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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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September 30, 2022: CTTI’s Digital Health Trials Hub Recommendations and Resources to Run Your Digital Health Trial (Marianne Chase, MGH, CTTI Team Lead, Jörg Goldhahn, ETH Zurich, CTTI Team Lead)

Rethinking Clinical Trials

– One of the key challenges in the field of decentralized trials and digital measures is sharing tools and collaboration between companies. There’s a growing understanding that we have to collaborate on the endpoints and digital technology to advance the field. Discussion Themes. Find CTTI’s recommendations and resources.

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A look back at the “Joint-efforts for pathogen genomics in public health surveillance” symposium

Sciensano

BE READY project, but it is only the beginning for the collaborations and innovations that have been fostered. BE READY project coordinator, Transversal activities in Applied Genomics ( TAG )). Dr. Marie-Alice Fraiture ( TAG ). Dr. Sigrid De Keersmaecker ( TAG ). Dr. Kevin Vanneste ( TAG ). Dr. Nancy Roosens (.

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Grand Rounds January 20, 2023: Collaborative Pragmatic Trials in Action: EVOLVE-MI (Mikhail Kosiborod, MD)

Rethinking Clinical Trials

This is a collaborative study with Academic-Industry Partnership with oversight by an Academic Executive Committee. Patient recruitment is through a network of sites in different geographic locations managed by a collaboration of AROs. EVOLVE-MI is enrolling 4,000 patients in 3 countries.

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Grand Rounds February 2, 2024: Strategies for Improving Public Understanding of FDA and the Products It Regulates…Why Should We Care, and What Might We Do? (Susan C. Winckler, RPh, Esq)

Rethinking Clinical Trials

Potential strategies: “Prebunk” health misinformation; collaborate with other organizations (within and beyond government); monitor and respond to misunderstandings. Observation 2: Information vacuums breed misunderstanding. Observation 3: Communication clarity, and consistency, matters.

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Insights from the 18th Annual Canadian Summit on Food Safety in 2024

XTalks

Several common themes included the integration of technology like blockchain and AI in food safety, the importance of a well-trained workforce and the continuous need for industry collaboration. Read on to gain valuable insights from top industry leaders and learn more about the exciting innovations in food safety.

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Grand Rounds October 6, 2023: Hybrid Studies Should Not Sacrifice Rigorous Methods (David M. Murray, PhD; Moderator: Jonathan Moyer, PhD)

Rethinking Clinical Trials

-This was an impactful collaboration between NIH investigators and study teams and methodologists. How can this kind of collaboration happen more widely? This collaboration was a special case with a very good biostatistics and design working group. However, I don’t have a great general solution for that issue at this point.