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BioWorld - Wednesday, March 18, 2026
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Ear, Nose & Throat

TMPRSS3 gene therapy prolongedly restores hearing in mouse model of human recessive deafness DFNB8

June 9, 2023
Currently, there are no treatments to reverse or prevent genetic hearing loss, which affects 1 in 500 newborns. Several gene replacement and overexpression preclinical studies targeting genetic hearing loss have shown success, as the inner ear can be accessed safely by local injection. However, all these gene therapy studies have been performed in neonatal animals, except one in the Otof gene; therefore, the suitability of the approach in the fully mature adult inner ear remains to be elucidated.
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Immuno-oncology

Lantern Pharma collaborates with Bielefeld University to develop cryptophycin-ADCs

June 9, 2023
Lantern Pharma Inc. has entered into a research collaboration with Bielefeld University to develop antibody-drug conjugates (ADCs) with therapeutic and antitumor potential. Using Lantern’s proprietary artificial intelligence (AI) platform, RADR, the collaboration will leverage insights from the recently developed RADR AI ADC module in combination with research from Bielefeld.
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Multiple sclerosis
Neurology/Psychiatric

Pipeline Therapeutics cleared to advance LPA1 antagonist for multiple sclerosis

June 9, 2023
Pipeline Therapeutics Inc. has received FDA clearance to initiate a phase I trial of PIPE-791 in healthy volunteers, with dosing expected to begin in the second half of this year. PIPE-791 is advancing toward clinical development for remyelination and neuroinflammation, with a leading indication of multiple sclerosis (MS).
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Cancer

MAP4K1 inhibitors detailed in Wigen Biomedicine Technology patent

June 9, 2023
Wigen Biomedicine Technology (Shanghai) Co. Ltd. researchers have identified mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1) inhibitors reported to be useful for the treatment of cancer.
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Diagnostics

AC Immune creates agents for imaging of α-synuclein aggregates

June 9, 2023
AC Immune SA has patented 4h-imidazo[1,5-b]pyrazole derivatives and labeled compounds targeting α-synuclein (SNCA). They are reported to be useful for diagnosis of multiple system atrophy, Parkinson’s dementia, Lewy body dementia, Alzheimer’s and Parkinson’s disease, among others.
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Endocrine/Metabolic

Cerevance prepares and tests new CD38 inhibitors

June 9, 2023
Cerevance Inc. has described N-(4-aminocyclohexyl)pyrimidine-4-carboxamide derivatives acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors. As such, they are reported to be useful for the treatment of aging, diabetic nephropathy, glaucoma, inflammatory disorders, metabolic syndrome, obesity and rheumatoid arthritis.
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Cancer

University of California Oakland and 1200 Pharma jointly develop GTPase KRAS inhibitors

June 9, 2023
Research at the University of California, Oakland and 1200 Pharma LLC has led to the identification of GTPase KRAS (G12C mutant) inhibitors reported to be useful for the treatment of cancer.
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Cancer

Allianthera (Suzhou) Biopharmaceutical patents new aryl hydrocarbon receptor agonists

June 9, 2023
Allianthera (Suzhou) Biopharmaceutical Co. Ltd. has patented new aryl hydrocarbon receptor (AhR) agonists reported to be useful for the treatment of cancer, lung diseases, bone, dermatological, eye, gastrointestinal, neurological and pancreatic disorders, among others.
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Infection

Nonmucosal intradermal dmLT-M. tuberculosis Ag85 complex combination vaccine confers enhanced mucosal immunity

June 9, 2023
Tuberculosis (TB) is the 13th leading cause of death in the world, and it is rising together with the increased prevalence of drug-resistant TB in many countries. The Bacille Calmette-Guerin (BCG) vaccine is the only available TB vaccine, and it has been given to more people than any other vaccine. While the BCG vaccine has saved tens of millions of lives, it confers suboptimal protection against pulmonary TB as it is limited to providing protection only until early childhood. Significantly, the BCG vaccine is administered intradermally to confer exceptional mucosal immunity as compared to most other vaccines, which are more commonly administered intramuscularly. Novel strategies to improve the duration of TB mucosal immunity are urgently needed.
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Concept of business partnership
Drug Design, Drug Delivery & Technologies

Iktos and Curreio establish AI-based collaboration to drive new drug design

June 9, 2023
Iktos SA and Curreio Inc. have established a collaboration agreement using artificial intelligence (AI) for new drug design. Under this collaboration, Iktos will leverage its de novo generative design technology in combination with Curreio’s cryo-electron microscopy (EM) platform to facilitate the rapid design of novel preclinical drug candidates for an undisclosed target.
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