Alethio Therapeutics (formerly known as Alethiomics Ltd.) has emerged from stealth with two antibody-drug conjugate (ADC) programs for the treatment of myeloproliferative neoplasms (MPNs).
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.
Sickle cell disease (SCD) is a genetic disorder caused by a single point mutation in the β-globin gene, leading to the production of abnormal hemoglobin. Patients with SCD lacking a compatible donor for allogeneic hematopoietic stem/progenitor cell transplantation can benefit from gene therapy approaches.
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.
Entasis Therapeutics Holdings Inc. has described mannan-binding lectin serine protease 2 (MASP2) and/or mannan-binding lectin serine protease 3 (MASP3) inhibitors reported to be useful for the treatment of thrombotic microangiopathy, ischemia-reperfusion injury, disseminated intravascular coagulation, graft-versus-host disease, diffuse alveolar hemorrhage syndrome, and renal, eye and cardiovascular disorders, among others.
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.
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