相关临床试验
8
5 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
N/A
进行中（未招募）
5
62.5%
招募中
3
37.5%
暂无批准数据
- Rudolph Tanzi and Reisa Sperling are leading a shift toward Alzheimer's prevention, with the fully enrolled AHEAD 3-45 trial testing lecanemab in cognitively normal individuals with elevated amyloid, results expected in the late 2020s. - A 2026 Annals of Neurology review supports combination disease-modifying therapy using repurposed drugs including GLP-1 receptor agonists, iron chelators, and chemical chaperones across multiple neurodegenerative targets. - More than 150 therapies are now in nearly 200 clinical trials targeting at least 17 different biological processes, with blood-based biomarkers like plasma pTau217 enabling earlier patient identification and trial enrichment. - Real-world data from the Alzheimer's Association's ALZ-NET shows significant functional benefits on daily activities within six months of treatment, while the U.S. POINTER trial found lifestyle interventions improved cognition in at-risk older adults.
- Scribe Therapeutics will showcase its X-Editor (XE) technology targeting mutations in Duchenne Muscular Dystrophy at the upcoming ASGCT Annual Meeting in May 2025. - The company will present joint research with Prevail Therapeutics, a Lilly subsidiary, demonstrating self-inactivating AAV vectors that achieved potent, transient editing in the murine central nervous system. - These presentations highlight Scribe's "CRISPR by Design" approach to developing in vivo genetic medicines, following a recent milestone in their collaboration with Eli Lilly to address neurological and neuromuscular diseases.
• Bayer's BlueRock Therapeutics initiated a Phase 1 trial of DA01, a cell-based therapy using stem cell-derived dopaminergic neurons, to replenish lost neurons in Parkinson's patients. • AskBio, another Bayer subsidiary, has begun a Phase 1b trial of its gene therapy for Parkinson's, aiming to halt or reverse motor control decline by delivering a gene for glial cell line-derived neurotrophic factor (GDNF). • The FDA granted fast-track review to BlueRock's DA01, potentially accelerating its development through increased communication with the regulator and a shorter review period. • These trials mark a significant step in Bayer's strategy to develop cell and gene therapies that could regenerate dopamine-producing neurons, addressing a major unmet need in Parkinson's disease.
• Prevail Therapeutics, a subsidiary of Eli Lilly, is evaluating PR001, an AAV9-based gene therapy, for Parkinson's disease (PD) patients with GBA1 mutations. • The Phase 1/2a PROPEL trial is an open-label, ascending dose study assessing the safety and efficacy of a one-time PR001 treatment. • The trial, which started in 2020 and is expected to complete in June 2029, is enrolling participants across multiple centers in the US and Israel. • Primary endpoints include monitoring treatment-emergent adverse events and assessing immunogenicity, while secondary endpoints focus on changes in glycolipid and enzyme activity levels.
• Multiple pharmaceutical companies are developing innovative gene therapies targeting specific mutations in frontotemporal dementia, with Denali Therapeutics' TAK-594/DNL593 showing promising Phase I/II results. • Scientists are exploring the genetic overlap between FTD and ALS, with companies like Wave Life Sciences developing antisense oligonucleotides that could potentially treat both conditions. • Researchers have identified a potential blood biomarker using neurofilament light chain protein, offering hope for earlier FTD detection and more effective treatment timing.
• Eli Lilly has agreed to acquire hearing loss gene therapy developer Akouos for up to $610 million, with an initial payment of $487 million and potential milestone-based payments of up to $123 million. • Akouos's lead candidate AK-OTOF, targeting otoferlin gene mutations in sensorineural hearing loss, has received FDA approval for clinical testing and could potentially benefit 200,000 patients worldwide. • The acquisition strengthens Lilly's gene therapy portfolio and expands its presence in genetic medicine, following its previous $1 billion acquisition of Prevail Therapeutics in 2020.
