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BioWorld - Wednesday, December 24, 2025
Home » Newsletters » BioWorld Science

BioWorld Science

Dec. 11, 2024

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Illustration of a T cell

Engineered T cells can serve as organ-specific delivery service

Researchers from the University of California San Francisco (UCSF) have successfully replicated the design of regulatory T cells, achieving local targeted immune suppression and protection from CAR T-cell cytotoxicity. Many of the treatments used so far in the context of inflammatory and autoimmune disorders lead to systemic immunosuppression. In this sense, limiting immunosuppression locally to targeted tissues may help overcome systemic toxicity. Read More
Concept of business partnership

Relation inks two GSK deals tackling osteoarthritis, fibrotic disease

Relation Therapeutics Ltd. has delivered the first commercial validation of its combined wet lab/in silico platform for homing in on disease-related genes in the thick of the genome, signing two collaboration agreements with GSK plc, with a potential value of $200 million per target. Read More
Silhouette of head, brain

Preclinical efficacy of M1/M4 receptor agonist in schizophrenia, cognitive impairment models

Researchers from Maplight Therapeutics Inc. presented preclinical data for the investigational muscarinic M1/M4 receptor agonist ML-007, being evaluated for the treatment of neuropsychiatric disorders, such as schizophrenia and Alzheimer’s disease (AD). The activity of ML-007 was compared to that of another muscarinic M1/M4 agonist, xanomeline. Read More
R&D money

Financing at Orthogon Therapeutics supports development of oral therapy for BK virus

Orthogon Therapeutics LLC has closed an oversubscribed financing round, with funding to be used to accelerate the development of its first-in-class treatments for managing BK virus reactivation in transplant patients. Read More

RMC-7977 targets RAS/MAPK pathway and sensitizes AML cells

The effective targeting of RAS-mutant acute myeloid leukemia (AML) still remains a challenge; RAS mutations are tied to relapse to targeted therapy, such as resistance to FLT3 inhibitors due to the RAS/MAPK pathway, for example. Read More
Antibody-drug conjugate illustration

FDA clears IND for B7-H4-targeting ADC

Nextcure Inc. has reported IND clearance from the FDA enabling initiation of a phase I trial of LNCB-74, a B7-H4-targeting antibody-drug conjugate (ADC) being developed for various cancers. Read More

Haihe Biopharma patents new PARP-1 inhibitors

Haihe Biopharma Co. Ltd. has disclosed poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors reported to be useful for the treatment of cancer. Read More

Discovery of FBPase inhibitor with improved activity and good druggability

Work at the Institute of Materia Medica of Beijing has led to the discovery and preclinical characterization of novel fructose-1,6-bisphosphatase (FBPase) inhibitors as potential therapeutic candidates for the treatment of type 2 diabetes. Read More

Pilatus Biosciences’ PLT-012 awarded orphan drug designation

Pilatus Biosciences Inc.’s PLT-012 has been awarded orphan drug designation by the FDA for treating liver and intrahepatic bile duct cancer. Read More
brain neurological

CAP-002 exerts robust and sustained phenotypic correction in genetic epilepsy

Epileptic encephalopathy due to mutations in the STXBP1 gene, also known as genetic epilepsy, is a rare disease characterized by intellectual disability, speech and motor impairment and behavioral issues, among others, that affects 1 in every 30,000 newborns and which has no approved therapies to date. Read More

Korean researchers divulge new phenaleno-isoquinolinium-based compounds for cancer

Scientists at Daegu-Gyeongbuk Medical Innovation Foundation and Korea Research Institute of Chemical Technology have synthesized phenaleno-isoquinolinium-based compounds reported to be useful for the treatment of cancer. Read More

Researchers in the Netherlands describe new PET and SPECT imaging agents

Researchers at Stichting AMC Foundation and Vrije Universiteit van Amsterdam have identified [18F]-labeled pyrazolidine-3,5-dione compounds targeting P2Y purinoceptor 12 (P2RY12; P2Y12) acting as PET and SPECT imaging agents. They are reported to be useful for the diagnosis of multiple sclerosis, Alzheimer’s disease and Parkinson’s disease. Read More
3D illustration of the cross-section of skin layers with atopic dermatitis

Astria’s OX40 antagonist gains US IND clearance for atopic dermatitis

Astria Therapeutics Inc. has gained IND clearance from the FDA for STAR-0310, a monoclonal antibody OX40 antagonist being developed for atopic dermatitis. Read More

New GTPase KRAS mutant inhibitors disclosed in Mirati patent

Mirati Therapeutics Inc. has divulged GTPase KRAS (G12S mutant) and/or GTPase KRAS (G12C mutant) inhibitors reported to be useful for the treatment of cancer. Read More

Roche reports preclinical data supporting NXT-007 trials for hemophilia A

At the recently concluded American Society of Hematology meeting, F Hoffmann-La Roche Ltd. presented promising results for their next-generation coagulation factor VIIIa (FVIIIa) mimetic bispecific antibody, NXT-007 (RO-7589655), supporting the ongoing phase I/II studies. Read More
Art concept for RNA and protein

Alltrna’s engineered tRNA restores protein production in MMA and PKU models

Alltrna Inc. has presented new preclinical data on the company’s first transfer RNA (tRNA) development candidate, AP-003. Read More

Frontier Medicines discovers new TP53 mutant stabilizers

Frontier Medicines Corp. has described cellular tumor antigen p53 (TP53) (mutant) stabilizers reported to be useful for the treatment of cancer. Read More

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