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BioWorld - Monday, December 8, 2025
Breaking News: Trump administration impacts continue to roil the life sciences sectorBreaking News: ASH 2025: Casgevy for kids? Expanding, improving SCD gene therapiesBreaking News: Trump administration impacts continue to roil the life sciences sectorBreaking News: Trump administration impacts continue to roil the life sciences sector
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Research lab illustration
Cancer

FTX-001, an ASO targeting human MALAT1 lncRNA with promising benefit-risk profile in preclinical models

June 25, 2024
Flamingo Therapeutics BV and Ionis Pharmaceuticals Inc.’s FTX-001 (also known as FLM-7523) is a potential first-in-class antisense oligonucleotide (ASO) targeting the human metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) long noncoding RNA (lncRNA); it is being developed for the treatment of cancer.
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CAR T cells attacking cancer cell
Immuno-oncology

Adicet’s λδ CAR T-cell therapy ADI-270 gains IND clearance for renal cell carcinoma

June 25, 2024
Adicet Bio Inc. has obtained FDA clearance of its IND application to evaluate ADI-270, an armored allogeneic λδ CAR T-cell therapy candidate targeting CD70-positive cancers, for the treatment of relapsed or refractory renal cell carcinoma (RCC).
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Close up of man's eye
Ocular

4D Molecular Therapeutics’ 4D-175 gains FDA clearance for phase I trial in geographic atrophy

June 25, 2024
4D Molecular Therapeutics Inc. has obtained IND clearance by the FDA for 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with geographic atrophy.
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Cancer

Kinase inhibitor AD-80 shows antileukemic effects in FLT3-ITD AML

June 25, 2024
Acute myeloid leukemia (AML) is characterized by the accumulation of immature blasts in the bone marrow or in the peripheral blood.
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Neurology/psychiatric

Seed funding at Epilepsygtx to advance gene therapy for focal refractory epilepsy

June 25, 2024
Epilepsygtx Ltd. has raised a total of $10 million in seed funding to support its development of gene therapies to treat focal refractory epilepsy.
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Illustration of DNA strand next to lungs
Respiratory

In vivo mRNA editing of cystic fibrosis mutations in mice

June 25, 2024
Scientists at Recode Therapeutics Inc. have developed an optimized lipid nanoparticle (LNP) to act on specific tissues.
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Concept art for targeting cancer
Immuno-oncology

Immutep licenses rights to anti-LAG-3 small molecules from Cardiff University

June 25, 2024
Immutep Ltd. has signed a license agreement with Cardiff University granting the company exclusive rights to develop and commercialize anti-LAG-3 small molecules.
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Digital handshake
Immune

Astrazeneca selects SLE target under collaboration with Benevolentai

June 25, 2024
Astrazeneca plc has added a novel target for systemic lupus erythematosus (SLE) to its discovery portfolio through its collaboration with Benevolentai Ltd. The novel target was discovered using Benevolentai’s artificial intelligence (AI) drug discovery platform and experimentally validated by Astrazeneca.
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Urology

EG 427 obtains IND clearance for gene therapy for neurogenic detrusor overactivity in spinal cord injury patients

June 25, 2024
EG 427 SAS has received IND clearance from the FDA for EG-110A, a gene therapy for the treatment of neurogenic detrusor overactivity in patients with spinal cord injury. A phase Ib/IIa study is being initiated.
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Sunrise in a desert with sand dunes
Inflammatory

Study gives insight into five inflammatory diseases, and the noncoding genome

June 25, 2024
By Anette Breindl
A recent paper has identified the enhancer ETS2, located in a so-called gene desert, as a contributor to five separate immune disorders. It also showed that one of ETS2’s target genes mediating this inflammation was the eminently druggable MEK, a kinase that is the target of the FDA-approved inhibitors Mekinist (trametinib, GSK plc), Mektovi (binimetinib, Array Biopharma Inc.), Cotellic (cobimetinib, Roche Holding AG) and Koselugo (selumetinib, Astrazeneca plc/Merck & Co. Inc.).
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