Featured Editorial

31. Tessera Therapeutics Advances In Vivo Gene Therapy For SCD 2/24/2025

    Tessera Therapeutics' CSO, Michael Holmes, Ph.D., shares information on the company's Gene Writers for sickle cell disease (SCD) and how their work has garnered investment from of the Bill & Melinda Gates Foundation to support their efforts.

32. 10 Ways To Speed Up CMC In Early-Stage Drug Product Development 2/21/2025

    The inherent complexity of living cell-based products, vector-based gene therapies, and highly sensitive analytical methods demands robust change management strategies.

33. What Do We (Really) Know About DNA Quality For In Vitro Transcription? 2/19/2025

    Gene therapies and nucleic acid medicines are dependent upon a starting DNA template that defines the protein or antigen. The sequence of this template DNA being correct is critical.

34. FDA Issues Draft Guidance On The Use Of AI To Support Regulatory Decision-Making For Drug And Biological Products 2/14/2025

    The U.S. FDA issued a draft guidance, Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products, on January 6, 2025. The public comment period ends April 7.

35. Cultivating A Thriving mRNA Ecosystem: Key Initiatives & Future Directions 2/13/2025

    Overall, I felt that the following initiatives/talking points were demonstrative of the types of creativity and symbiosis that we will need much more of in the near and long-term to foster a healthy and fruitful RNA ecosystem. 

36. FDA Finalizes Guidance On The Advanced Manufacturing Technologies Designation Program 2/10/2025

    The FDA released final guidance on the Advanced Manufacturing Technologies Designation Program at the end of December. This article shares a summary, analysis, and how it compares to the 2023 draft.

37. IP Considerations For Early-Stage mRNA Therapeutics Development 2/7/2025

    Fortunately, navigating the patent landscape is hardly a “doomsday” scenario. But it is a challenge biotechs need to tackle early so they don’t end up too far down the path toward the clinic or in the clinic mired with delays and expensive litigation. Here, Rothwell Figg partner Dan Shores shares several strategies companies may choose to employ when facing a legal gray area to protect their products from patent litigation upon commercialization.

38. A Primer On The RNA Patent Landscape 1/31/2025

    In the first- of this three-part article, Shores gives us an important primer on the RNA legal landscape as it stands today. As many of us are working on crafting the next generation of mRNA/RNA products and LNPs, I wanted to get his sense of how our continued scientific advancement is likely to impact the IP landscape in the long-term.

39. From Promise To Precision: Defining The Therapeutic Niche for mRNA 1/21/2025

    CureVac CEO Alexander Zehnder shares his thoughts/visions on how mRNA neoantigen vaccines could become even more revolutionary for patient populations in the future. Though it may be early days, this conversation is a great case study for how RNA companies are striving to keep both scientific and commercial market opportunities top of mind from the earliest days of development.

40. mRNA Empowering Engineered In Vivo Cell Therapies 1/21/2025

    Recent advances in non-viral delivery technologies have enabled a first generation of genetically modified immune cells in vivo. Deploying these new approaches can address challenges with traditional ex vivo CAR-T cell therapy.