Regenerative Medicine

The UK Medical Research Council, a public body, and LifeArc, an independent charity, are making £16 million available to establish a network of centres that will offer clinical grade viral vectors and translational and regulatory guidance to support academic-led patient trials of new gene therapies.

Switzerland-based Santhera Pharmaceuticals Holding AG is to work with Peter Yurchenco of Rutgers University, US on a novel gene therapy approach for treating a congenital muscular dystrophy caused by mutations in the LAMA2 gene. Santhera has entered into two agreements, the first with the university and the second with Prof Yurchenco, to research gene replacement as a therapy for LAMA2-related muscular dystrophy.

Belgium-based Bone Therapeutics SA has extended its financial runway into the second quarter of 2021 after negotiating equity, bond and loan deals that yielded €15 million in new capital. The proceeds will be used to advance two lead assets for bone health into late-stage development.

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.

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.