Regenerative Medicine

Two gene therapy approvals

Country
United States

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 gives Q3 sales

Country
United Kingdom

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 to go private

Country
United Kingdom

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.

AZ partners with Cellectis

Country
United Kingdom

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.

CORRECTION: SSI Strategy buys NDA Group

Country
United States

In an article published on www.mednous.com on 21 August 2023 entitled SSI Strategy buys NDA Group, the leadership of the new group has been corrected to show that Douglas Locke, Chief Executive Officer of SSI Strategy, has been named CEO of the combined group. A corrected version follows.

Tenpoint to treat vision loss

Country
United Kingdom

A new company has launched in the UK with the goal of using both ex vivo and in vivo cell therapy approaches for the treatment of ocular diseases. Tenpoint Therapeutics Ltd disclosed its plans on 12 July and concurrently announced a $70 million Series A financing led by F-Prime Capital, Sofinnova Partners, and British Patient Capital. The UCL Technology Fund was also part of the financing syndicate.

Gene therapy for haemophilia A

Country
United States

The first gene therapy to treat severe haemophilia A was approved by the US Food and Drug Administration on 30 June, adding a novel treatment to the existing group of drugs for the bleeding disorder. The therapy, Roctavian, is an adeno-associated virus (AAV) vector-based therapy intended to be used as a one-time treatment. It is administered as a single dose by intravenous infusion.

Cell therapy for diabetes

Country
United States

The US Food and Drug Administration approved the first allogeneic cell therapy for type 1 diabetes on 28 June, making it possible for people with severe hypoglycaemia to receive an additional treatment for achieving targeted blood glucose levels. Lantidra is an allogeneic pancreatic islet cell therapy made from deceased donor pancreatic cells.

AAVantgarde gets capital

Country
Italy

AAVantgarde Bio Srl of Italy has raised €61 million in a Series A financing to develop genetic medicines for inherited retinal diseases. The financing was co-led by Atlas Venture of the US and Netherlands-based Forbion.

Mendus receives ATMP certificate

Country
Sweden

Mendus AB of Sweden announced on 19 June that it has received a certificate from the European Medicines Agency confirming that the manufacturing preparations and preclinical data for its lead dendritic cell vaccine candidate meet the current regulatory standard. The EMA’s advanced therapy medicinal product certificates may be issued to cell and gene therapy developers at the start of product development to verify quality controls. They are not required for a future regulatory submission.