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BioWorld - Monday, April 20, 2026
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Art concept for RNA and protein
Endocrine/metabolic

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

Dec. 11, 2024
Alltrna Inc. has presented new preclinical data on the company’s first transfer RNA (tRNA) development candidate, AP-003.
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Brain waves
Neurology/psychiatric

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

Dec. 11, 2024
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.
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Hematologic

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

Dec. 11, 2024
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.
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3D illustration of the cross-section of skin layers with atopic dermatitis
Dermatologic

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

Dec. 11, 2024
Astria Therapeutics Inc. has gained IND clearance from the FDA for STAR-0310, a monoclonal antibody OX40 antagonist being developed for atopic dermatitis.
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Immuno-oncology

Pilatus Biosciences’ PLT-012 awarded orphan drug designation

Dec. 11, 2024
Pilatus Biosciences Inc.’s PLT-012 has been awarded orphan drug designation by the FDA for treating liver and intrahepatic bile duct cancer.
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Endocrine/metabolic

Discovery of FBPase inhibitor with improved activity and good druggability

Dec. 11, 2024
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.
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Antibody-drug conjugate illustration
Immuno-oncology

FDA clears IND for B7-H4-targeting ADC

Dec. 11, 2024
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.
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Cancer

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

Dec. 11, 2024
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.
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Dollar sign droplet above test tube
Infection

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

Dec. 11, 2024
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.
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Silhouette of head, brain
Neurology/psychiatric

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

Dec. 11, 2024
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.
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