Regenerative Medicine

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.

Syncona Ltd, an evergreen investment group, has confirmed its target for growth over the next 10 years while reporting a decline in the value of its net assets for the 12 months ended 31 March. The UK-based group ended the year with net assets of £1,254.7 million, compared with £1,309.8 million the previous year. Gains were reported from currency movements and from Beacon Therapeutics, a recently launched gene therapy company targeting retinal diseases. However these were offset by a decline in the value of the company’s listed life science holdings.

Syncona Ltd, the UK investment group, has launched its third gene therapy company directed against retinal diseases, including an inherited monogenic disorder that leads to progressive vision loss in males. The launch of Beacon Therapeutics Holdings Ltd was announced on 12 June and follows the UK group’s acquisition and restructuring of Applied Genetic Technologies Corp (AGTC) of the US. Beacon’s assets include a late-stage programme from AGTC in X-linked retinitis pigmentosa, the inherited monogenic disorder, and two proprietary pre-clinical assets identified by Syncona.

Quell Therapeutics Ltd has secured its first major partnership for the development of its regulatory T cell technology for the treatment of autoimmune diseases. An agreement with AstraZeneca Plc, announced on 9 June, will bring the two parties together to research and develop potential therapies for Type 1 diabetes and inflammatory bowel disease. AstraZeneca is to make an $85 million upfront payment to Quell comprising cash and equity. If the collaboration is successful, with the commercialistion of new products, milestone payments could exceed $2 billion.

DiogenX SAS of France has raised €27.5 million in Series A finance from a venture capital syndicate led by the Boehringer Ingelheim Venture Fund and Roche Venture Fund to advance a candidate drug for type 1 diabetes into clinical development. The drug is a recombinant protein that modulates the Wnt/beta-catenin signalling pathway, a regulator of key cellular functions. In preclinical studies it has shown an ability to regenerate insulin-producing beta cells, according to Johannes Zanzinger, investment director at the Boehringer Ingelheim fund.

Novo Nordisk A/S is to collaborate with the Canadian biotech company Aspect Biosystems Ltd to use bioprinting technology to advance its work in cell therapies for diabetes and obesity. Novo is a leading producer of insulins and more recently glucagon-like peptide 1 medicines for the treatment of diabetes. It has also been building a presence in regenerative medicine.

The economic environment may be difficult, but there’s still plenty of money looking for good science, according to a panel of venture capitalists offering advice to start-ups at the Terrapinn Advanced Therapies Congress in London on 14 and 15 March. The conference came shortly after the collapse of Silicon Valley Bank, and all three VCs called on standard banks to become more start-up friendly, and advised start-ups to diversify their funds across a number of banks, wherever possible.

uniQure NV started 2023 with regulatory approvals in both the US and the EU for its gene therapy for haemophilia B as well as a pipeline of seven clinical and pre-clinical gene therapy projects – all in the field of neurology. Hemgenix (etranacogene dezaparvovec) for haemophilia B treats a genetic bleeding disorder and is the first gene therapy for this disease. Until its approval, the standards for haemophilia care were factor replacement therapies as well as a bispecific antibody treatment from Roche specifically developed for haemophilia A.

Adaptimmune Therapeutics Plc has initiated a merger with a US cell therapy company in order to expand its pipeline and develop therapies for solid tumours. The UK-founded company, which has offices in Oxford and Philadelphia, US, is to combine with TCR2 Therapeutics Inc in an all-share transaction valued at about $103 million. The combined company will be 75% controlled by Adaptimmune shareholders, and 25% by those from TCR2. The company will list on the Nasdaq market under Adaptimmune’s stock symbol and be led by Adrian Rawcliffe, the current Adaptimmune CEO.

The Food and Drug Administration is to allow Intellia Therapeutics Inc to include the US in its global trial of a genome editing therapy for hereditary angioedema (HAE), a rare protein deficiency that can cause swelling in multiple parts of the body. To date, the Phase 1/2 study has been taking place at sites in the Netherlands, New Zealand and the UK. The addition of sites in the US will increase the geographical reach of the programme, as well as the number of patients receiving the experimental therapy. Intellia is based in Cambridge, US.