Cell and gene therapy (CGT) is one of the most advanced areas of medicine that can effectively transform our own bodies into disease-fighting agents. 

In the rapidly evolving landscape of modern medicine, CGT have emerged as groundbreaking approaches with the potential to revolutionize how we treat a myriad of diseases. As the biopharma industry and research institutions ramp up their efforts in this domain, a thorough understanding of global trends becomes paramount. This FAQ delves into the promising developments in the field of CNG treatment, focusing on innovative therapies, clinical trials, and their potential impact on patient outcomes. DOWNLOAD THE FULL WHITEPAPER HERE

What is the Current Status of Approvals in the CGT Sector?

By the end of 2023, over 12 novel CGT might be approved in the United States, Europe, or both. For example, the first CRISPR therapy-based medicine to treat Sickle cell disease and Beta thalassemia from Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG might be licensed for patients in the United States, United Kingdom, and European Union in the next year or two. Similarly, gene therapy for Duchenne Muscular Dystrophy, a condition whose R&D journey has been marred by setbacks, might receive fast track approval by the FDA in 2023. In addition, Intellia Therapeutics, Inc.’s in vivo gene-editing solutions for two rare diseases - ATTR amyloidosis and hereditary angioedema - are making progress in the clinic. 

Furthermore, the scientific community has begun to pay a lot of attention to CAR T-cell treatments for cancer. They have demonstrated a comparable capacity to eradicate extremely advanced leukemias and lymphomas as well as postpone disease development for a longer period, despite not being used as frequently as immune checkpoint inhibitors. Approximately six CAR T-cell therapies have received FDA approval for the treatment of hematological malignancies (lymphomas and leukemias), and most recently multiple myeloma. 

What are Some of the New Treatments?

A number of gene therapies reached the bedside in 2021. Notable among these is Zolgensma, Novartis’ gene therapy medicine for spinal muscular atrophy (SMA). Likewise, both Legend Biotech/Janssen and Bristol Myers Squibb/ bluebird bio received approval for multiple myeloma CAR-T therapies in 2022. 

By the end of 2023, over 12 novel CGT might be approved in the United States, Europe, or both. 

Where are the Clinical Trials being Conducted?

CNG therapy trials percentage share by region for the 5Y period (2018-2022) shows that participation in:

  • APAC increased from 41% in 2018 to 48% in 2022
  • North America and Europe together dominated with 54% in 2018 but fell to 47% in 2022 The Rest of the World (ROW) retained a tiny share of roughly 5%, demonstrating the worldwide reach of CGT trials beyond the key regions. 

Overall, the data illustrates the industry’s dynamic nature, with APAC continuing to develop, North America and Europe maintaining their dominance, and ROW adding to global diversity in CGT research. 

What is the Projected Growth in this Sector?

According to Evaluate Pharma’s global sales predictions, biologic drug sales excluding CGT treatments are expected to increase by 5% from 2021 to 2026, from around $400 billion to over $500 billion. CGT treatments, on the other hand, are predicted to expand from $4 billion in sales per year to over $45 billion over the same timeframe, representing a substantially higher CAGR (compound annual growth rate) of over 60%. Hence companies with excellent CGT pipelines emerge as attractive acquisition targets due to the potential of quick revenue growth compared to other biologics and conventional pharmaceuticals. 

What are Some of the Global CGT Trial Trends?

Of the approximate 2,000 CNG based trials initiated in the 5Y period, more than 70% of the trials involved a single country, with fewer than 30% involving multiple locations. 

Mainland China and the United States, each with over 600 trials, were the leading locations in CGT trials. 

In the 5Y period from 2018 to 2022, the APAC region’s single country CGT trials share was 90%. This proportion was much greater than the 69% share for CGT trials of North America and 35% share for CGT trials of Europe. 

Which therapeutic areas are being targeted with CGT?

Oncology appeared as the most actively researched category across all three regions of APAC, North America, and Europe. However, the number of oncology research undertaken in APAC was more than double that of North America and more than 10 times that of Europe. 

APAC also demonstrated leadership in CGT studies for musculoskeletal problems, cardiovascular diseases, hematological and respiratory disorders. 

When the top indications explored in these trials were analyzed, hematological malignancies emerged as the most widely researched indications. Lymphomas, specifically ALL, B-cell NHL, DLBCL, FL, and AML, as well as multiple myeloma, received a lot of attention. Following hematological malignancies, solid tumors, gastrointestinal tract cancers, gynecological cancers, pancreatic cancers, and lung cancers were also extensively researched areas. 

Overall, APAC led in most of the main indications studied in CGT trials, closely followed by North America. 

What are the Molecule Sub-types in Trials?

In the 5Y period between 2018 and 2022, there were notable global trends in the distribution of CNG therapy trials by subtype. Gene modified cell therapy accounted for the highest share of 43% of the CGT trials done over this timeframe. Cell therapy came second with 39% share of trials, while gene therapy accounted for about 18%, which is less than a fifth of the CNG trials. 

What are the Manufacturing Innovations and Scaling Production Capacity Developments?

Scaling up manufacturing processes is a pivotal challenge in gene therapy. As gene therapies become more sophisticated, the ability to produce these treatments at scale is a critical step towards making them accessible to a broader patient population. Innovative technologies are paving the way for efficient and scalable manufacturing, bridging the gap between research and widespread therapeutic application.

What does the future hold?

CNG stand as beacons of hope in a rapidly evolving healthcare landscape. As the global community collaborates to advance these therapies, we are witnessing a seismic shift in how diseases are treated. With their potential to rewrite the medical playbook by altering genes and harnessing the body's cellular machinery, cell and gene therapies are unlocking new frontiers in medicine. By understanding clinical trial global trends, patient recruitment metrics, funding dynamics, manufacturing innovations, and emerging therapies, we are not just witnessing history – we are actively shaping the future of healthcare.