After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.
Psychedelic drugs continued to make regulatory and clinical headway, as non-believers converted to believers in the category once regarded with skepticism, to say the least.
Zhongzhi Pharmaceutical Holdings Ltd. made a $3 million investment in a series A financing round of stem cell therapy developer Gabaeron Inc. Dec. 21, expected to help propel Gabaeron’s preclinical Alzheimer’s disease (AD) candidate into phase I testing.
Major depressive disorder (MDD) notched another late-stage failure in the form of Neumora Therapeutics Inc.’s data from the phase III Koastal-1 study, the first of three replicant trials in the pivotal program testing kappa opioid receptor antagonist navacaprant.
Among the most profound results presented at the 2024 European Society for Medical Oncology Congress were the 10-year data from the Checkmate-067 and Keynote-006 trials of Opdivo and Keytruda as first-line agents in advanced or metastatic melanoma in which 10-year overall survival topped 40%. The success of checkpoint blockade, however, has not extended to all tumor types, but in 2024, molecular studies have led to advances in gene therapies and a multitude of approaches that have opened the door to hope.
Zhongzhi Pharmaceutical Holdings Ltd. made a $3 million investment in a series A financing round of stem cell therapy developer Gabaeron Inc. Dec. 21, expected to help propel Gabaeron’s preclinical Alzheimer’s disease (AD) candidate into phase I testing.
Among the most profound results presented at the 2024 European Society for Medical Oncology Congress were the 10-year data from the Checkmate-067 and Keynote-006 trials of Opdivo and Keytruda as first-line agents in advanced or metastatic melanoma in which 10-year overall survival topped 40%. The success of checkpoint blockade, however, has not extended to all tumor types, but in 2024, molecular studies have led to advances in gene therapies and a multitude of approaches that have opened the door to hope.
Lario Therapeutics Ltd. has divulged voltage-dependent R-type calcium channel subunit α-1E (Cav2.3) blockers reported to be useful for the treatment of neurodegeneration, neurodevelopmental disorders, epilepsy, Parkinson’s disease, endocrine disorders, cerebral vasospasm, pain and epileptic encephalopathy.
Nico Therapeutics Inc. has described NLRP3 inflammasome inhibitors reported to be useful for the treatment of Alzheimer’s disease and nonalcoholic fatty liver disease (metabolic dysfunction-associated steatotic liver disease [MASLD]).
Deficiencies in the SLC6A1 gene, encoding the γ-aminobutyric acid transporter GAT-1, are associated with infantile encephalopathy with intellectual disability. Like other neurodevelopmental disorders, SLC6A1-related disorders lack effective treatments; therefore, gene therapy using viral vectors has been proposed as a potential therapeutic strategy.
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