The word “niche” implies a specialized environment. But to Fiona Doetsch, the stem cell niche is anything but. For brain stem cells, “the whole organism is the niche,” Doetsch told the audience at the third plenary session of the International Society for Stem Cell Research (ISSCR) annual meeting in Hamburg this week. It’s a surprising idea at first, given the brain’s protection from many circulating substances via a series of barriers, including the blood-brain barrier and the blood-cerebrospinal fluid barrier.
The primary-endpoint win by Sensorion SA in a phase II proof-of-concept study with SENS-401 (arazasetron) in hearing loss made public March 11 brought renewed interest in the space, where a number of players are advancing gene therapies. The story marches on, with Montpellier, France-based Sensorion due to discuss the product July 13 at the International Conference on Cochlear Implants and Other Implantable Technologies in Vancouver, British Columbia.
Nonalcoholic steatohepatitis was renamed, for the first time in 34 years, to metabolic dysfunction associated steatohepatitis (MASH), but a name change is far from being the biggest development in the field, according to experts at Bioplus Interphex (BIX) Korea 2024.
The industry is looking, with renewed hope, to the “promise” of messenger RNA (mRNA) therapeutics for a wide range of diseases beyond COVID-19, and not only in vaccine form but also for gene and cell therapies.
The obesity market is hot with Novo Nordisk A/S generating $6.3 billion in sales from its semaglutide-containing drugs, Ozempic, Saxenda, Rybelsus and Wegovy, in the first quarter of 2024 and Eli Lilly and Co. bringing in $2.3 billion for its tirzepatide-containing drugs, Zepbound and Mounjaro, in the same quarter. It appears unlikely the market will be a two-horse race for much longer though.
Coming off a presentation at the European Alliance of Associations for Rheumatology meeting, where a single report of disease recurrence in a lupus patient overshadowed promising early data for Kyverna Therapeutics Inc.’s autoimmune candidate, KYV-101, the Emeryville, Calif.-based company rallied on news of a case report describing the first use of the CD19-targeting CAR T-cell therapy in a patient with stiff-person syndrome, a rare, progressive neurological autoimmune disorder for which there is limited treatment.
During the basic science morning track on the last day of this year’s Annual Congress of the European Hematology Association (EHA), the attention was focused on oncogenic transcription factors and complexes considered turning points within the acute myeloid leukemia (AML) arena.
For the time being, Madrigal Pharmaceuticals Inc.’s Rezdiffra (resmetirom) stands alone as the only U.S. FDA approved treatment for treating nonalcoholic steatohepatitis (NASH). But new and positive data from multiple companies show Madrigal may soon have company.
Myeloproliferative neoplasms (MPNs) can only be cured, to date, using allogeneic stem cell transplantation which, in turn, only works for up to 20% of patients. As calreticulin (CALR) frameshift mutations are the second most common cause of MPNs, targeting this endoplasmic reticulum resident protein is one of the strategies emerging at the forefront of hematological malignancies research.
Abtis Co. Ltd., Dong-A ST’s newly incorporated subsidiary since December 2023, is making headway with its leading Claudin18.2-targeting antibody-drug conjugate (ADC) candidate, AT-211, according to Abtis CEO Taedong Han. “About 80% of gastric cancers do not have HER2 overexpression, but 77% overexpressed Claudin18.2,” Han told Bio Korea 2024 audience members on May 9, stressing that AT-211 was found to be highly potent against cancer cells expressing Claudin18.2 in ADC cell viability studies.
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