Weekly Spotlight - 17.10.24

In the News

Wearable Sensors Aid Early Huntington's Detection and Treatment Trials

The FDA is funding research at the University of Rochester Medical Center to explore wearable sensors for early-stage Huntington's disease. These sensors aim to track subtle motor symptoms like gait and chorea, potentially aiding clinical trials. This initiative offers hope for early intervention and improved treatment outcomes.

New Hope for Huntington's: Interrupting Genetic Code to Delay Symptoms

Scientists are exploring a new approach to delay Huntington's disease by introducing CAG repeat interruptions in DNA. Dr. Jong-Min Lee's team used base editing technology to add these interruptions in cells and mice, showing promise for future therapies. This innovative method could potentially slow disease progression.

LoQus23's New Funding Boosts Huntington's Disease Research

LoQus23 Therapeutics has secured £35 million in Series A funding to advance its small molecule drug for Huntington's Disease. The company, based at Babraham Research Campus, focuses on DNA repair mechanisms to slow neurodegeneration. This funding supports pre-clinical and initial clinical studies, offering hope for patients.

Huntington's Disease Treatment Market to Reach £1.9 Billion by 2032

The Huntington's Disease Treatment Market is projected to reach £1.92 billion ($2.5 billion) by 2032, driven by a 20.8% CAGR. This growth stems from increased disease prevalence, advancements in genetic testing, and greater funding. Tetrabenazine leads the market, with North America showing significant demand due to improved diagnostics.

Oxford-Harrington Rare Disease Centre

David Cameron: Britain's Role in Advancing Rare Disease Treatments

David Cameron's article in The Times highlights Britain's potential to lead in treating rare diseases through genomics. Inspired by personal tragedy, Cameron reflects on the loss of his son Ivan to Ohtahara syndrome, a rare condition. He notes the rapid advancements in genomics since the early 2000s, when sequencing a genome was costly and time-consuming. Today, it is affordable and quick, enabling personalised medicine. Cameron's 100,000 Genomes Project marked a significant step in this field, and now, Genomics England and the NHS plan to sequence 100,000 newborns' genomes to identify rare diseases.

Cameron announces his role as chair of the Oxford-Harrington Rare Disease Centre's advisory council, a partnership aiming to develop 40 new treatments for rare diseases in the next decade. He emphasises Britain's strengths: a history of DNA research, a national health system, and a robust venture capital industry. Coupled with US expertise, this partnership is poised for breakthroughs in genomics.

The implications are vast. Genomics can help millions globally, as 80% of rare diseases have a genetic basis. Understanding one condition can unlock insights into others, including common killers like cancer and heart disease. Gene therapy, where Britain excels, offers potential to correct genetic defects, lessening or reversing conditions.