Regenerative Medicine

The chimeric antigen receptor (CAR) T cell therapy Kymriah (tisagenlecleucel) has delivered positive data in a Phase 2 study of patients with relapsed or refractory follicular lymphoma, the second most common form of non-Hodgkin lymphoma, Novartis announced on 4 August. The global ELARA trial met its primary endpoint of complete response rate as assessed by an independent review committee. No new safety signals were observed.

ReNeuron Group Plc has reported further positive long-term data for its cell therapy for retinitis pigmentosa (RP), a group of hereditary eye diseases that can lead to the progressive loss of sight and ultimately blindness. Data from a Phase 2a trial continued to show a meaningful clinical effect from the therapy at all intervals in time from the first treatment, and for the time, out to 18 months.

The gene therapy developer Freeline Therapeutics Ltd has closed a $120 million extended Series C financing round in order to bring its lead programme for haemophilia B into a pivotal trial. Simultaneously on 30 June, it announced a possible initial public offering (IPO) of its shares in the US. The timing and the terms of such an offer have not been decided. Freeline’s majority shareholder is Syncona Ltd, formerly part of the Wellcome Trust.

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.