Despite missing the primary endpoint, the treatment improved vision for some patients.

J&J’s XLRP Gene Therapy Didn’t Meet Primary Endpoint in Phase 3 Clinical Trial

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* 💉 Rocket Pharma CRASHES after gene therapy death
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* 🩸 Synthetic blood substitute breakthrough—or ploy?
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🚬 Vaping Scandal, 🥩 Fake Meat & Cancer, 🩸 Synthetic Blood: Truth Revealed! 🔥

Astellas is doubling down on gene therapy while others retreat. Richard Wilson, SVP Primary Focus Lead, Genetic Regulation at Astellas, joins In Vivo to discuss why the company believes AAV technology will transform medicine despite current industry headwinds.

🎯 Key Topics:
- Why Astellas opened a massive new gene therapy manufacturing facility
- Building an end-to-end AAV platform from research to commercial
- Strategic partnerships driving CNS and rare disease programs
- Maintaining team morale in a challenging investment climate
- Regulatory outlook and the path to commercial success

Richard also plays a role in Astellas' West Coast Innovation Center and serves on the Alliance for Regenerative Medicine board. He shares insights on manufacturing at scale, patient-centric culture, and the long-term potential of gene therapy.

Perfect for biotech investors, pharma executives, and anyone tracking the evolution of genetic medicines.

🔗 More insights: insights.citeline.com/in-vivo

Astellas Doubles Down on Gene Therapy Despite Industry Headwinds

Audio roundup of selected biopharma industry content from Scrip over the business week ended July 18, 2025. In this episode: – J&J delivers in Q2 despite Stelara drop; Sarepta cuts jobs as Elevidys slumps; AI is inevitable but needs to be applied appropriately; China’s mid-cap companies thrive; and a look behind Glenmark’s landmark trispecific deal. 
Article links: https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-HYQOHZ42CNCQRPF6HCKGLTHUKM/

This episode was produced with the help of AI text-to-voice and voice emulation tools.

Playlist: soundcloud.com/citelinesounds/sets/scrips-five-must-know-things

Scrip's Five Must-Know Things - July 22, 2025

Tevard Biosciences CEO Daniel Fischer discusses how engineered suppressor tRNAs enable read-through of premature stop codons to restore full-length protein production in genetic diseases. Fischer, whose daughter has Dravet syndrome, covers the company's lead programs in dilated cardiomyopathy and DMD, advantages over traditional gene therapy approaches, and the path to human proof-of-concept.

Tevard CEO Sees Hope for HIs Daughter In His Company's tRNA Platform

Audio roundup of selected biopharma industry content from Scrip over the business week ended September 26, 2025. In this episode: Pfizer jumps back into obesity with Metsera bid; Roche maps out its obesity ambitions; UniQure’s encouraging gene therapy results for Huntington’s; BMS plans US pricing for Cobenfy in the UK; and vaccine skepticism affects vaccine commercial prospects.  
https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-HCVAK32IGREDHMH5D4I3RFY6AA/

This episode was produced with the help of AI text-to-voice and voice emulation tools.

Playlist: soundcloud.com/citelinesounds/sets/scrips-five-must-know-things

Scrip's Five Must-Know Things - Sept. 30, 2025

Audio roundup of selected biopharma industry content from Scrip over the business week ended October 17, 2025. In this episode: big pharma still sees value in cell and gene therapy; what to look out for at ESMO; world first for Kelun’s TROP2 ADC; J&J expects alignment with White House policies; industry uncertainties aired at BioFuture meeting. 
Story links: https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-ADAR4VZIGRFIFCOMXI7UTZS76U/

This episode was produced with the help of AI text-to-voice and voice emulation tools.

Playlist: soundcloud.com/citelinesounds/sets/scrips-five-must-know-things

Scrip's Five Must-Know Things - Oct. 20, 2025

Audio roundup of selected biopharma industry content from Scrip over the business week ended October 31, 2025. In this episode: third quarter dealmaking picks up; Novartis still has M&A firepower even after Avidity acquisition; Hanmi’s obesity contender shows Phase III promise; NVIDIA partnership progresses Lilly’s AI plans; and Lilly builds gene therapy push with Adverum buy. 

Story links: https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-MQMX2YHSAVHBTJFR4RAEAYNFNQ/

This episode was produced with the help of AI text-to-voice and voice emulation tools.

Playlist: soundcloud.com/citelinesounds/sets/scrips-five-must-know-things

Scrip's Five Must-Know Things - Nov. 4, 2025

本期播客内容来自《Scrip》杂志，精选内容涵盖截至2025年10月31日当周的生物制药行业的商业动态。本期节目采用人工智能文本转语音及语音仿真技术制作。

本次要闻: 本期要闻——第三季度并购交易回暖； 诺华收购Avidity后并购火力依旧；韩美制药肥胖症候选药物展现三期试验潜力；英伟达合作推动礼来人工智能计划；礼来收购Adverum强化基因疗法布局。

Story links: https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-MQMX2YHSAVHBTJFR4RAEAYNFNQ/

Playlist: on.soundcloud.com/gnr7PaOwflNKiAO7QX



Scrip 五件必知要闻: 药闻周析 - 全球制药风云聚焦 (Chinese-language podcast)

The Foundation funded preclinical studies for the approach and launched Opus. 

Opus Genetics Launches Gene Therapy Clinical Trial for Best Disease

Citeline places biopharma and medical device professionals, and those who focus on these industries, at the forefront of knowledge and insight, by providing the perfect combination of news and information together with penetrating insight and analysis. Citeline is a leader in the field of healthcare industry information.

The Foundation Fighting Blindness is the driving force in the global development of treatments and cures for blinding diseases. To learn more, go to FightingBlindness.org.

Empowering & Connecting People To Real Solutions For Health

New tracks tagged #genetherapy