Regenerative Medicine

Healthcare professionals are being advised to monitor patients and clinical trial participants who receive treatment for cancer with a chimeric antigen receptor (CAR) T cell therapy for the risk of secondary malignancies. In a commentary in The New England Journal of Medicine on 25 January, Nicole Verdun and Peter Marks write that while secondary cancers from these medicines appear to be relatively rare, they need to be identified. Drs Verdun and Marks are both senior officials at the US Food and Drug Administration. 

An experimental chimeric antigen receptor (CAR) T cell therapy intended for the treatment of multiple sclerosis received a fast track designation from the US Food and Drug Administration on 19 January, indicating its potential to address a major medical need. The therapy is being developed by Kyverna Therapeutics Inc, a biotech company incorporated in 2018 and located in Emeryville, California, US.

The UK government, through its Innovate UK agency, has increased its five-year funding commitment to the Cell and Gene Therapy Catapult, an incubator for researchers and small companies working on novel advanced therapy medicinal products (ATMPs). Announced on 8 January, the new financial award is £80 million which represents a 35% increase from the previous five year period. 

Galapagos NV continued to advance its plan to bring the manufacture of cell therapies closer to patients under an agreement with Thermo Fisher Scientific Inc to supply services to a new production site in California, US. Announced on 4 January, the collaboration is the latest iteration of a strategy being rolled out by Paul Stoffels, a former chief scientific officer at Johnson & Johnson Inc, who became the Galapagos CEO on 1 April 2022.

Oxford Biomedica Plc of the UK has acquired ABL Europe SAS of France giving it a significant presence on the continental European market as a contract development and manufacturing organisation (CDMO). ABL Europe is owned by Institut Mérieux, a holding company controlled by the Mérieux family of Lyon.

AstraZeneca Plc is to expand its pipeline of cell and gene therapies with the acquisition of the China-based company Gracell Biotechnologies Inc which has a chimeric antigen receptor (CAR) T cell therapy in development for multiple myeloma. The deal value is $1.2 billion which includes an upfront cash payment as well as potential contingent value payments linked to the achievement of an undisclosed regulatory milestone. The transaction is expected to close in the first quarter of 2024.

The US Food and Drug Administrations has issued approvals for two new cell-based gene therapies for the treatment of sickle cell disease, a rare but life-threatening blood disorder that affects millions of people throughout the world. The approvals, announced on 8 December, are for treatments which have demonstrated an ability to restore haemoglobin function in patients using different mechanisms of action.

Immunocore Holdings Plc, a developer of T cell receptor immunotherapies, has reported third quarter revenue for its first approved product, Kimmtrak (tebentafusp), for metastatic uveal melanoma. The therapy was approved in both the US and the EU in 2022. In the third quarter Kimmtrak delivered net sales of £49.7 million, an increase of 9% from a year earlier. Of this sum, £34.5 million was generated in the US; £15 million from Europe; and £0.2 million from the rest of the world. Since its first authorisation, Kimmtrak has been approved in more than 35 countries.

Freeline Therapeutics Holdings Plc, a UK gene therapy company, is to become a private company once again following an agreement with a portfolio company of Syncona, its majority shareholder and co-founder, to acquire all of the shares that Syncona doesn’t already own. Syncona co-founded Freeline in 2015 with the aim of developing gene therapies for chronic debilitating diseases. In 2020, Freeline went public with the issue of 8.8 million American Depositary Shares (ADSs) on the US Nasdaq market, raising $158.8 million.

AstraZeneca Plc is to work with gene editing technologies developed by Cellectis SA of France to identify up to 10 candidate cell and gene therapies for cancer, and autoimmune and rare diseases. Founded in 1999, Cellectis has technology for creating allogeneic chimeric antigen receptor (CAR) T cells by gene editing cells from a healthy donor. The editing technology disables genes that cause donor cells to attack the host.