Regenerative Medicine

BioMarin Pharmaceutical Inc has taken steps to extend its footprint in gene therapy with a preclinical collaboration with Dinaqor AG of Switzerland to develop therapies for rare genetic cardiomyopathies. Cardiomyopathies are diseases of the heart muscle, a significant proportion of which are inherited. Dinaqor will received an undisclosed upfront payment from BioMarin and is eligible for development, regulatory and commercial milestones. BioMarin is also investing in the Swiss company.

US-based SwanBio Therapeutics Inc has raised an additional $52 million in an expanded Series A financing to advance a candidate gene therapy for the inherited neurological disorder adrenomyeloneuropathy (AMN). The new financing brings the total raised in the round to $77 million. It will enable to company to conduct studies leading up to a first clinical trial as well as build up its manufacturing capacity.

Sangamo Therapeutics Inc is to exclusively license cellular conversion technology from UK-based Mogrify Ltd in order to develop allogeneic cell therapies for the treatment of inflammatory and autoimmune diseases. The two companies announced the agreement on 21 April, but did not disclose the size of the upfront and milestone payments.

Evotec SE has set up a gene therapy research and development site in Austria, broadening its drug discovery capabilities to include advanced therapies as well as small molecules and biologics. The company announced the new site on 6 April, while simultaneously disclosing a contract with Takeda Pharmaceutical Co Ltd to generate gene therapies for the Japanese company’s four therapeutic areas: oncology, rare diseases, neuroscience and gastroenterology.

The Zolgensma gene therapy for spinal muscular atrophy, which was approved by the US Food and Drug Administration in May 2019, has now received a positive review by the European Medicines Agency. Pending approval by the European Commission, it is expected to be available to treat babies and young children across the EU soon. Spinal muscular atrophy is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

Llft BioSciences Ltd of the UK has teamed up with King’s College London to create a new version of its cell therapy for cancer which uses a special type of neutrophil to attack solid tumours. The cell therapy is still in preclinical development, but the company will work with the university to develop a new version using induced pluripotent stem cells (iPSCs). It believes the iPSC version will be easier to manufacture and will deliver significant cost savings to patients. Clinical trials of the new therapy are expected to start in 2022.

Human macrophages that were genetically engineered with chimeric antigen receptors (CARs) have demonstrated an anti-cancer effect in mice, suggesting a new application for cell therapy in solid tumours. Results from preclinical studies of the new molecule were published in Nature Biotechnology on 23 March 2020.

UK-based Oxford Biomedica Plc has secured a new agreement to supply lentiviral vectors for gene therapies, this time with Juno Therapeutics, a member of the Bristol-Myers Squibb group. Announced on 18 March, the non-exclusive licence gives BMS access to the UK company’s lentiviral vector platform and is coupled with a five-year clinical supply agreement.

Positive long-term data have been reported for a cell therapy being tested in patients with retinitis pigmentosa, a group of hereditary diseases of the eye that can lead to a progressive loss of vision and blindness. Results from the ongoing US Phase 1/2a study of the human retinal progenitor cell therapy showed a meaningful clinical effect at all points in time out to 12 months after treatment.

GlaxoSmithKline Plc has expanded further into cell therapy with a partnership aimed at generating drug candidates for solid tumours. The collaboration is with Immatics Biotechnologies GmbH of Germany which has been working for several years with the MD Anderson Cancer Center in Texas, US on engineering T cells to fight cancer.