Seven rare disease programs make up the first round of participants in the US FDA’s ‘Operation Warp Speed’-style program. Sponsors will benefit from ‘rapid, ad hoc’ communication with the agency.

Technical challenges and lackluster investor sentiment about the near-term commercial potential of cell and gene therapies in neurological disorders may be holding the field back.

The US biotech’s TSHA-102 has shown surprisingly early signs of functional improvement in a single patient treated in its Phase I/II Rett syndrome trial, promoting a major stock surge supported by the news of a $150 private placement and regulatory updates.

In tandem with buying the rest of Kleo, Tonix licenses molecular degradation technology from Yale. Neurogene, University of Dundee partner on cell therapies for neurological disorders.