Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.

2023 saw a number of gene therapy companies go to the wall, but those that can combine clinical significance and commercial success can build sustainable businesses.

Novartis revealed detailed terms and a 4 October spin-off date for Sandoz’s standalone debut. Also, MIRA launched a small IPO, but two firms plan to go public in SPAC mergers. Also, Mirati’s $345m offering leads follow-ons offerings and Sangon Biotech raised $290m in venture capital.

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.