CLINICAL TRIALS ARTICLES

  • Personalized mRNA Cancer Immunotherapies: Closing The Biological Knowledge Gap

    it was a great honor to take Advancing RNA on the road to London to CPI’s/BIA’s/Imperial College London’s RNA Vaccines & Therapeutics Conference to moderate a discussion on personalized cancer immunotherapies. In the following two-part article, I’ll share the advancements needed in two areas — biological knowledge and manufacturing — to ensure we’re capable of meeting patients’ needs in the personalized sector.

  • The In Vivo CAR-T Playbook: Differentiating Your mRNA Candidates

    Fortunately, as my conversation with ParcelBio CEO David Weinberg emphasized, there’s still a lot of room to grow in terms of what our RNA-based products can be and how they can perform. Here in part 2 of this two-part article, Weinberg walks me through how ParcelBio is approaching this market and helping define what a competitive mRNA product will look like in the in vivo CAR-T space. 

  • Inside the In Vivo CAR-T Boom: Where Does mRNA Fit In?

    I had the chance to sit down with ParcelBio’s CEO David Weinberg to learn more about the emerging market dynamics in the in vivo CAR-T space, with a particular focus on the challenges facing RNA biotechs looking to navigate this up-and-coming therapeutic class.

  • What Baby KJ Taught Us About Patient & Physician Communication

    Over the past few weeks, I’ve been sharing my takeaways from the overall event, as well as from a specific panel featuring JHU’s Jeff Coller, CHOP’s Rebecca Ahrens-Nicklas, and Plowshare Therapies’ Kevin Strauss. Together, these three discussed the biggest learnings and takeaways from the miraculous Baby KJ experience that we very much hope to replicate in the future. As you can likely imagine, patient and physician communication played a large role in the discussion at hand. 

  • What Baby KJ Taught Us About mRNA COGS

    Here, in part 2 of this three-part series, I share a few details that came to light about the COGS behind Baby KJ’s therapy production. Obviously, we know why it’s important to keep these figures in mind as we move forward; but I particularly liked Strauss’ reminder that it’s our job to “think systematically about how we’re going to create a safety net of sustainable, accessible product that can be widely distributed at an affordable cost.”

  • What Baby KJ Taught Us About Sustainable mRNA Development

    Of course, these conversations are rarely simple, and the learnings are multifaceted. In the following three-part series, I’ll share my 3 biggest takeaways from the discussion between Coller, Ahrens-Nicklas, and Strauss. Here in part 1, I’ll start by parsing out the learnings about drug development we can take away from the overall heroic efforts to dose Baby KJ. 

CLINICAL TRIALS VIDEOS

Tessera Therapeutics' CEO, Dr. Michael Severino, joins Host Erin Harris to discuss Gene Writing, the biotech's genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source. Severino discusses why delivery has always been a challenge for gene editing technologies and why Gene Writing is different when it comes to efficient delivery.

Discover how the clinical research bridge between China and the US supports accelerated clinical development from early to late phase trials while ensuring the inclusion of MRCT data packages in future regulatory applications.

The Advancing RNA Live panelists share several inconsistencies they’ve encountered working with global regulators, as well as on which aspects they’re seeing regulatory agencies applying greater scrutiny in mRNA development today.

Among the more prevalent genetic conditions, Duchenne muscular dystrophy affects an estimated one in 3,500 male births worldwide. It's caused by mutations of the DMD gene, which regulates the production of a protein called dystrophin. Approved DMD treatments haven't demonstrated strong clinical outcomes, but James McArthur, Ph.D. and his team at PepGen are seeking to change that with a pipeline of disease-modifying peptide-conjugated oligonucleotide candidates derived from the company's Enhanced Delivery Oligonucleotide platform. The Business of Biotech caught up with Dr. McArthur at PepGen's Cambridge headquarters to learn more.

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