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SHANGHAI, China — YolTech Therapeutics announced that the first patient has been dosed with YOLT-101, the company’s in vivo genome editing candidate being developed as a single dose, potentially curative therapy for Familial Hypercholesterolemia(FH), marking the commencement of an Investigator-Initiated Trial (IIT). Familial Hypercholesterolemia (FH) is a genetic disorder that...
A young Galway dad has had his life turned upside down after a sneeze led to a horror injury giving him the back of a 90-year-old man. It was in July last year when 36-year-old Adrian Gordon sneezed and felt his back pop, leading to a horrific six months of...
After Kara Miller woke up with blurry vision she couldn’t blink away, her primary care doctor spotted something that would change her life. Kara, then 17, had a lesion on one of her optic nerves. Not only did the damaged nerve explain why she was seeing double, it was telltale...
Corbeil-Essonnes (near Paris), France – Yposkesi, a leading Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapies, today announces the launch of project SKY, the construction of Yposkesi’s second commercial cell and gene therapy facility and new global resource for drug developers of biologics, also called ATMPs...
Cologne, Germany – Zanubrutinib, a second-generation Bruton tyrosine kinase (BTK) inhibitor, is approved for several therapeutic indications. The German Institute for Quality and Efficiency in Health Care (IQWiG) investigated whether the drug has an added benefit compared with the appropriate comparator therapy for adult patients with chronic lymphocytic leukaemia (CLL)...
DUARTE, California – The novel drug, zanzalintinib (XL092), showed promising cancer-fighting abilities in patients with advanced clear-cell renal cell carcinoma (ccRCC), according to initial results from the STELLAR-001 clinical trial. According to trial results, which were presented at the 2023 International Kidney Cancer Symposium, antitumor activity was observed in patients...
Zealand Pharma A/S a biotechnology company focused on the discovery and development of innovative peptide-based medicines, today announced financial results for the 12-month period from January 1 to December 31, 2022 and provided a corporate update.
Copenhagen, Denmark – The FDA rejected Zealand Pharma’s glucagon receptor agonist in congenital hyperinsulinism (CHI) after identifying problems at a third-party manufacturing site. The company said it remains confident that it can resubmit in the first half of 2024. Zealand revealed last week that the regulator issued a complete response...
CELEBRATION, Fla. — Zevra Therapeutics Inc.,  a rare disease therapeutics company, today announced it resubmitted its New Drug Application (NDA) for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC) to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Based on standard...