Cell and gene therapy are emerging treatments for genetic diseases and cancers. No doubt that the biotech industry is growing exponentially at 30% CAGR. It is expected to reach around US$ 15bn by 2025. However, biotech companies that manufacture such CGT treatments still operate manually and face challenges such as unscalability, human judgement errors, irregular batch results, complex supply chain, irregularity at the patient and cellular level, cell expansion count, and the rigid programming time of the batch disposition process. All this leads to batch loss, contamination risk, low quality, variation in batch-to-batch results – these invariably increase the production cost and fail to meet the compliance guidelines.

How is Digitizing manufacturing processes beneficial?

Digitization is a crucial step to automatize CGT manufacturing processes. But digitization doesn’t only mean converting paper documents to digital records. The companies need to adopt an automated manufacturing process that is simple and agile, compliant with the FDA rules, and adhere to a shorter production timeline.

Artificial intelligence and machine learning technologies have a major role to play in the automation of CGT manufacturing. It helps in the following ways:

• Cloud-based infrastructure that allows centralized storage platforms that can be accessed and analyzed on a real-time basis
• Lower bar for the entry of developers
• Data-driven decision making
• Removal of manual handling of products to ensure zero contamination and improve the efficiency of cell-based products
• Wrong handling of paperwork can lead to poor batch records, (EBR) Electronic Batch Recording system ensures zero error during the clinical trial
• It speeds up the design of manufacturing batch records
• It reduces any deviations from cGMP and follows all the regulations laid by FDA. It ensures faster approvals
• Digitized data management can make the commercialized supply chain efficient and economical
• Digitized processes reduce the timelines for clinical trials, registration, and market authorization
• Automation ensures trials can be carried on even during any crisis or pandemic. The systems can operate online and still meet the delivery deadlines

Challenges faced due to the digitization of CGT and biotech manufacturing process

Any change in process is marred with challenges. The same applies to the biotech manufacturing processes.

Sourcing automation tools from various sources

CGT is produced on a small limited scale. To increase production, the companies will need to source more equipment from various vendors. However, for the vendors to supply automated and flexible integrations at low cost is often a hindrance. Hence the CGT therapies manufacturer settles for simple equipment customizations that can be used to enhance or modify a few specific needs. The requirement of personnel with board skill set is still high in this kind of set-up. Sourcing fully automated customized equipment with flexible product options is a challenge.

Limited documentation & traceability in lab-scale automation

CGT is still carried out in lab-setting on small scale. They are unable to provide required documents to meet cGMP guidelines like batch reports and continued process verification (CPV-Auto^TM). So, in such cases, the data is entered manually which poses a data integrity risk and fails to meet CFR21 part 11 compliance.

Operation Systems are time-consuming

When we speak about scaling up the production, the supporting operations systems need to be strong. The operation systems like compliance, support set-up, security, business continuity, lifecycle management are all sourced from different vendors. Hence this often poses an operational burden for the on-site automation engineers.

Retraining & upskilling of Employees

Specific skillset is needed from the human counterpart to ensure smooth automation transition. Due to employee pushback attitude and lack of training, few technological tools are yet to be fully optimized

Way ahead

Digitization holds the key to transforming the entire biotech manufacturing process. The challenge is how to make the transition from small lab R&D to cGMP manufacturing smooth. Companies need to invest in developed automation solutions. This will help to restructure operations, adhere to compliance and enable in development of high-quality marketable products with cost efficacy.

About Aventior

Data restructuring, CPV-Auto^TM, Digital pathology, Data engineering, and Visualization are the services offered by Aventior to various pharma, life science, and biotech companies. They provide robust technical support using AI-backed technology. To know more about their services and the solutions they provide, do write to info@aventior.com.

