Gene Therapy Trials

Gene Therapy Trials Global Market Outlook:

• The global gene and cell therapy clinical trials market is estimated at $12.9 billions in 2023, and it is expected to grow at a CAGR of 22-26 percent from 2021 to 2027

• Compared to 2018, an increase of 20 percent was observed for clinical development of cell and gene therapies. FDA is proactively encouraging innovative product development and clinical trial design for gene therapies, and they are predicting that they might approve 10 or more products per year by 2025

• CAR T-cell therapy is growing, due to its personalized nature of the therapy and adoption of advanced technology

• China is the focal point of gene therapy trials, and Japan is also coming at par with China. Hence, the APAC region is growing significantly

Gene Therapy Trials Market Overview

The global gene and cell therapy clinical trials market is estimated at $12.9 billions in 2023, and it is expected to grow at a CAGR of 22–26 percent. By 2025, FDA predicts they may approve 10–20 new cell and gene therapy products per year. Among the CGT market, the gene therapy market accounts for around 72 percent of the overall market

• The gene therapy market is growing and collaborations among biotech players have increased, due to increase in number of approvals for gene therapy clinical trials. These therapies are also being accepted largely by physicians as well as patients. Between 2020−2021, around 201 deals were finalized among biopharma companies concerning cell and gene therapy development

• Oncology and monogenic diseases contribute majorly to the overall market share. Viral vector is more preferred over non-viral vectors in gene therapies, because of the various benefits, like high efficiency of gene transduction, specific gene targeting and delivery, safety

• Even though the market is growing, a few hiccups in the gene therapy market are safety and efficacy related, prolonged lab procedures for trial studies, lack of knowledge of product interaction with the host, and the most importantly, the high cost of gene therapy products–mainly due to complexity of manufacturing of these advanced therapies and regulatory approval process

• The UK is establishing itself as a hub for cell and gene therapies and contributes to almost 12 percent of cell and gene therapies, its Y-o-Y no. of trials have also doubled. The studies conducted are majorly backed up by international companies, leading them to reach recruitment phase at a faster rate

Gene Therapy Trials : Supply Dynamics

2023 will mark the beginning of design era. Using new technology, biopharma will be able to construct and create smarter molecules that will enable them to control the novel biology that was found in the previous decade.

• About 55 percent of the gene therapy pipeline consists of medicines that are delivered in vivo. Ex vivo gene therapy, which involves genetic modification performed to cells outside the body comprises of 45 percent of the pipeline and is mainly dominated by use of autologous, patient-specific cells, which are re-introduced into the body post genetic engineering

• There is also an increasing focus toward allogenic cell therapy, which uses genetically modified donated cells “off-the-shelf”

• Oncology and rare diseases are the major therapeutic area of focus, where gene therapies are being developed. 43 percent of the rare disease gene therapy is further rare oncologic diseases. There is relatively lower activity of gene therapy in other therapeutic areas. Neurology segment contributes approximately 78 percent in terms of gene therapy market, and this is mainly due to the usage of spinal muscle atrophy therapies

Cost Drivers and Cost Structure : Gene Therapy Trials

• FTE rates (staff) contribute to 40 percent of the cost and can be negotiated on the basis of the engagement and long term contracts. Site selection can be managed by increasing partnership with academia, hospital networks or by considering FACT institutions.

Insights On Gene Therapy CRO Landscape

Service landscape for gene therapy in rare disease is a fragmented market, wherein the top CRO and niche CRO hold an average experience of conduction on 30
studies
• Supplier selection can be done on case-by-case basis with regards to gene therapy modalities and global trial landscape
• Sponsors prefer engagement with mid-sized CRO, with a niche therapeutic focus given their lean management structure and flexibility
• Long-term collaborations are currently forming the basis of gene therapy engagement

Case Study: A mid-sized pharma company wanted to launch a vaccine using adenovirus vectors. During supplier selection, they took into consideration the platform
expertise, in terms of the kind of partners they had for adenovirus vaccine delivery. Finally, two CROs (one from USA and other vendor from Europe) were selected
on a regional basis and engaged on a project-based model.