With its hands full developing QRL-101 and QRL-201, both in clinical trials for amyotrophic lateral sclerosis with plans to expand into other neurodegenerative diseases such as frontotemporal dementia, Quralis Corp. has decided to out-license its preclinical ALS and FTD drug, QRL-204.

Researchers from Macquarie University have detailed the discovery of a novel gene therapy vector targeting pathological TAR-binding protein 43 (TDP-43), CTx-1000, as a potential therapeutic candidate for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) – two diseases characterized by cytoplasmic deposition of the nuclear TDP-43.

Acurastem Inc. has raised nearly $7 million in grant funding from the National Institutes of Health (NIH) and the Department of Defense (DoD) to advance research in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.

Psilera Inc. has selected behavioral variant frontotemporal dementia (FTD) as the lead...

Coya Therapeutics Inc. intends to expand proposed indications for COYA-302 beyond amyotrophic lateral sclerosis (ALS) to include frontotemporal dementia (FTD) and Parkinson’s disease.

Acurastem Inc. has entered into a license agreement with Takeda Pharmaceutical Company Ltd. to develop and commercialize Acurastem’s PIKFYVE-targeted therapeutics, including AS-202, an innovative antisense oligonucleotide (ASO) for the treatment of amyotrophic lateral sclerosis (ALS).

There is a strong link between the loss of TAR DNA-binding protein 43 (TDP-43) function and the development of frontotemporal dementia (FTD)...

Several neurodegenerative disorders have TAR DNA-binding protein 43 (TDP-43) inclusions as a pathological hallmark; thus, the development of PET tracers able to detect TDP-43 aggregates is essential to advance the diagnosis and treatment monitoring in diseases such as frontotemporal dementia, amyotrophic lateral sclerosis and others.

Researchers from Applied Genetic Technologies Corp. have reported preclinical data for AGTC-601, a novel AAVrh10-granulin (GRN) gene therapy being developed for the treatment of frontotemporal dementia (FTD) with GRN mutations.

Frontotemporal dementia (FTG-GRN) is a fatal neurodegenerative disease caused by heterozygous mutations in the GRN gene, encoding progranulin (PRGN). Researchers at Arkuda Therapeutics Inc. reported on ARKD-104, a novel potent orally bioavailable PGRN-enhancing compound that increased PRGN secretion and lysosomal granulins in multiple relevant cell lines.