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Being a part of the biomedical research field, cell and gene therapy targets to treat, cure, as well as prevent genetic and acquired disease. But both these works differently. In Cell therapy, cells are modified or altered outside the body and then injected into the patient’s body. In the case of gene therapy, new genes are introduced, or existing defective genes are replaced or inactivated within the cell. This therapy is done within the body or at times outside of the body. 

Cell and gene therapy are been tested under clinical trials. Scientists are exploring the different aspects of the therapy, its efficiency, and the risk involved. A very small number of such trials have been approved. FDA approved the first cell and gene therapy product, which is by Kymriah (Novartis). The others include Yescarta (Gilead) and Zynteglo (Bluebird). Tecartus has been the latest therapy approved and can be used in the U.S. 

The FDA believes that the gene therapy application will be doubled each year and as per the reports by 2025, the United States will approve 10 to 20 therapies every year. Cell and gene therapy is all set to revolutionize the healthcare system. We have seen large companies investing in the same. 

From the lab to the Masses

These therapies are extremely expensive due to their labor-intensive manufacturing process and it is marred with challenges when it comes to commercialization. For the commercialization of cell and gene therapy, we need to consider regulatory guidelines, manufacturing, patient education, logistics, and more. Hence companies are looking to develop a safe manufacturing process, at the correct scale and affordable cost. In the case of this therapy “one size doesn’t fit all” unlike other therapies and products. They are complex that can be manufactured in various ways that use different vectors and cell lines.

Gene therapy Manufacturing

Gene and cell therapy manufacturing is a crucial aspect when we speak about the commercialization of the therapy. The viral vector capacity of manufacturing is 1 – 2 orders which is much lower than the requirements. The Covid-19 has dealt another blow to manufacturing as viral vectors are used for vaccine development programs as well. We would need to evaluate the current manufacturing process to revolutionize cell and gene therapy and make it commercially viable and available. 

Evaluation of Manufacturing Process

Scalability 

The manufacturing process needs raw materials, cell substrates, and process consumables. The amount of time required to source such material for manufacturing is crucial. As the product progresses through clinical trials and gradually to commercialization, the requirement for treatment increases. Hence forecasting the manufacturing scale is important during the process development stage.

Manufacturing options – in-house or outsourced

The manufacturing process depends on important factors like the cost involved, the skill requirement, and the time needed. Depending upon the above the companies need to decide if they want to manufacture using in-house facilities or outsourced. The capital needed to set up the manufacturing unit is huge. The skilled resources are presently few. Hence depending upon the scalability and to reduce cost, it will be viable to outsource the process.

Sterilized Manufacturing 

The present sterile filtration methods are inadequate for gene therapy. The level of risk is greater for modified gene cell therapies so the pharma companies must check the aseptic control while producing viral vectors. A shift to automated processes to reduce operator handling will reduce the risk of contamination. 

Reconsidering the Regulatory Framework

In the year 2020 FDA had stopped few clinical trials due to the high usage of AAV vectors for treatments. This proved to be a setback for therapy manufacturing. FDA has appointed more reviewers and has amended the regulatory framework, but they expect more data from developers to understand the gene therapy characteristics. Reconsidering the regulatory framework especially for gene therapy will be a major boost for manufacturing.

Logistics 

The therapy needs stringent temperature controls and product security during transportation. Since the treatments are personalized, the timelines are crucial – the collection from patient to the manufacturing location and back to patient needs to be complete in strict timelines. Hence right from the packing to the mode of transport to active shipment, live tracking plays a vital role. The logistics must be planned with an excellent level of expertise for the commercialization of cell and gene therapy manufacturing.

Conclusion 

Cell and gene therapy will soon become a reality and will overcome the manufacturing challenges. The heavy influx of investments, clinical success, swift product approvals, focus on improving resources for the manufacturing process, and supply chain have all fueled to revolutionize cell and gene therapy manufacturing. 

