Regenerative Medicine

The US Food and Drug Administration has placed a clinical hold on uniQure NV’s gene therapy programme in haemophilia B following a possibly related serious adverse event, the company announced on 21 December. The hold affects the company’s pivotal Phase 3 study called HOPE-B which recently reported positive initial data at the annual meeting of the American Society of Hematology (ASH).

Syncona Ltd has provided £26.6 million in Series A funding to Resolution Therapeutics Ltd, a company it founded in August 2020 to exploit the wound healing capabilities of macrophages. Macrophages are cells of the innate immune system involved in the inflammatory response to injury or infection as well as in orchestrating the wound healing process that follows.

The Roche Group is to in-license viral vector technology from the German biotech company CEVEC Pharmaceuticals GmbH in order to increase its manufacturing capacity for gene therapies. This will specifically enable the company’s subsidiary Spark Therapeutics to scale up production of its marketed product Luxturna for retinal diseases which is delivered by an adeno-associated virus (AAV). Luxturna was approved in the US for a rare form of inherited vision loss in 2017. An EU approval followed in 2018.

Bayer AG is to pay up to $4 billion to acquire Asklepios BioPharmaceutical Inc (AskBio), a US gene therapy company with a clinical-stage pipeline and a significant manufacturing capacity. It will pay $2 billion upfront for the company and up to $2 billion in success-based milestones, of which three quarters are expected to be due over the next five years.

The gene and cell therapy industry has significant potential. But this will only be realised if manufacturers share information about their technologies, enabling the advanced therapies to be produced at scale and marketed at affordable prices.

Freeline Therapeutics became a public company on 7 August with the issue of 8.8 million American Depositary Shares on the US Nasdaq market raising gross proceeds of $158.8 million for the development of its portfolio of gene therapy products. The lead product, FLT180a, is in a Phase 1/2 study for patients with haemophilia B, a blood clotting disorder caused by a missing or defective Factor IX clotting protein.

T-knife GmbH, a new company with technology for humanising T cell receptors derived from transgenic mice, has raised €66 million in a Series A financing round in order to develop therapies to treat solid tumours. Current gene based cell therapies for cancer have been highly successful in treating haematological malignancies. However thus far they have been unable to overcome multiple barriers for the treatment of solid tumours.

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.