Cell and Gene Therapy Snapshot

Cell and gene therapies are the game changers everyone is looking out for.

Dr. Aviral Maheshwari, Vice President; Dr. Anuj Gupta, Director

Next Wave: Cell and Gene Therapies (CGTs)

Perceived as next-generation curative alternatives for multiple rare diseases and cancers, CGTs are estimated to grow at a CAGR of 33.6% between 2020 and 2029. Players in the CGT market are looking to ride the tide of M&A activity in this space, which is expected to increase exponentially over the next 2-3 years. While there are multiple assets in development, there are many challenges (price, durability and logistics) that will prevent CGTs from becoming a preferred treatment option in a therapeutic area. For example, high cost to patients and related infrastructure needs have restricted their access, and there is a pressing need for healthcare systems and reimbursement models to evolve to accommodate these upcoming game changers.

The Potential of Curative Alternatives Driving Growth in Gene Therapies (GTs)

Most therapeutic candidates target rare diseases and cancers, which are difficult to treat and do not have a curative therapy.

CGTs Can Become SOC for Many Diseases in the Near Future

Global pharmaceutical companies are actively looking to acquire in-development CGTs to fuel their growth and leadership across rare diseases and cancers.

Investors’ confidence in CGTs is consistently growing, with an exponential rise in the number of deals and overall value of deals seen in 2019.

“We should think of cellular therapies as an emerging modality rather in a way that antibodies were 20 or 30 years ago. And they take time to optimize, but I can absolutely assure you that there are next-generation candidates coming forward.”

– Rupert Vessey, R&D President, Celgene

BMS CEO, Giovanni Caforio, emphasized the potential for the company to take a leadership position in cellular therapies, and thereby become a market leader in oncology, cardiology, immunology and inflammation.

CGT Developers are Leveraging Viral Vectors

71% of the GTs in development leverage established viral vectors, such as adeno-associated virus (AAV), adenovirus and lentivirus, to ensure better efficacy and safety.

More Than 85% of Therapeutic Leads are in the Early Development Stages

While CGTs currently target chronic, difficult-to-treat indications, the roadmap is being set to develop them as personalized therapies for common indications in the future.

Infrastructure and Logistics Driving Cost to Patients

The cost of therapy and related logistics are going to challenge the transition and adoption of CGTs as personalized therapies for common indications.


Although CGTs are positioned as game changers for rare diseases and cancers, they are far from becoming accessible and affordable therapeutic solutions. CGT developers need to work their way around all involved stakeholders, such as regulators, payers, physicians and patients. They must develop innovative and unconventional strategies in order to ensure CGTs become accessible to the last mile. 

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