RNA Clinical Trials Articles

31. FDA's Platform Guidance Raises Long-Term Questions For mRNA Makers 9/5/2024

    As I read through the FDA's draft platform designation guidance and listened to various panel discussions the past few months, a handful of additional observations and considerations came to mind that are important to reiterate for the RNA space, particularly as we advance into therapeutics development.

32. Two "Spicy" Takeaways On The FDA's Platform Designation Guidance 8/29/2024

    Over the past few weeks since the guidance was released, I’ve spent some time listening to industry conversations and reading the guidance and related articles. In the following series of articles, I’ll share a few general impressions and (slightly spicy) thoughts I’ve been mulling over in response to the draft guidance. 

33. Is This The Hardest Question Facing mRNA Therapeutics Makers? 8/18/2024

    As I’ve been reminded on a few different occasions, there is a difficult truth we may need to wrap our brains around in certain situations: Just because we can make an mRNA therapy for an indication, doesn’t necessary mean we should — or that the patient population will necessarily want it or be able to access it. 

34. AI And Pharmaceutical Development: WHO Calls For Ethical Framework, Good Governance 4/8/2024

    The World Health Organization (WHO) recently released a document titled Benefits and risks of using artificial intelligence for pharmaceutical development and delivery describing the ethical issues and governance considerations for using AI in drug discovery, preclinical development, and clinical trials.

35. FDA's Updated Guidance On Human Genome Editing: New Implications & Remaining Questions 2/27/2024

    On Jan. 29, 2024, the FDA released the final version of the guidance document on human gene therapy products incorporating genome editing. This article shares what changed between the draft and now final version of the guidance, and what open questions remain for the industry and scientific community to help resolve.

36. 1 In 300 People In The U.S. Is Living With HIV. That's Not Okay With Jeff Galvin. 2/13/2024

    By modifying CD4 T cells using miRNA, Jeff Galvin’s company thinks it can deliver a functional cure for HIV. Here’s the ongoing story of Addimmune and its unique clinical strategy.

37. 2024's Market Outlook For Cell & Gene Therapies 2/9/2024

    RSM is back for the third consecutive year to provide an outlook analysis of the cell and gene therapy market. This article discusses the 2024 state of the macroeconomy, its effects on cell and gene therapy companies, growing approval pipelines and approvals, funding factors, and the unique prospects for the market.

38. FDA Draft Guidance On “Rare Diseases: Considerations For The Development Of Drugs And Biological Products” 1/26/2024

    Accenture Applied Life Science Solutions' Raj Bandaru, Ph.D. breaks down, summarizes, and shares detailed insight on the recent FDA Draft Guidance that addresses “Rare Diseases: Considerations for the Development of Drugs and Biological Products.”

39. RNA Vaccines: Just Scratching The Surface 12/28/2023

    RNA vaccines have shown promise in preventing infectious diseases and treating cancer, but clinical development varies markedly between the two. HDT Bio’s Dr. Berglund discusses this and how they can be improved.

40. How To Break RNA's “4-Minute Mile” 12/20/2023

    Four CEOs provide their take on some of the hot topics of discussion in the RNA therapeutics space. Whether they be homing in on platform development, much-needed scientific advancements, or commercialization considerations, each of these experts’ quotes speaks to the important “training efforts” we should be building into our “workout” regimens to condition our companies and our products to achieve (and surpass!) RNA’s own “four-minute mile(s).”