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Home » Huntington’s disease

Articles Tagged with ''Huntington’s disease''

Neurology/psychiatric

Nucleic acid splicing modulators disclosed in Ribopeutic patent

March 7, 2025
Ribopeutic Inc. has divulged nucleic acid splicing modulators (particularly, Huntingtin [HTT; HD] [mutant] and/or transcriptional activator Myb [c-Myb]) reported to be useful for the treatment of cancer and Huntington’s disease.
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Trimtech Therapeutics’ team

UK firm Trimtech emerges with $31M seed round for TRIM21 bispecifics

March 5, 2025
By Karen Carey
Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation treatments for neurodegenerative and inflammatory diseases.
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3D illustration of RNA and proteins
Neurology/psychiatric

Harness going after new target, FAN-1, in Huntington’s disease

Jan. 29, 2025
By Nuala Moran
Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.
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Doctor with brain illustration, businessman with dollar sign illustration
Neurology/psychiatric

Series B financing at Atalanta Therapeutics to advance RNAi therapies for CNS diseases

Jan. 29, 2025
Atalanta Therapeutics Inc. has completed a $97 million series B financing to support its investigational RNA interference (RNAi) therapies for KCNT1-related epilepsy and Huntington’s disease toward IND filings this year and through clinical proof of concept.
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3D illustration of RNA and proteins
Newco news

Harness going after new target, FAN-1, in Huntington’s disease

Jan. 28, 2025
By Nuala Moran
Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.
Read More
Histological staining of the caudate nucleus from a person with Huntington’s disease.
Neurology/psychiatric

Huntington’s neurodegeneration starts at 150 repeat expansions

Jan. 17, 2025
By Mar de Miguel
Having 35 copies of the CAG triplet in the gene that causes Huntington’s disease is not a problem. Inheriting 40 could be a sign that goes unnoticed for decades, until reaching 80. From there, the process accelerates and neural death occurs when reaching 150 repeats. Huntington’s disease neurodegeneration is not determined by what, but by how much, according to a study conducted at the Broad Institute.
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Illustration of human brain and dna

Uniqure stock doubles as FDA points to a BLA in Huntington’s

Dec. 10, 2024
By Lee Landenberger
The U.S. FDA has greenlit the first steps of Uniqure NV’s accelerated approval pathway for gene therapy AMT-130 to treat Huntington’s disease. The agency said data from the ongoing phase I/II studies compared to natural history external control are muscular enough to get the process going without having to dive into additional studies.
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Brain with handshake and cityscape

Phase II Huntington’s tie-up with Novartis could edge $3B for PTC

Dec. 2, 2024
By Randy Osborne
PTC Therapeutics Inc. said a “highly competitive process with several parties involved” led to the deal centered on the firm’s Huntington’s disease program with Novartis AG, an arrangement that brings $1 billion up front along with as much as $1.9 billion in development, regulatory, and sales milestone rewards.
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Illustration of double helix
Neurology/psychiatric

Sarepta Therapeutics licenses programs from Arrowhead Pharmaceuticals

Nov. 27, 2024
Sarepta Therapeutics Inc. has signed a licensing and collaboration agreement with Arrowhead Pharmaceuticals Inc. to obtain exclusive global rights to multiple clinical, preclinical and discovery-stage programs for rare genetic diseases of the muscle, central nervous system (CNS) and the lungs. The deal includes ARO-DUX4, ARO-DM1, ARO-MMP7 and ARO-ATXN2.
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Sage shuts down Huntington’s program after phase II failure

Nov. 20, 2024
By Lee Landenberger
The bad news keeps piling up for Sage Therapeutics Inc. Having absorbed other study stumbles in the past few months, the company now has halted development of dalzanemdor in treating Huntington’s disease after top-line phase II data showed it missed a statistically significant difference compared to placebo on the primary endpoint.
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