Asklepios BioPharmaceutical Inc, a wholly-owned subsidiary of Bayer AG, has entered into a multi-year research collaboration and option agreement with ReCode Therapeutics Inc. Under the agreement, the companies will work together to discover genetic medicines that precisely target a disease. This will be done by developing technology for the use of single vectors as delivery vehicles for gene editing and DNA cargoes.
The developers of the first chimeric antigen receptor (CAR) T cell therapies are to merge their assets under a deal that they say will speed up manufacturing and create new opportunities for treating solid tumours. The deal involves the acquisition of Tmunity Therapeutics Inc of Philadelphia, US, by Kite Pharma, a business unit of Gilead Sciences Inc. The financial terms of the transaction were not disclosed.
AstraZeneca Plc is to acquire a Dutch company with technology for developing T cell therapies for cancer which target antigens specific to a patient’s tumour. The company, Neogene Therapeutics Inc, was founded in Amsterdam, the Netherlands in 2018 and has a pipeline of candidate therapies that consist of engineered T cells equipped with receptors targeting tumour-specific antigens, also known as neoantigens.
Researchers from nine academic centres, together with PACT Pharma Inc of the US, have reported data from a Phase 1 study of a novel cell therapy which produced initial signs of clinical activity in patients with a variety of solid tumours. The treatment is an allogeneic T cell receptor (TCR) T cell therapy which has been edited to recognise the patient’s specific cancer. This was done by equipping the TCR with a neoantigen specific to the cancer.
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.