The death of a 6-year-old girl after receiving an experimental brain-directed gene-editing therapy at the Xinhua Hospital in Shanghai has reopened debate over how regulators should oversee first-in-human personalized gene therapies.
Tuojie Biotech (Shanghai) Co. Ltd. has prepared and tested NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors found to be potentially useful for the treatment of amyotrophic lateral sclerosis, Alzheimer’s, Parkinson’s disease, diabetic neuropathy, glaucoma, viral infections, chemotherapy-induced peripheral neuropathy and stroke, among others.
Mira Pharmaceuticals Inc. has reported continued progress toward IND-enabling studies of its two lead preclinical programs, MIRA-55, an investigational oral nonopioid drug candidate for chronic inflammatory pain, and SKNY-1, an investigational oral drug candidate for obesity and addiction-related disorders.
TSC Alliance and Apertura Gene Therapy LLC have announced progress in their collaboration to advance gene therapies to treat tuberous sclerosis complex (TSC) with the completion of a preclinical pilot study evaluating an AAV gene therapy approach for TSC1.
Adjuvia Therapeutics Inc. has closed a $8 million series seed financing round to advance its work developing therapeutics to treat mitochondrial dysfunction and disease.
Shanghai Jingxin Biopharmaceutical Co. Ltd. and Zhejiang Jingxin Pharmaceutical Co. Ltd. have synthesized new muscarinic acetylcholine receptor and sigma receptor agonists potentially useful for the treatment of schizophrenia, depression, Parkinson’s and Alzheimer’s disease.
Researchers from Aglaeapharma Inc. and Alpherabio LLC reported the discovery and preclinical characterization of CC-10137, a nonopioid α2A-adrenergic receptor (α2AAR) agonist designed for peripheral activity.
Shape Therapeutics Inc. has been awarded a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance SHP-201, the company’s lead gene therapy candidate.
New drug approvals, financings and deals were seen across the Asia-Pacific biotech sector this week, with China’s National Medical Products Administration (NMPA) granting clearances to Takeda Pharmaceutical Co. Ltd.’s oral orexin receptor 2 agonist oveporexton for narcolepsy, as well as Haisco Pharmaceutical Group’s ciprocopan (HSK-39297) for paroxysmal nocturnal hemoglobinuria.
Otsuka Pharmaceutical Co. Ltd. will likely look next to expand the label of Simtriyo (centanafadine) into patients with comorbid anxiety, having won the U.S. FDA’s green light as a once-daily extended-release capsule for attention deficit hyperactivity disorder (ADHD) in adults plus pediatrics age 6 years and older who weigh at least 20 kg (about 44 lbs.).
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