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BioWorld - Friday, January 23, 2026
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Cardiovascular

Optimized MYBPC3 construct using novel cardiotropic capsid for the treatment of MYBPC3-associated HCM

Oct. 24, 2024
Researchers from Affinia Therapeutics Inc. have described the development and preclinical evaluation of a new AAV-based gene therapy, designed using a novel cardiotropic capsid, for the potential treatment of MYBPC3-associated hypertrophic cardiomyopathy (HCM).
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Cancer

New dimeric radiotracers for FAP-positive tumors presented

Oct. 24, 2024
Fibroblast activation protein (FAP) is absent or expressed at a very low level in normal tissues. However, it is overexpressed in around 80% of solid tumor stroma.
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Concept art for damaged DNA structure
Cancer

AI screening advances Rakovina’s DDR drug discovery work

Oct. 24, 2024
Rakovina Therapeutics Inc. has obtained a short-list of recommended drug candidates from an initial artificial intelligence (AI) screening effort using the Deep Docking AI platform under a collaboration with the University of British Columbia.
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Oncolytic virus concept illustration
Cancer

UP Oncolytics’ oncolytic virus-based therapy awarded US orphan drug designation to treat malignant glioma

Oct. 24, 2024
The FDA has awarded orphan drug designation to UP Oncolytics LLC’s oncolytic virus-based therapy (ZIKV-SJRP/2016) to treat malignant glioma.
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3D pancreas illustration
Cancer

Anti-uPAR antibody eradicates aggressive pancreatic mouse tumor

Oct. 24, 2024
At the European Association of Nuclear Medicine meeting, Monopar Therapeutics Inc. presented the preclinical characterization of MNPR-101, a first-in-class urokinase-type plasminogen activator receptor (uPAR)-targeting radiopharmaceutical agent with high stability in vivo and more than 1 week of shelf-life in its labeled form, [177Lu]-MNPR-101.
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DNA on digital background
Endocrine/metabolic

Novel precision B-cell gene therapy shows promise for the treatment of hypophosphatasia

Oct. 24, 2024
Be Biopharma Inc. has developed a CRISPR/Cas9-based precision B-cell gene therapy to deliver active tissue non-specific alkaline phosphatase (ALP) for the potential treatment of hypophosphatasia (HPP).
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Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia
Cancer

Harmonic Discovery presents FLT3 kinase inhibitor with enhanced safety profile

Oct. 24, 2024
FMS‐like tyrosine kinase 3 (FLT3) is a type III receptor tyrosine kinase validated as a therapeutic target for acute myeloid leukemia (AML) and regarded as an indicator of poor prognosis. Unfortunately, current FLT3 inhibitors, such as midostaurin, quizartinib or gilteritinib, often lead to myelosuppression or cardiovascular toxicity.
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Brain cancer illustration
Cancer

ESGCT 2024: Steps forward in gene and cell therapies for brain tumors

Oct. 24, 2024
By Mar de Miguel
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
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Immuno-oncology

[177Lu]LNC-1004 enhances the efficacy of anti-PD-L1 therapy in cancer

Oct. 23, 2024
The combination of radiotherapy with immune checkpoint blockade shows promise for synergistic cancer therapy efficacy. LNC-1004 is a vector targeting fibroblast activation protein (FAP) conjugated with Evans Blue for enhanced tumor uptake and retention. Xiamen University and the National University of Singapore have presented data regarding the [177Lu]LNC-1004 radiopharmaceutical in combination with anti-PD-L1 therapy for the treatment of pan-cancers.
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Neurology/psychiatric

Humanwell Healthcare describes new angiotensin AT2 receptor antagonists

Oct. 23, 2024
Humanwell Healthcare (Group) Co. Ltd. has patented compounds acting a type-2 angiotensin II receptor (AGTR2; AT2) antagonists reported to be useful for the treatment of neuropathy and neuropathic pain.
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