“Our goal is that by the end of this call, you’ll share our conviction in the totality of the data.” So said Agios Pharmaceuticals Inc. CEO Brian Goff as the company reported top-line data from its 52-week Rise Up trial testing mitapivat in sickle cell disease showing the oral pyruvate kinase activator hit statistical significance on one of two primary endpoints and two of three key secondary endpoints.

Fifteen years since the first patient was treated, and after being ditched by two companies, the EMA is recommending approval of Waskyra (etuvetidigene autotemcel), the first gene therapy for treating Wiskott-Aldrich syndrome.

Just as investors were looking ahead to news by year-end on Prelude Therapeutics Inc.’s SMARCA2-targeted degraders, the firm said work in the space will be halted, with efforts shifting toward the mutant selective JAK2V617F JH2 inhibitor program by way of a new deal with Incyte Corp.

Biomarin Pharmaceutical Inc. plans to divest its pioneering gene therapy for hemophilia, Roctavian (valoctocogene roxaparvovec), and remove what had been considered a potential blockbuster from the portfolio in order to grow the company.

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.