[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.]  mike [@mike98572986](/creator/twitter/mike98572986) on x XXX followers Created: 2025-07-24 00:48:13 UTC Interview about gene therapies The future of neuroscience, according to Dr. Putzki, resembles oncology in the 1990s — a field on the verge of rapid expansion and breakthroughs. Among the most promising areas: Targeted therapies for neurodegenerative diseases like ALS, Parkinson’s and Alzheimer’s, where delivery methods and target engagement are central challenges Next-generation AAV vectors, which aim to improve the safety and efficacy profile of gene therapies Regulatory innovation, including the use of surrogate endpoints, confirmatory data collection post-approval and alternative trial designs CRISPR-based therapeutics, which remain in early days but hold potential to address genetically defined neurological disorders But perhaps the greatest bottleneck remains the blood-brain barrier — and how to overcome it non-invasively. “If we can solve delivery into the CNS without surgery, we can unlock transformative therapies across a range of conditions,” said Dr. Putzki. Gene therapies developed for orphan diseases have shown notably higher success rates across all stages of clinical development compared to the average drug. These therapies are XX% more likely to advance past Phase I, XX% more likely to succeed in Phase II, and XX% more likely to complete Phase III. Even during regulatory review, orphan gene therapies outperform, with a XX% higher likelihood of successfully completing a New Drug Application (NDA) or Biologics License Application (BLA). This underscores their potential to address conditions that were previously untreatable.  XXX engagements  **Related Topics** [alzheimers](/topic/alzheimers) [promising](/topic/promising) [Post Link](https://x.com/mike98572986/status/1948183402378887191)
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mike @mike98572986 on x XXX followers
Created: 2025-07-24 00:48:13 UTC
Interview about gene therapies
The future of neuroscience, according to Dr. Putzki, resembles oncology in the 1990s — a field on the verge of rapid expansion and breakthroughs. Among the most promising areas:
Targeted therapies for neurodegenerative diseases like ALS, Parkinson’s and Alzheimer’s, where delivery methods and target engagement are central challenges
Next-generation AAV vectors, which aim to improve the safety and efficacy profile of gene therapies
Regulatory innovation, including the use of surrogate endpoints, confirmatory data collection post-approval and alternative trial designs
CRISPR-based therapeutics, which remain in early days but hold potential to address genetically defined neurological disorders
But perhaps the greatest bottleneck remains the blood-brain barrier — and how to overcome it non-invasively. “If we can solve delivery into the CNS without surgery, we can unlock transformative therapies across a range of conditions,” said Dr. Putzki.
Gene therapies developed for orphan diseases have shown notably higher success rates across all stages of clinical development compared to the average drug. These therapies are XX% more likely to advance past Phase I, XX% more likely to succeed in Phase II, and XX% more likely to complete Phase III.
Even during regulatory review, orphan gene therapies outperform, with a XX% higher likelihood of successfully completing a New Drug Application (NDA) or Biologics License Application (BLA). This underscores their potential to address conditions that were previously untreatable.
XXX engagements
Related Topics alzheimers promising