A technology that combines transcriptomic data and AI enables a novel approach to drug discovery based on the state of cells, how they behave and which genes they express. The Drugreflector model, developed by scientists at Cellarity Inc., learns from gene expression profiles and predicts which compounds could induce beneficial changes in that cellular state to develop a treatment.

The U.S. FDA named the first nine recipients of the recently unveiled commissioner’s national priority voucher (CNPV) program aimed at addressing unmet public health needs by shortening regulatory review times to as little as one to two months. For one of those firms, Disc Medicine Inc., which submitted an NDA for bitopertin for rare genetic disorder erythropoietic protoporphyria in September, that could mean a potential approval before the end of 2025.

Paroxysmal nocturnal hemoglobinuria (PNH) – the rare blood disorder characterized by premature destruction of red blood cells – found itself in the spotlight, as did shares of Omeros Corp. (NASDAQ:OMER), which closed Oct. 15 at $10.42, up $6.32, or 154%, on word of an asset purchase and license agreement with Novo Nordisk A/S for zaltenibart (formerly OMS-906).

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.

As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.

Word Sept. 4 from Agios Pharmaceuticals Inc. that the U.S. FDA extended the PDUFA date for the sNDA related to Pyrukynd (mitapivat), after the Cambridge, Mass.-based firm submitted a proposed risk evaluation and mitigation strategy (REMS), drew Wall Street’s attention to the regulatory approach.

CSL Ltd. inked a potential $2.1 billion deal with Dutch biotech company Varmx BV to develop VMX-C001 as a new treatment to restore blood coagulation in patients taking a factor Xa inhibitor.

Enlaza Therapeutics Inc. will take charge of research through nominating candidates in its potentially $2 billion-plus deal with Vertex Pharmaceuticals Inc. For its efforts, Enlaza is getting $45 million in an up-front payment and equity investment, plus the opportunity to bring in more than $2 billion in research, development, regulatory and commercial milestones and tiered royalties on net sales.

CSL Ltd.’s stock tumbled 16.88% on the news that it plans to cut 3,000 jobs and to hive off its Seqirus vaccine unit in a cost-cutting measure to save more than $500 million per year over the next three years.