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.
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 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 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.