Regenerative Medicine

RMAT for gene therapy

Country
United States

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 gets venture funds

Country
United States

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 completes offering

Country
United States

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 raises finance

Country
United States

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.

Treatment for rare disease

Country
United States

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.

BioNTech and Autolus collaborate

Country
United Kingdom

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.

Amtagvi approved by FDA

Country
United States

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. 

FDA comments on CAR T risks

Country
United States

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. 

Fast track for CAR T cell therapy

Country
United States

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.

UK raises funding for Catapult

Country
United Kingdom

The UK government, through its Innovate UK agency, has increased its five-year funding commitment to the Cell and Gene Therapy Catapult, an incubator for researchers and small companies working on novel advanced therapy medicinal products (ATMPs). Announced on 8 January, the new financial award is £80 million which represents a 35% increase from the previous five year period.