ARTICLES BY ANNA ROSE WELCH

• Peter Marks: An Early Preview of mRNA, Gene Therapy Platforms

  11/13/2023

  Though Marks emphasized that this concept/designation at the regulatory level is still somewhat of a “we’ll-know-it-when-we-see-it” phenomenon, his remarks this summer — as well as a few additional discussions that have occurred throughout 2023 — add a bit more clarity to our understanding of what a gene therapy platform can be.

• “With This MSA, I Thee Contract:” The “Make-or-Breaks” In CGT Outsourcing Negotiations

  11/6/2023

  To help us all feel a bit stronger in the face of a negotiation, Haskett and Graskemper did not shy away from providing their go-to contracting best practices and tips for tackling an MSA and Quality Technical Agreement (QTA). While our discussion was far from exhaustive, they singled out several potentially showstopping areas that demand greater prescriptiveness in our MSAs for the sake of much stronger and long-term outsourcing partnerships. 

• What’s Mine Is (Maybe) Yours: Risk-Sharing In CGT Outsourcing

  10/25/2023

  Here, Haskett and Graskemper walk me through the different nuances of risk-sharing in the current CGT outsourcing space and how they identify the best partner, platform, and intellectual property (IP) strategy when reviewing and (re)negotiating an MSA.

• “Once Upon A Platform”: A Realistic Look At “Plug & Play” CGT Development

  10/2/2023

  We can’t just talk broadly about our need for more advanced capabilities from our outsourcing partners without closely examining the current maturity of CDMOs' AAV-based gene therapy and LVV-based cell therapy platforms. Lucky for us, Haskett and Graskemper shared their top tips and tricks for evaluating the seaworthiness of current CGT platform offerings. 

• From Capacity To Comparability: The Shifting Onus In Cell & Gene Therapy Outsourcing

  9/18/2023

  As we continue progressing beyond the COVID (i.e., the seller’s) economy, both Haskett and Graskemper agree we’re steadily seeing biotechs taking on a much more empowered role in negotiating with and securing CDMO partners. But they also see several shifts in the CGT development landscape that could impact both biotechs and their outsourcing partners equally — for the better, or if we’re not careful, for the worse.

• Sarepta’s FDA Adcom: Manufacturing Comparability In The Real World

  9/8/2023

  There are a few must-know details about the why & how of Sarepta’s comparability exercise, which I’ll outline for you here. But it wasn’t the facts and figures underpinning the comparability assessment itself that I found the most noteworthy; rather, this adcom served as an important reminder about the overall role comparability can and likely will play in the regulation of our advanced therapies moving forward.

• FDA To Sarepta’s Gene Therapy: “Express Yourself…Maybe?”

  8/21/2023

  Though we cling to — and simultaneously despair over — the fact that regulatory experiences are product-specific, the hurdles/debates Sarepta faced remind us of the scientific and clinical complexities our future CGT products will no doubt be faced with during their own BLA reviews.

• “Alice In [The mRNA] Wonderland:” 3 Realities Facing The mRNA Industry

  8/7/2023

  The mRNA industry is an exciting place to be, and I have no doubt we will celebrate some significant triumphs in the future. But there are three critical realities (or perhaps difficult truths) I was reminded of during the mRNA Therapeutics Summit worth reiterating to keep us grounded through all our uninhibited dreaming.

• A Meditation On Change In The CGT Manufacturing Space

  7/31/2023

  A few weeks ago, I published an article recapping my biggest takeaways from the PDA ATMP conference. Throughout the many technical discussions of the two-day event, I was predominantly struck by how often the conversation circled back to the importance of humility and self-improvement in our CMC. Since publishing that article, I’ve continued to read and hear more about the importance of “growing up” in CMC. 

• A “Portrait” of Risk In Cell & Gene Manufacturing

  7/21/2023

  The eventual shape of our industry became increasingly top-of-mind as I listened to the final expert panel at the PDA ATMP conference, during which several experts shared the industry challenges currently keeping them up at night. Not surprisingly, many of these experts’ concerns revealed the tenuous relationship we as an industry have with our (often vastly different) assessments of risk in the CGT manufacturing paradigm. 

• From The Lion King To Cell & Gene: “The Circle of Life” For Sustainable CGT Products & Businesses

  7/10/2023

  We tend to think of sustainability predominantly in terms of environmental/social initiatives. However, the 2023 AGC CDMO Summit presentations also shed light on three additional types of sustainability — and the mindsets underpinning them — that are essential for establishing an efficient business without placing our manufacturing and quality initiatives at risk.

• “Our CMC, Ourselves:” 3 Iterative [Self] Improvements In Cell & Gene Manufacturing

  6/30/2023

  Here, I’ll outline the three areas in which I felt the discussions at the PDA ATMP conference revealed how we can better “close the gap” between where we currently are and where we know our manufacturing processes can be in the future. 

• The “Corps de Manufacturing:” Defining Flexibility In Cell & Gene Therapy Tech Platforms

  6/15/2023

  Though the word “flexible” will inevitably appear in any conversation about a manufacturing facility, paradigm, or platform, we rarely stop to flesh out what such flexibility can mean and/or may look like. Here, in part two of this series, Abe Maingi of Inceptor Bio and Jason C. Foster of Ori Biotech help me chisel out how they are thinking about flexibility in tech provider partnerships in the heterogenous ATMP space. 

• Defining Risk-Sharing In Cell & Gene Therapy Manufacturing Partnerships

  6/7/2023

  In part 1 of this two-part article, Jason C. Foster, CEO and executive director of Ori Biotech, and Abe Maingi, VP of business development and co-founder of Inceptor Bio, share their perspectives on how partnerships must evolve to mitigate the unique scientific and financial risks facing autologous cell therapies. 

• Where Fly-Fishing Meets mRNA: The Art Of mRNA Immunogenicity

  5/22/2023

  In the final installment of this two-part series comparing fly-fishing and mRNA immunogenicity, I summarize some of the basic immunology-centric underpinnings of our mRNA development efforts and the enduring immunogenicity questions and concerns shaping and re-shaping these efforts today.