This month, two reported deaths linked to experimental gene-editing treatments in China have raised questions around clinical oversight and transparency. Meanwhile, the FDA has remained active across the sector, granting Ultragenyx its first gene therapy approval while extending the review of Capricor’s Duchenne Muscular Dystrophy cell therapy and placing REGENXBIO’s Hunter syndrome programme on clinical hold. Elsewhere, Sangamo's bankruptcy auction has demonstrated the continuing value of advanced therapy platforms and late-stage assets, while Bristol Myers Squibb's decision to end its partnership with Cellares highlights the challenges of translating next-generation manufacturing technologies into established commercial processes.
Here are the developments you need to know from August.
Safety and transparency under scrutiny in China
Two deaths involving children who received experimental gene-editing treatments in China have intensified discussion around the oversight of investigator-initiated trials (IITs). HuidaGene disclosed that a child died during a CRISPR-based DMD trial, the fourth patient to receive a therapy whose first two patients showed unclear efficacy. Separately, an investigation by Science and Retraction Watch reported the death of a six-year-old girl seven days after an experimental base-editing infusion. Neither death was disclosed at the time, both cases underscore the field’s need for rigorous preclinical safety assessment and transparent reporting.
FDA activity from approvals, extensions and clinical holds
Ultragenyx secured its first FDA approval, with accelerated clearance Genglycos, a one time AAV8 gene therapy for glycogen storage disease type 1a, just over a year after a manufacturing-related rejection for another candidate.
Capricor Therapeutics received a three-month FDA review extension for its Duchenne cell therapy deramiocel, pushing the PDUFA date to 22 November after an advisory committee voted 9-3 against efficacy in July.
REGENXBIO’s Hunter syndrome gene therapy RGX-121 was placed on clinical hold after spinal MRI abnormalities emerged in five asymptomatic participants treated three to six years-ago, a reminder that safety signals for gene therapies can resurface years after a single administration.
Sangamo sells assets and BMS end partnership with Cellares
Following its bankruptcy filing, Sangamo Therapeutics completed a competitive auction of its core assets. PTC Therapeutics acquired the Fabry disease programme ST-920 for $11 million upfront plus up to $100 million in milestones, while Eli Lilly paid $50 million for Sangamo’s delivery and gene-editing programmes.
Elsewhere, Bristol Myers Squibb ended its manufacturing partnership with Cellares, resulting in a planned workforce reduction of around 100 employees. The partnership, announced in 2024 and valued at up to $380 million, was intended to support the use of Cellares' automated Cell Shuttle platform for CAR-T manufacturing. BMS said that, following a comprehensive evaluation, the Cell Shuttle could not meet the requirements necessary to manufacture commercial Breyanzi.
Cellares strongly disputes this characterisation. In a statement provided to Phacilitate, the company said that it does not discuss confidential customer programme details publicly but noted that the Cell Shuttle has manufactured GMP drug product for an FDA-regulated clinical programme, with doses meeting release specifications and being administered to patients.
In case you missed it: The future of Europe’s ATMP ecosystem
In July, Phacilitate partnered with BIOCAT to bring together leaders from across biotech, policy and industry to discuss the future of Europe's advanced therapy ecosystem.
The discussion explored Europe's execution gap, the potential impact of the EU Biotech Act and 2026 Horizon Europe Health Calls, and the structural barriers limiting the region's competitiveness.
Featuring Montse Daban Marin, Miguel Forte, Paolo Morgese, Jacqueline Barry and Jim Lund, the webinar examined the practical actions needed to accelerate innovation, strengthen manufacturing and improve patient access across Europe.
Watch the full discussion on demand: Europe's ATMP Roadmap Webinar
Meet the ATE Innovation Exchange finalists
We also announced the six finalists for the Advanced Therapies Europe Innovation Exchange, taking place on 9 September.
The finalists - Harmonix, Alterna Therapeutics, StemSight, CureAge Therapeutics, CREATe Therapeutics and Korecyte Bio represent a diverse range of emerging approaches, spanning CNS delivery, RNA therapeutics, regenerative ophthalmology, genetic medicines and next-generation cell and gene therapies.
The companies will pitch their technologies live to a panel of investors and dealmakers, bringing together founders, investors, pharma executives and strategic partners to explore the next generation of advanced therapy innovation.
Find out more about the Innovation Exchange
Join us at Advanced Therapies Europe
There is now less than a week to go until Advanced Therapies Europe, taking place 7–9 September 2026 at the InterContinental Hotel, Barcelona.
Join Europe's advanced therapies community for three days of discussion, collaboration and insight across clinical development, manufacturing, scientific innovation, investment and commercialisation.
View the full ATE speaker list
Register for Advanced Therapies Europe
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