Second child death in China gene-editing trial raises safety and transparency concerns
A second child death linked to a gene-editing treatment in China has raised questions about clinical oversight, safety and transparency in the country’s rapidly developing advanced therapies sector.
HuidaGene was testing an experimental CRISPR-based therapy for Duchenne muscular dystrophy (DMD) in children through an investigator-initiated trial (IIT), that allows hospitals to initiate investigator-led studies without prior approval from national regulators. The company presented early data from its first patients at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in 2025, but subsequently went largely silent, with its clinical trial later listed as complete.
The company has now disclosed that a child participating in the study died, prompting renewed scrutiny of the trial’s conduct and reporting. The child was the fourth participant to receive the therapy, even though the results from the first two patients showed it not to be clear that the therapy worked at all. The development follows the recent disclosure of another child death in China following an experimental brain gene-editing treatment, in a case involving a six-year-old girl. That death occurred in March 2025 but again, was not publicly disclosed at the time.
The cases have intensified debate around the oversight of the investigator-led advanced therapy trials, particulary where regulatory pathways differ from those used in the US and Europe. For a field where interventions can carry irreversible biological consequences, the incidents underscore the importance of robust preclinical safety assessment and transparent reporting.
Read more via STAT News and Science
Hopstem announced the first closing of Series C to advanced iPSC-derived neural cell therapy
China-based Hopstem Biotechnology has announced the first closing of its Series C financing, bringing additional capital its iPSC-derived neural cell therapy platform. The financing includes new investment from Tasly Capital, Haibang Investment and Guangzhou Health Industry Investment, alongside continued backing from existing investor Longmen Capital. The company has not disclosed the amount raised in the first closing, with further closings expected.
Proceeds will support the clinical development and commercial-scale manufacturing of Hopstem’s lead candidate hNPC01. The allogeneic off-the-shelf therapy is an iPSC-derived forebrain neural progenitor cell product being developed across multiple neurological indications. The programme has gained significant regulatory momentum in the US this year. hNPC01 received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for chronic ischemic stroke in June, followed by IND clearances and Fast Track designations for chronic motor dysfunction associated with haemorrhagic stroke and traumatic brain injury. The programme is now being developed across three brain injury indications.
The financing reflects continued investor interest in scalable, off-the-shelf cell therapy platforms, particularly where companies can demonstrate both clinical progress and a credible route towards commercial manufacturing.
Read more via BioSpace
Skylark Bio doses first patient gene therapy trial for childhood deafness
Skylark Bio has dosed the first patient in a Phase 1/2 trial of SKY-GJB2, a one-time gene therapy targeting GJB2-related sensorineural hearing loss which is one of the most common forms of genetic deafness in children.
The therapy is designed to deliver a functional copy of the GJB2 gene to supporting cells in the cochlea, restoring production of connexin 26, a protein essential for normal hearing. The SONIX trial is expected to enrol around 10 children aged between nine months and seven years, with preliminary data anticipated before the end of 2026 and further readouts expected in 2027.
GJB2 mutations are estimated to account for around 15–20% of congenital hearing loss cases in the US, yet there are currently no approved treatments specifically for GJB2-related hearing loss. Patients typically rely on hearing aids or cochlear implants.
The programme also comes shortly after the FDA clearance of Regeneron's Otarmeni, the first gene therapy approved for congenital hearing loss, which targets OTOF-related disease. Skylark is using an AAV vector and intracochlear delivery approach, with manufacturing support from Forge Biologics.
The start of dosing highlights the expanding application of gene therapy in paediatric genetic disorders and the potential for validated delivery approaches to accelerate development across different genetic causes of disease.
Read more via pharmaphorum and Clinical Trials Arena
Epicrispr raises $90M Series C for epigenetic editing therapy
Epicrispr Biotechnologies has raised $90 million in an oversubscribed Series C financing to advance EPI-321, an experimental epigenetic editing therapy for facioscapulohumeral muscular dystrophy (FSHD). Unlike conventional gene editing approaches that alter DNA sequences, EPI-321 is designed to regulate gene expression without cutting or rewriting DNA. The therapy uses CRISPR-based technology to suppress DUX4, a gene whose abnormal expression drives muscle degeneration in FSHD.
The company has completed enrolment in an early-stage clinical study, with six-month follow-up data expected in early October. Early clinical data have indicated potential effects on muscle volume and biological markers associated with DUX4 suppression, although the dataset remains small and early.
The financing was co-led by Octagon Capital and Janus Henderson Investors, with participation from investors including Fidelity Management & Research, Sanofi Ventures and Cormorant Asset Management. The latest round brings Epicrispr's total funding since inception to approximately $213 million.
The deal provides another indication of growing investor interest in next-generation genetic medicines that can modulate gene activity without permanently changing the underlying DNA sequence. As the sector looks beyond first-generation editing technologies, epigenetic approaches could offer a potentially broader and more controllable route to treating genetic disease.
Read more via Biopharma Dive
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