Cell therapy weekly: US and Australian approvals for Dravet syndrome gene therapy candidate
This week: a gene therapy candidate for SCN1A+ Dravet syndrome has received Investigational New Drug clearance from the US FDA and Clinical Trial Approval from the Australia Therapeutic Goods Administration, Novo Holdings (Gentofte, Denmark) and Catalent (Somerset, UK) have entered into a merger agreement and AstraZeneca (Cambridge, UK) has announced a US$300 million investment in a new cell therapy facility.
The news highlights:
- US and Australian approvals for Dravet syndrome gene therapy candidate
- Controlling shareholder of Novo Nordisk to acquire Catalent
- AstraZeneca expands US cell therapy manufacturing capacity
US and Australian approvals for Dravet syndrome gene therapy candidate
Encoded Therapeutics’ (CA, USA) gene therapy candidate for SCN1A+ Dravet syndrome has received Investigational New Drug clearance from the US FDA and Clinical Trial Approval from the Australia Therapeutic Goods Administration. Dravet syndrome is a severe disorder of the central nervous system, with over 85% of cases resulting from loss-of-function mutations in the SCN1A gene. The gene therapy, ETX101, aims to address the underlying cause of the disease by delivering a transgene that encodes an engineered transgene that will selectively upregulate the expression of the SCN1A gene.
Encoded will initiate Phase I/II clinical trials in the US and Australia, both with the primary aim of evaluating the safety and tolerability of ETX101.
“ETX101 represents a groundbreaking advancement in the therapeutic landscape for Dravet syndrome, with potential not only for seizure management but also for addressing the broader spectrum of non-seizure manifestations. [The clinical trials] ENDEAVOR and WAYFINDER are the first step in bringing a potentially one-time, disease-modifying gene therapy to the Dravet community and we are excited to be partnering with leading experts in the care of patients with Dravet syndrome to begin clinical trials in the US and Australia in the coming months,” said Sal Rico, Chief Medical Officer of Encoded.
Controlling shareholder of Novo Nordisk to acquire Catalent
Novo Holdings, a holding and investment company responsible for the assets and wealth of the Novo Nordisk Foundation (Hellerup, Denmark), and Catalent have entered into a merger agreement. In this merger, Novo Holdings will acquire Catalent in an all-cash deal valued atUS$16.5 billion based on enterprise value. Any outstanding shares of Catalent will be acquired by Novo Holdings for US$63.50 per share.
The merger is expected to close by the end of 2024, after which Catalent’s shares will cease trading on the New York Stock Exchange and the company will transition to being privately held.
John Greisch, Executive Chair of the Catalent Board and Chair of the Strategic and Operational Review Committee, said: “This transaction delivers significant, certain and premium value to our stockholders. Novo Holdings believes in our vision and will provide Catalent with a strong foundation as we continue developing, manufacturing and supplying top products.”
AstraZeneca expands US cell therapy manufacturing capacity
AstraZeneca has announced a US$300 million investment in a cutting-edge facility in Rockville, Maryland. The investment aims to launch the company’s cell therapy manufacturing platforms, which will support cancer trials and future commercial supply. AstraZeneca’s cell therapy portfolio includes CAR-T therapies for hepatocellular carcinoma, prostate cancer and other solid tumors at various stages of clinical and preclinical development.
Pam Cheng, Executive Vice President of Global Operations & IT and Chief Sustainability Officer, AstraZeneca, stated: “We are incredibly excited that more than 150 new highly skilled jobs are being created to bring our scientific work and therapies to clinical trials which could transform the lives of patients around the world. This new $300 million investment will accelerate our ambition to make next-generation cell therapy a reality, ensuring that we are ready to scale and meet the demands of patients.”
