-Host iPSC Study Plans
The Australian biotech company is hoping to tackle aGvHD in Europe, which appears to have twice as many cases as the USA.
Interview: From Zero to One, How Terumo’s Ecosystem Blueprint Is Helping Scale Cell Therapies in Asia
Cell and gene therapies are advancing quickly, but the systems needed to deliver them at scale are still lagging behind. Terumo’s new Ecosystem Blueprint aims to bridge that gap in Asia-Pacific by...
