News

A new company which is developing RNA editing therapeutics launched on 19 September with $30 million in initial funding led by ARCH Venture Partners, one of the biotech industry’s largest financiers. The company, AIRNA Corp Inc, plans to use the funds to advance a product candidate to treat the inherited genetic disease alpha-1 antitrypsin deficiency. This is a deficiency that raises a person’s risk of lung disease including the risk of contracting chronic obstructive pulmonary disease. It will also investigate therapies for high unmet needs.

A fund created by the Bill & Melinda Gates Foundation to support private enterprise has awarded $5 million to Transimmune AG of Germany to enhance the potency of messenger RNA (mRNA) vaccines for the treatment of infectious diseases. Transimmune, which is a spin-out of Yale University in the US, will initially focus on a therapeutic vaccine for people living with HIV.

A new research collaboration potentially covering multiple therapy types for the treatment of cancer was unveiled by Moderna Inc and Immatics NV on 11 September. The partnership brings together Moderna’s messenger RNA (mRNA) technology with Immatics’ expertise in engineering T cell receptors (TCRs) and developing biologics. The goal is to develop bispecific therapies, vaccines and cell therapies for cancer.

Novo Nordisk A/S is to use artificial intelligence and real-world data to discover new drugs for cardiometabolic diseases under a collaboration with Valo Health Inc, a three-year old US company founded by venture capitalists.

Laboratoires Pierre Fabre SA has acquired a biotech company founded by Versant Ventures as part of a drive to expand its pipeline in oncology. The company, Vertical Bio AG, has a monoclonal antibody poised to enter clinical development for patients with non-small cell lung cancer – specifically those with mutations or amplification of the c-MET protein.

GSK Plc has received US approval for Ojjaara (momelotinib), a Janus kinase inhibitor for the treatment of myelofibrosis patients with anaemia. The authorisation is based on positive results from two Phase 3 trials both of which were designed to show a reduction in manifestations of the disease. Myelofibrosis is a rare blood cancer that can lead to severely low blood counts, including anaemia and thrombocytopaenia.

Yescarta, a chimeric antigen receptor (CAR) T cell therapy, has delivered positive results in a Phase 2 study of patients with relapsed or refractory large B cell lymphoma after one prior therapy. The patients were all ineligible for chemotherapy and autologous stem cell transplantation. Yescarta (axicabtagene ciloleucel) is already approved for several lymphoma indications in the US and Europe.

A Phase 2 trial of a gene therapy for geographic atrophy secondary to dry age-related macular degeneration has been stopped for lack of efficacy, Syncona Ltd, the UK developer, announced on 11 September. This followed a recommendation by the trial’s independent data monitoring committee which concluded that the overall benefit/risk assessment of the therapy didn’t support continuation.

ROME Therapeutics Inc, a US company developing therapies based on the dark genome, has secured $72 million from a Series B round extension bringing the total amount raised up to $149 million. Strategic investors Johnson & Johnson Innovation-JJDC Inc and Bristol Myers Squibb Inc joined the funding syndicate alongside new and existing investors.

In an article published on www.mednous.com on 21 August 2023 entitled SSI Strategy buys NDA Group, the leadership of the new group has been corrected to show that Douglas Locke, Chief Executive Officer of SSI Strategy, has been named CEO of the combined group. A corrected version follows.