GxP Insights: Developments in Cell & Gene Therapy

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Each edition delivers into a key topic within your industry, including expert perspectives, and career insights to help you stay ahead.

What you can expect:

• Industry News: Stay updated with the latest developments, trends, and breakthroughs in your field.
• Career Tips: Enhance your career with advice on skills, networking, and job opportunities tailored to your field.
  
  

What's happening in Cell & Gene Therapy?

Cell and gene therapy has grown into a multi-billion-pound sector, with over 20 gene therapies approved in Europe alone and hundreds of ongoing clinical trials globally. The market is projected to exceed £50 billion by 2027 as these innovative therapies become increasingly central to personalised medicine. 

The success of CAR-T therapies: paving the way for more advanced treatment

Gene therapy has transformed blood cancer treatment by modifying patients' immune cells to target cancer with high efficacy. Recent data from the University of Colorado Cancer Center highlights its expansion from third-line to second-line treatment for high-risk lymphoma, with nearly 50% of diffuse large B-cell lymphoma patients achieving remission at three months, often with durable results.
 
Researchers are now testing dual-target CAR-T therapies to boost remission rates to 70-80%. While risks like cytokine release syndrome persist, improved management has reduced these concerns. The development of “off-the-shelf” CAR-T therapies could further enhance accessibility by eliminating the need for extensive cell engineering. 

CRISPR-therapies: real-world applications and expanding trial

CRISPR technology drives fundamental change in gene-editing therapies, particularly in treating genetic disorders like sickle cell anaemia and beta-thalassemia. Trials by Vertex Pharmaceuticals and CRISPR Therapeutics show 90% of participants achieving durable symptom relief, potentially eliminating severe pain crises and frequent transfusions.
 
Research suggests CRISPR-based treatments could soon become standard for sickle cell disease, targeting the defective hemoglobin gene. Early trials show near-complete symptom relief in initial patients. In 2025, CRISPR trials will expand to more complex diseases like muscular dystrophy and certain cancers, showcasing its growing potential as a therapeutic tool.
 
More on this topic here: https://innovativegenomics.org/news/crispr-clinical-trials-2024/ 

Regulatory changes: aiming to accelerate innovation

Regulators are adapting to the rapid rise of cell and gene therapies. In the UK, the MHRA has updated frameworks to expedite reviews while ensuring safety and efficacy. Similarly, the FDA is streamlining access to gene therapies.

In 2024, the FDA’s CBER held listening sessions with patients to address safety concerns, aiming to shape guidelines that balance streamlined approvals with rigorous safety standards.

Key takeaways

🧬 Expanding Beyond Cancer: Researchers are applying CAR-T therapies to solid tumours and expanding CRISPR trials to target complex diseases like muscular dystrophy and inherited cancers, broadening the scope of these treatments.

🏭 Improving Manufacturing and Access: Simplifying production with “off-the-shelf” CAR-T therapies and scalable biomanufacturing aims to boost affordability and access to these life-saving treatments

🖥️ Integrating AI in Genomics: AI-driven data analysis is set to enhance treatment precision, shorten timelines, and improve patient outcomes in personalised medicine.

Companies to watch in Cell & Gene Therapy

Several pharmaceutical companies are at the forefront of cell and gene therapy advancements:

1. Vertex Pharmaceuticals and CRISPR Therapeutics (Boston, Massachusetts): the FDA is reviewing their groundbreaking CRISPR-based therapy for sickle cell disease. If approved, this could become the first CRISPR-based treatment to enter the market, representing a landmark moment in gene editing.

2. Bluebird Bio (Somerville, Massachusetts): after achieving approval for gene therapy treatments for beta-thalassemia, bluebird bio continues to expand its innovative portfolio. The FDA’s ongoing safety review of its Skysona therapy for cerebral adrenoleukodystrophy highlights the importance of regulatory diligence in advancing safe and effective treatments.

3. Kite Pharma (Gilead Sciences) (Santa Monica, California): a leader in CAR-T cell therapies, Kite has reported positive survival outcomes in both clinical trials and real-world applications for relapsed and refractory large B-cell lymphoma. In collaboration with Bristol Myers Squibb, Kite has streamlined CAR-T cell therapy manufacturing, reducing turnaround times to approximately 14 days, improving access for patients.
   
   
4. Bristol Myers Squibb: partnering with Kite Pharma to advance CAR-T cell therapy production. Their focus on reducing manufacturing timelines is crucial to scaling access to life-saving treatments.

5. Moderna: known for its mRNA vaccine innovation, Moderna is now entering the gene therapy space with a focus on rare diseases. This expansion positions the company as a leader in the next wave of therapeutic advancements.

6. Eli Lilly: recently achieved a major breakthrough with Tirzepatide, which reduced the risk of worsening heart failure events in clinical trials. Demonstrates the company’s dedication to addressing unmet medical needs with innovative therapies.

7. Pfizer: leveraging its global infrastructure to scale CAR-T therapy efforts and enhance manufacturing capacity.Poised to meet the growing demand for cell-based therapies.

8. Sarepta Therapeutics: recently signed a global licensing agreement to expand its gene therapy portfolio for Duchenne muscular dystrophy. This move further solidifies its leadership in addressing rare neuromuscular diseases.
   
   

Skills in demand for Cell & Gene Therapy

Stay ahead of these trends by ensuring your capabilities align with industry needs:

1. Commissioning, Qualification & Validation Engineering
 📈 Why It’s Needed: With advancements in CAR-T and CRISPR therapies, scaling production and ensuring compliance will require validation of systems and facilities.
📋 Key Skills: IQ, OQ, PQ processes, HVAC and environmental monitoring, temperature mapping, and equipment validation.
 
2. AI / Machine Learning
 📈 Why It’s Needed: Integrating AI and genomics demands advanced data infrastructure and automation for personalised therapies.
📋 Key Skills: Automation systems (e.g., Metasys, Rockwell), bioinformatics pipelines, cloud migration, and scaling data architecture.
 
3. Quality Assurance
 📈 Why It’s Needed: Regulatory changes and accelerated timelines necessitate rigorous quality oversight to maintain standards and resolve issues efficiently.
📋 Key Skills: CAPA development, quality audits, QMS optimisation, lean methodologies like Six Sigma and process walk-downs.
 
4. Regulatory Affairs
 📈 Why It’s Needed: Constantly evolving regulations for cell and gene therapies require expertise to navigate approvals and compliance for cutting-edge treatments.
📋 Key Skills: Experience with regulatory bodies (FDA, MHRA, EMA), managing INDs, NDAs, BLAs, and 510(k) submissions, and CMC strategy delivery. 

Growth Areas

Cell and gene therapy is thriving, especially on the U.S. East Coast in hubs like Massachusetts, North Carolina, Pennsylvania, New Jersey, and Minnesota. These regions host leading pharmaceutical companies, innovative biotech startups, and advanced manufacturing facilities. With innovation accelerating, demand for niche talent is soaring. Whether you're an industry expert or seeking to enter the field, we want to connect with you.

i-Pharm GxP operates at the heart of these industries, working closely with big players and innovators to support their developing needs. If you are passionate about the future of life sciences and want to explore opportunities in this exciting field, we’d love to hear from you. 

If you'd like to connect with one of our experts, please don't hesitate to contact us here.

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