FDA Streamlines CGT Development, China Scrutiny and In Vivo CAR-T Momentum Builds – Weekly News Round-up

FDA proposes new pathway to accelerate cell and gene therapies

The US FDA has released draft guidance designed to help accelerate the development and approval of cell and gene therapies by reducing unnecessary CMC requirements.

The guidance encourages sponsors to leverage existing scientific knowledge and priori platform experience rather than repeatedly generating data that may already be well understood within the field. Under the proposal, developers may be able to reference established manufacturing knowledge, platform technologies and previously generated evidence when preparing regulatory submissions.

The guidance arrives at a critical time for the advanced therapies sector. As more cell and gene therapies progress into late-stage development and commercialisation, regulators are increasingly seeking ways to balance rigorous oversight with the need to bring transformative treatments to patients more efficiently.

While the guidance remains in draft form and will undergo public consultation, it reflects a broader FDA effort to modernise regulatory frameworks for emerging therapeutic modalities and could significantly influence future development strategies across the sector.

Read more via Biospace and FDA

US lawmakers seek tighter controls on China biotech partnerships

Geopolitical tensions continue to influence the biotechnology industry, with US lawmakers advancing new proposals aimed at restricting investments and partnerships involving Chinese biotechnology companies.

A bipartisan group in Congress has introduced the Biotechnology Innovation and National Security Act (BINSA), legislation that would expand government oversight of certain biotechnology transactions involving China. The proposal builds on broader concerns around national security, intellectual property protection, supply chain resilience, and the strategic importance of biotechnology innovation.

If enacted, the legislation could increase scrutiny of licensing deals, research collaborations, acquisitions, and investment agreements between US organisations and Chinese biotech firms. Supporters argue that tighter controls are necessary to protect critical technologies and reduce dependence on overseas capabilities in strategically important sectors such as genomics, biomanufacturing, and advanced therapeutics.

While some stakeholders support measures to strengthen domestic biotechnology infrastructure, others warn that overly restrictive policies could limit scientific collaboration, reduce investment opportunities, and slow innovation in areas where global partnerships have historically played an important role.

Read more via Biopharma dive and Fierce biotech

Kelonia reports encouraging in vivo CAR-T results at ASCO

Among the clinical highlights emerging from ASCO 2026, Kelonia Therapeutics presented early data supporting the potential of its in vivo CAR-T platform for the treatment of multiple myeloma.

All 18 patients with relapsed or refractory multiple myeloma treated with KLN-1010 had no cancer in their bone marrow one month after treatment, as determined by a minimal residual test. All patients able to be assessed six months post-treatment remained MRD-negative, and the patient with the longest follow-up also remained clear of disease.

Unlike conventional CAR-T therapies, which require patient cells to be collected, engineered outside the body, and then reinfused, Kelonia's approach aims to generate CAR-T cells directly within the patient through targeted gene delivery. The promise of in vivo CAR-T has attracted growing interest across the advanced therapies industry due to its potential to simplify manufacturing, reduce treatment costs, and improve patient accessibility. Eliminating the complex ex vivo manufacturing process could help address some of the major operational and logistical challenges that have limited broader CAR-T adoption.

The strong response data presented at ASCO further reinforces growing confidence that in vivo cell engineering could emerge as one of the next major innovations in the cell therapy space. As multiple companies race to validate their platforms clinically, the coming years are likely to determine whether these technologies can deliver on their promise of making personalised cell therapies more scalable and widely accessible.

Read more via First Word Pharma and Pharmaceutical Technology