[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.] [@mike98572986](/creator/twitter/mike98572986) "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 Parkinsons and Alzheimers 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"  [@mike98572986](/creator/x/mike98572986) on [X](/post/tweet/1948183402378887191) 2025-07-24 00:48:13 UTC XXX followers, XXX engagements "$clpt $qure $rgnx Very good post by James Wilson big difference between those programs and the above companies the above companies are doing exactly this This contrasts with the relatively good safety profile of the broader portfolio of AAV products that deliver lower doses of systemic vector or use localized routes of administration. Overall low doses direct delivery As doctor Nicole Paulk has said Low doses + local admin = the way"  [@mike98572986](/creator/x/mike98572986) on [X](/post/tweet/1944904778636779711) 2025-07-14 23:40:08 UTC XXX followers, 2832 engagements "At the end of the day best thing we can all do is stay humble this game is extremely hard if Novartis can spend a billion dollars upfront and a couple months later find out that drug is likely useless do I believe that I can do the due diligence of a company like them Definitely not it is a perfect example of how tough biotech truly is. All each individual can do is what they are comfortable with understand the risks and go from there. I think anyone that has extreme confidence in anything is likely fooling ourselves. All we can do is make bets that make sense to each individual and live with"  [@mike98572986](/creator/x/mike98572986) on [X](/post/tweet/1945901343904047506) 2025-07-17 17:40:08 UTC XXX followers, XXX engagements "Or we can also argue if there is another drug for hd that is coming out very near term that will be a threat (better numbers easier to do etc) that would be another good bear thesis imo"  [@mike98572986](/creator/x/mike98572986) on [X](/post/tweet/1945890080939471223) 2025-07-17 16:55:22 UTC XXX followers, XXX engagements
[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.]
@mike98572986
"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 Parkinsons and Alzheimers 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"
@mike98572986 on X 2025-07-24 00:48:13 UTC XXX followers, XXX engagements
"$clpt $qure $rgnx Very good post by James Wilson big difference between those programs and the above companies the above companies are doing exactly this This contrasts with the relatively good safety profile of the broader portfolio of AAV products that deliver lower doses of systemic vector or use localized routes of administration. Overall low doses direct delivery As doctor Nicole Paulk has said Low doses + local admin = the way" @mike98572986 on X 2025-07-14 23:40:08 UTC XXX followers, 2832 engagements
"At the end of the day best thing we can all do is stay humble this game is extremely hard if Novartis can spend a billion dollars upfront and a couple months later find out that drug is likely useless do I believe that I can do the due diligence of a company like them Definitely not it is a perfect example of how tough biotech truly is. All each individual can do is what they are comfortable with understand the risks and go from there. I think anyone that has extreme confidence in anything is likely fooling ourselves. All we can do is make bets that make sense to each individual and live with" @mike98572986 on X 2025-07-17 17:40:08 UTC XXX followers, XXX engagements
"Or we can also argue if there is another drug for hd that is coming out very near term that will be a threat (better numbers easier to do etc) that would be another good bear thesis imo" @mike98572986 on X 2025-07-17 16:55:22 UTC XXX followers, XXX engagements