Regeneron Pharmaceuticals Inc. and Tessera Therapeutics Inc. have established a global collaboration to develop and commercialize TSRA-196, Tessera’s lead investigational in vivo gene editing program for the treatment of α-1 antitrypsin deficiency (AATD).
Ascletis Pharma Inc. has selected ASC-37 oral tablets as a clinical development candidate for the treatment of obesity. The company expects to submit an IND application to the FDA in the second quarter of next year.
Kedrion SpA’s investigational plasma-derived treatment for congenital aceruloplasminemia has been awarded European orphan drug designation by the EMA. The company is working to advance this treatment toward clinical development in Europe.
In an effort to identify stronger and safer α-glucosidase inhibitors, researchers at the Chinese Academy of Sciences prepared a series of diarylpentane derivatives, the most promising of which, [I], reversibly inhibited α-glucosidase with an IC50 of 18 µM.
CSPC Pharmaceutical Group Ltd. has gained clinical trial clearance from China’s National Medical Products Administration (NMPA) for JMT-206 for weight management in individuals with obesity or overweight and at least one weight-related comorbidity.
Glucocorticoid replacement therapy is the current standard of care for congenital adrenal hyperplasia (CAH). However, new therapeutic strategies that can better recapitulate physiological requirements and reduce morbidity and mortality among CAH patients are urgently needed. Despite the promise of gene therapy for correcting monogenic disorders, the strategies investigated to date have not yielded satisfactory results.
A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided.
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
Could GLP-1 receptor agonists (GLP-1RAs), already used in obesity and diabetes, be repurposed as drugs to slow aging? Hong Kong, one of the places in the world with the highest human longevity, is also home to a scientific study on the effects of GLP-1RAs. For the first time, scientists at the Chinese University of Hong Kong (CUHK) have assessed their pharmacological potential in later life using a multiomics preclinical approach.
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