Blog
November 15, 2021

Preparing for Cell and Gene Therapy 3.0

Advanced therapies are at the forefront of innovation in medical science and the sector is experiencing rapid, multi-dimensional growth. From cell and gene therapies (CGTs) to personalized cancer vaccines, these patient-specific therapeutics represent a new era of treatments and hope for patients with a wide range of serious diseases, including cancer, central nervous system disorders, cardiac disorders, and infectious diseases. 

There are currently more than 5,000 unique cell therapy and gene therapy products in development,1 many representing a new wave of “CGT 3.0,” building on the dendritic cell and CAR-T cell approvals over the last decade. Additionally, the field is increasingly utilizing cutting edge technologies such as AI to precisely target tumor cells and develop AAV capsids that evade the immune system.2,3

Title: The number of unique advanced therapy products is growing rapidly
Subtitle: Growth in the advanced therapies sector is multi-dimensional. The technology types are evolving, new cell types are being explored, and the gene therapy sector is expanding. 
[Reference 1]

Two newer therapy modalities with significant momentum are allogeneic (donor-based) therapies and therapies targeting solid tumors. More than 40% of the unique new products in development are allogeneic CGTs and nearly 20% are personalized cancer vaccines.1 Each will potentially solve key existing challenges, but bring new complexities of their own, further reinforcing the need for industry-wide operational innovation to support the evolving science.

Allogeneic therapies: operational challenges

Allogeneic therapies are of great interest because of the potential to develop “off-the-shelf (OTS)” — or “one to many” — doses of a therapy, where one donor’s cells can be used to create multiple doses of a therapy. OTS allogeneic therapies could potentially solve the starting material problems of autologous therapies and some of the supply chain complexity, but introduce new challenges.

Introducing donors to the process increases the intricacy around traceability, data, and coordination of events. The donor — along with their data and product journey events—must be matched, traced, and coordinated with the patient’s COI and COC. This is known as “Look back, look forward” (LBLF), and it is vital that accurate and accessible LBLF is established and maintained for long-term follow up needs. Additionally, not all allogeneic therapies operate in a true “one to many” paradigm, and identifying and enrolling donors add a new, complicated dimension. 

Solid tumor therapies: operational challenges

Solid tumors have exceedingly hostile, elusive micro-environments and have often proved resistant to immunotherapies. Many of the potential new therapies being developed rely on tumor tissue samples as a foundational raw material or component. 

Tumor collections for CGTs and personalized cancer vaccines are often involved, highly variable, and difficult to manage. The collection, storage, and transportation requirements are varied and sensitive and scheduling with surgeons takes additional coordination. In addition, the health care providers and systems working with cancer patients are often not set up to operate to cGMP standards or  serve as an integral part of cell therapy manufacturing. 

[Reference 4]

Solutions for a successful CGT 3.0

The industry is at a pivotal point in preparing for the scaling and industrialization required to treat larger patient populations. There is an opportunity to solve operational challenges facing “CGT 3.0” through a combination of innovation and proven success strategies. In upcoming articles in December 2021, we will explore each of the topics from this blog post in more detail and provide learnings and strategies from key industry veterans on how to successfully navigate this new wave of innovation.

Author Biographies

Joe DePinto, Chief Commercial Officer, Vineti

Joe brings more than 28 years of executive leadership in biotech, pharmaceuticals, specialty pharma, and cell therapy to Vineti. He joins us from Cardinal Health, where he served as the President of Specialty Solutions, leading one of the fastest-growing businesses within the Fortune 14-ranked company. Prior to Cardinal Health, Joe’s roles included leadership positions at top pharmaceutical companies, including Johnson & Johnson and Lilly. His core leadership competencies include leading all aspects of strategy, drug development, investor relations, and commercialization with multiple global launches. He also previously served in executive roles at Sunesis Pharmaceuticals, Dendreon, ImClone, and Abraxis.

Richard Gaeto, Principal, SupplyLinc

Rich has been a Technical Operations Executive in the biotech and the Cell and Gene therapy industry for over 30 years. He has an extensive background in commercial launches such as Enbrel, Provenge, Neulasta, and Recothrom, global supply chain strategies, and systems integration regarding personalized medicine, Chain of Custody and Chain of Identity. He has held executive positions at Iovance as Sr. Vice President of Technical Operations, Vice President of Technical Operations at ZymoGenetics, and Vice President of Commercial Operations at Dendreon. In addition to on-going consulting roles, Rich has recently completed a full-time Head of Operations position at Imvax focusing on CDMO selection, build out of internal manufacturing, and establishment of an integrated solution regarding CRO, Manufacturing, and Patient Scheduling. Prior to these activities, Rich has held positions at Amgen, Immunex, and Centocor.

References

  1. Evaluate Pharma, Vineti proprietary database. 2020 and 2021.
  2. AI-driven ‘Smart’ Cell Therapy for Treating Cancer. BioTechScope. December 2020.:  https://biotechscope.com/ai-driven-smart-cell-therapy-treating-cancer/
  3. AI Can Make Gene Therapy Design More Effective. ClinicalOmics. February 2021.:  https://www.clinicalomics.com/topics/precision-medicine-topic/gene-therapy/ai-can-make-gene-therapy-design-more-effective/
  4. Choy, Michael. Peter Marks Discusses the Road Ahead for Cell and Gene Therapy. Bcg.com. August 2021.: https://www.bcg.com/publications/2021/peter-marks-discusses-cell-gene-therapy