Regenerative Medicine

Genentech Inc and its Swiss parent, Roche, have ended a cell therapy partnership with Adaptimmune Therapeutics Plc which was focused on developing allogeneic T cell therapies for cancer. The reason for the termination, announced on 10 April, wasn’t given. However it comes at a time when other large companies are making staff redundant or divesting early-stage biotech projects. On 11 April, Syncona, the investment group, announced plans to sell a cell therapy portfolio company to Century Therapeutics Inc. This was to prioritise capital allocation on later-stage assets.

An experimental gene therapy for the rare neurological disorder, Canavan disease, has been given a regenerative medicine advanced therapy (RMAT) designation by the US Food and Drug Administration. RMAT designation is a regulatory incentive awarded by the agency to cell or gene therapy products which show promise for treating or reversing the course of a serious disease.

Obsidian Therapeutics Inc of Cambridge, US, has raised $160.5 million in an oversubscribed Series C financing round to advance a tumour-infiltrating lymphocyte (TIL) therapy in the clinic. TILs are treatments where tumour-infiltrating lymphocytes are removed from a patient’s tumour, grown in large numbers in a laboratory, and returned to the patient to help the immune system fight cancer. TILS are polyclonal cells with diverse receptors. Their advantage is said to be their ability to detect a wide range of tumour-associated antigens.

Genprex Inc, a US-based gene therapy company, has raised $6.5 million from a direct share offering to support its gene therapy programmes for cancer and diabetes. Announced on 22 March, the funding will support development of the company’s nanoparticle technology for delivering functional genes to the body, as well as a new AAV vector approach.

Capstan Therapeutics Inc, a US biotech company with proprietary lipid nanoparticle technology, closed an oversubscribed Series B financing round on 20 March to advance an in vivo cell therapy product for autoimmune disorders. The round was led by RA Capital Management with participation from five new investors including Forbion, Johnson & Johnson Innovation, and Sofinnova Investments. The proceeds will be used to advance CPTX2309, an in vivo engineered chimeric antigen receptor (CAR) T cell therapy and to further develop the company’s pipeline.

The US Food and Drug Administration approved a new therapy for a rare genetic disease on 18 March, indicating a widening of the scope of regulatory authorisations where there is a serious unmet medical need. The product, Lenmeldy (atidarsagene autotemcel), is a gene therapy intended as a one-time treatment for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). MLD is a rare genetic disease that affects the brain and nervous system.

Syncona Ltd, the evergreen investment group, announced on 8 February that its portfolio company Autolus Therapeutics Plc is to collaborate with BioNTech SE to develop new therapies in oncology. The project will aim to advance both companies’ CAR T cell programmes towards commercialisation. These include BioNTech’s cell therapy for solid tumours, BNT211, and Autolus’ two CAR T cell therapy programmes, AUTO1/22, and AUTO6NG, for oncology indications. Under the terms of the agreement, BioNTech has agreed to purchase $200 million of Autolus’ American Depositary Shares in a private placement.

A cell therapy for melanoma, the first of its kind, was given an accelerated approval by the US Food and Drug Administration on 16 February for patients whose disease has progressed despite earlier treatment with a checkpoint inhibitor or a personalised cancer therapy. The therapy, Amtagvi (lifileucel), is a tumour infiltrating lymphocyte cell therapy (TIL) which consists of T cells that have been derived from a patient’s own tumour. 

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.