AstraZeneca Plc is to work with gene editing technologies developed by Cellectis SA of France to identify up to 10 candidate cell and gene therapies for cancer, and autoimmune and rare diseases. Founded in 1999, Cellectis has technology for creating allogeneic chimeric antigen receptor (CAR) T cells by gene editing cells from a healthy donor. The editing technology disables genes that cause donor cells to attack the host.
Sarepta Therapeutics Inc disclosed top-line results on 30 October from a Phase 3 confirmatory study of its gene therapy for Duchenne muscular dystrophy (DMD) which showed statistical significance for key secondary endpoints, but failed to meet the primary endpoint on a measure of motor function. The drug, Elevidys (delandistrogene moxeparvovec), was given an accelerated approval on 22 June 2023 based on a surrogate endpoint. The newest study was intended to confirm this clinical benefit.
Bimzelx (bimekizumab), a monoclonal antibody that inhibits two cytokines driving inflammation, has been approved in the US for the treatment of moderate to severe plaque psoriasis. The Food and Drug Administration cleared the drug for use on 18 October, more than a year after issuing a complete response letter to the developer, UCB SA of Belgium. The CRL was related to certain “pre-approval inspection observations,” according to the company.
Aiolos Bio Inc launched as a new company on 24 October with an asset for the treatment of asthma and $245 million in Series A financing from a syndicate co-led by Forbion, Atlas Venture, Bain Capital Life Sciences, and Sofinnova Investments. The launch was announced just two months after Aiolos acquired rights to a monoclonal antibody that inhibits thymic stromal lymphopoietin (TSLP) from a company in China. TSLP is a cytokine and regulator of the immune response to allergens, viruses and pollutants.
Novo Nordisk A/S has expanded its core franchise in diabetes with the acquisition of a candidate drug for uncontrolled hypertension with potential application for cardiovascular and kidney diseases. The developer is KBP Biosciences PTE of Singapore which is expected to receive up to $1.3 billion for the drug.
Forbion, the Netherlands-based venture group, has launched a new company, Tessellate BIO BV, to develop treatments for cancers that exploit deficiencies in two or more genes leading to cell death. Known as synthetic lethality, the approach has particular relevance for precision medicine. Tessellate was founded by BioGeneration Ventures, a joint venture with Forbion that focuses on company creation and early-stage finance.
UK-based Antiverse Ltd is to extend a collaboration with GlobalBio Inc, a US antibody engineering company, to identify new checkpoint inhibitors for cancer. This follows the successful generation of two candidate anti-PD-1 antibodies that are now entering preclinical development. Financial terms of the agreement were not disclosed.
The collaboration combines Antiverse’s discovery technology, which is based on artificial intelligence, with GlobalBio’s semisynthetic libraries for the discovery and optimisation of antibody-based therapeutics.
The Roche Group is to pay $7.1 billion upfront to acquire Telavant Holdings Inc, developer of a late-stage antibody therapeutic for inflammatory bowel disease (IBD). The deal also includes a near-term milestone payment of $150 million.
Telavant’s lead product, RVT-3101, is a monoclonal antibody that inhibits TL1A, a member of the tumour necrosis family which is expressed in immune cells. It is abnormally expressed in autoimmune diseases, including IBD.
Agomab Therapeutics NV, a Belgian biotech founded in 2017 to explore the potential of a critical biological pathway to achieve tissue repair, closed a $100 million Series C financing round on 11 October to support clinical development of a drug for fibrostenosing Crohn’s disease. The drug, AGMB-129, is a small molecule inhibitor of ALK5 (also known as TGF beta receptor 1), a regulator of fibrosis. Fidelity Management & Research Company led the round, with participation from new investors EQT Life Sciences, Canaan, Dawn Biopharma and existing investors.
Professors Katalin Karikó and Drew Weissman have been awarded this year’s Nobel Prize in Physiology or Medicine for their work on nucleoside base modifications that enabled the development of vaccines against Covid-19. Their findings have fundamentally changed our understanding of how mRNA interacts with our immune system according to the Nobel Assembly, which awards the prize.