Novartis confirms nine cases of tumours in patients treated with gene therapy Zolgensma
Novartis has confirmed nine reported cases of tumours in patients previously treated with Zolgensma, its AAV based gene therapy for spinal muscular atrophy (SMA). Of these cases, two have shown evidence of vector integration into patients’ DNA, while the remaining seven have not been linked to the treatment.
The news surfaced via patient advocated on social media before any formal communication, with parents reporting the Novartis had written to families at the same time. The reports have prompted renewed scrutiny of the long-term safety profile of AAV -based gene therapies. The potential risk of tumorigenicity associated with vector integration is already included in Zolgensma’s prescribing information. However, with more than 6,000 patients having received the therapy, the reports reinforce the importance of continued long-term patient monitoring and transparent safety reporting.
Read more via BioSpace
Resolution Therapeutics announces interim Phase I/II data for regenerative macrophage therapy in end-stage liver disease
Resolution Therapeutics has reported six-month interim data from is Phase I/II EMERALD trial of RTX001, an autologous engineered regenerative macrophage therapy (RMT), in patients with end-stage liver disease (ESLD).
The interim analysis compared nine patients treated with RTX001 against 21 patients receiving standard of care in an external control study. Two of nine patients treated with RTX001 (22%) experienced the composite clinical outcome of death, liver transplantation or protocol-defined hepatic decompensation within six months of the first dose, compared with eight of 21 patients (38%) in the external control group, representing a 42% relative risk reduction. The treatment was well tolerated, with no infusion-related serious adverse reactions and no severe adverse events related to treatment.
Biomarker analyses demonstrated reductions in markers of inflammation and fibrogenesis, supporting the therapy’s proposed anti-inflammatory and anti-fibrotic mechanism. With enrolment now complete, Resolution plans to advance RTX001 towards a pivotal US trial, following a scheduled pre-IND meeting with the FDA in December 2026.
Read more via Resolution Therapeutics
Ultragenyx cuts 20% of its workforce and scales back R&D investment
Ultragenyx is reducing its workforce by approximately 20% and discontinuing several development programmes as part of a wider restructuring strategy aimed at achieving profitability in 2027. The company expects to reduce its annual R&D expenditure to approximately $350 million in 2027, around half of its 2025 spending.
The restructuring follows clinical setbacks across its pipeline, including the discontinuation of GTX-102 following unsuccessful Phase III results in Angelman syndrome and the decision to end development of UX701, an investigational gene therapy for Wilson's disease, following insufficient clinical benefit. The company will instead prioritise its six commercial products and a smaller number of development programmes, including DTX301 for ornithine transcarbamylase deficiency.
The decision reflects the increasing pressure on biotech companies to balance pipeline investment with financial sustainability, particularly where clinical setbacks and rising development costs affect the commercial viability of investigational therapies. Ultragenyx's approach also highlights a broader shift towards more selective R&D investment, with the company indicating that future pipeline expansion will rely on targeted internal research and smaller, strategically focused deals.
Read more via Fierce Biotech
CIRM awards $6.9 million grant to advance stem cell therapy for heart regeneration
The California Institute for Regenerative Medicine (CIRM) has awarded a $6.9 million grant to support the preclinical development of an investigational stem cell therapy designed to regenerate cardiac muscle following severe heart attacks. Developed by researchers at USC Stem Cell, the approach aims to address myocardial infarction with cardiogenic shock, a condition associated with significant heart damage and high mortality.
The therapy uses stem cell-derived cardiac muscle cells to replace damaged heart tissue, with the aim of restoring cardiac function rather than simply managing symptoms. The proposed treatment is designed as an off-the-shelf product, potentially enabling large-scale manufacturing and broader patient access. However, patients are expected to require immunosuppressive medication to prevent rejection.
CIRM funding will support further preclinical studies, manufacturing process development and clinical planning, with initial human trials targeting late 2027 at USC and the University of Washington. The programme represents a significant step towards translating stem cell-derived cardiac tissue into clinical application, while highlighting the importance of scalable manufacturing, product consistency and safety in advancing regenerative cell therapies.
Read more via News Medical
)