About Aventior 

Data restructuring, CPV-Auto^TM, Digital pathology, Data engineering, and Visualization are the services offered by Aventior to various pharma, life science, and biotech companies. They provide robust technical support using AI-backed technology. To know more about their services and the solutions they provide, do write to info@aventior.com.

Tell Us for more about your requirements here

The post Revolutionizing Cell and Gene Therapy manufacturing first appeared on Aventior.

Finding cure for cancer

Cancer is the most dreaded and relentless disease. Though, as per the latest statistics, the survival chances of cancer patients have increased dramatically over the years, the war is far from over. This disease is linked to genetic defects. The introduction of genes specifically to alter the defective gene has fueled various development to find a cure for cancer. It is speculated that cell and gene therapy (CGT) can be used to cure cancer and also other serious illnesses like cystic fibrosis, heart diseases, hemophilia, diabetics, AIDS to name a few.

 Presently various clinical trials are conducted in the United States. Once it gets approved, cell and gene therapy can be a life-changing treatment option for people afflicted with cancer and other serious illnesses.

More about cell and gene therapy

Cell therapy is where viable cells are implanted or injected into a patient. This helps to increase the medicinal effect. This therapy is used widely in treating a patient afflicted with Cancer. In the 19th century, it experimented with animals, however, further studies and research showed that cell therapy can be used in humans to avoid body rejecting transplanted organs. Now it is used to treat autoimmune disease, urinary problems, repair spinal cord injuries, and improve the weakened immune system.  

Gene therapy focuses on the genetic modification of the cell. The defective genetic cell is repaired as part of the treatment. It aims to fix the genetic problem at its core by modifying the protein of the mutated cell. 

The goal of both the therapy – cell and gene – are the same but they are still vastly different. Cell therapy uses healthy cells to cure the ailment and gene therapy changes the gene structure by duplicating the structure of the healthy cell.

Both the therapies are costly and difficult to implement. Researchers have been working on gene therapy for 40 years. In the year 2017, U.S. Food and Drug Administration had approved the first gene therapy treatment – Kymriah. We do have a long way to go to make cell and gene therapy available for people at large.

Cell and gene therapy soon a reality?

Personalized medicine to cure cancer

After years of research, we have a chance to beat the relentless Cancer. Due to genetics research, we know how the disease develops and affects the patient. Cell and gene therapy uses the body’s own T-cells to beat the infected cancerous cells. The treatment thus received will be personalized to patients to cure cancer. 

Life Science Industry keen on expansion 

 Knowing the potential in cell and gene therapy many companies are entering the markets. With this significant interest shown by big companies, cell and gene therapy may get a major boost. For example, a company like Novartis is all set to expand Kymriah treatment.

Regulatory Support

Regulatory bodies are now open and collaborative to recognize and acknowledge the potential of cell and gene therapy treatments. They are gearing up to set up a standardized system to monitor the treatments. 

Limitations

The stage of advancement and the support it is gathering across the world is encouraging. But the real challenge is to scale it up to its real potential. Since it uses the patient’s cell for treatment – it is very specific to that patient. It becomes difficult to standardize the process. Many more innovative manufacturing technologies will need to be employed to scale it up. The cost-effectiveness needs to be worked out. It would need to be a collaborative effort from all the stakeholders. The side effects of such treatments need to be analyzed. 

Cell and Gene therapy is definitely the best treatment for curing cancer and it has the potential to play a major role in curing other diseases as well. Now how soon the ambition of making it a reality happens that only time will tell.

Any advancement in the field of medical science is made possible with research through data collection, clinical trials, and constant monitoring of how the product or service actually performs. The life Science industry is led by quality management initiatives that are driven by data. Real-World Data and Real-World evidence are the backbones of the healthcare system. The large unstructured data processing and analysis is the key to the success of the Pharma and Life Science industry. Aventior provides the complete technical know-how and support to Life science, Biotech, and Pharma companies to build their complete digital ecosystem. To know more about our services and the solutions we provide, kindly write to us info@aventior.com.

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