Regenerative Medicine

A new gene therapy company has been launched in the US and UK by a group of academic-entrepreneurs who aim to treat genetic diseases of the eye with therapies delivered by a herpes simplex virus (HSV) vector. The company, Eudora, plans to employ an HSV-1 vector as a delivery vehicle instead of the commonly used adeno-associated virus (AAV) vector. In an announcement on 31 October, Eudora said its HSV vehicle could deliver more drug than an AAV vector, one of the most widely used virus vectors in the industry.

The European Medicines Agency has launched a new project to help academic scientists and those working for non-profit organisations understand the regulatory requirements for developing and eventually commercialising cell, gene and tissue engineered therapies. The project complements the agency’s priority medicines scheme, PRIME, which was launched in 2016 to provide support to developers of medicines that meet a major public health need.

France-based SparingVision SAS has raised €75 million in a Series B financing round to bring its two lead gene therapy assets into clinical development for the treatment of retinitis pigmentosa, an inherited retinal disorder. The adeno-associated virus vector therapies will deliver mutation agnostic treatments to the eye with a view to correcting the disorder.

The first cell-based gene therapy for the treatment of patients with beta thalassaemia, an inherited blood disorder, was approved by the US Food and Drug Administration on 17 August. Zynteglo (betibeglogene autotemcel) is a one-time treatment administered as an ex vivo lentiviral vector gene therapy. A functional beta globin gene is added into a patient’s cells outside of the body and then infused back into the patient. The treatment process is comprised of several steps and will be administered at qualified treatment centres in the US.

Agomab Therapeutics NV has secured an additional $40.5 million in Series B funding in order to progress its pipeline of small molecule and antibody therapeutics for regenerating damaged tissue. The latest funding follows $74 million raised in 2021, bringing the total generated from the round up to $114 million. Pfizer Inc led the latest facility through its investment vehicle Pfizer Breakthrough Growth Initiative.

The European Medicines Agency has recommended the conditional approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for haemophilia A. If approved, it would be the first gene therapy for this disease in Europe. The conditional approval carries with it a requirement that the developer, BioMarin Pharmaceutical Inc, provide follow-up data from ongoing clinical trials to show the therapy continues to be safe and efficacious for patients.

It is often said that cell and gene therapies are a ‘process’ rather than typical drugs. This is based on the fact that their success depends on the quality of their starting materials and how they are manufactured. This could be an autologous chimeric antigen receptor (CAR) T cell-based gene therapy, or any one of the many allogeneic products in clinical development. Manufacturing is the key strategic issue for developers of these therapies, according to speakers at the On Helix annual meeting in Cambridge, UK on 7 July.

Spain-based VIVEbiotech SL has received regulatory approval for an upgrade of its lentiviral vector manufacturing capacity that will significantly expand its ability to produce vectors for both in vivo and ex vivo gene therapies. The company currently has 40 programmes ongoing with four dedicated to in vivo administration.

Galapagos NV is to acquire two companies with a presence in cell therapy as it takes steps to broaden its business under new leadership. The Dutch company has reached separate agreements to purchase CellPoint BV, a manufacturing company in the Netherlands, and AboundBio Inc, a US drug discovery company with an antibody library and antibody engineering capabilities.

Two French companies have joined forces to co-develop manufacturing technologies for AAV-based gene therapies. Coave Therapeutics is to contribute its expertise on viral vectors to the collaboration, while ABL, a contract development and manufacturing organisation, will provide production skills and laboratory space in Lyon.