Cell and gene therapies are widely considered to be the future of treating several, often life-threatening conditions, and are hence a growing area for pharma and biotech companies alike. In this paper we take a look at the landscape for cell and gene therapies, highlighting some of the key assets in the field. There are currently a handful of approved treatments on the market, but several hundred in the development pipeline.
Cell and gene therapies are some of the most revolutionary advances in treatment in recent years, potentially leading to the next paradigm shift in therapies. Although initial drug approvals have been for relatively small patient groups and rare diseases, the significant pipeline of cell and gene therapy studies currently underway will significantly expand the impact of these treatments, and unleash their genuine, unprecedented potential.
Cell and gene therapies are a rapidly growing area within the biopharmaceutical landscape. The global cell and gene therapy market generated ~$2.3 billion in 2020 and is predicted to grow at a CAGR rate of 64.0% during the forecast period 2020-2026. Growth is driven by a number of factors including increasing investment into next-generation therapies, experimental therapies moving through clinical trials as well as from regulatory bodies such as the FDA to support approvals and aid transition of therapies to commercial stage products. We have highlighted some of the key future themes expected in this paper.
A large proportion of the therapies in clinical trials are being developed by small to mid-size biotech’s, providing plenty of opportunities in this space for M&A, product licensing deals and investment. As the technology matures, large pharma companies are expected to shift focus towards owning the technology versus partnering, as demonstrated by recent large acquisitions. The majority of assets under trials target cancers, sensory organs and blood disease as well as rare, genetic conditions across multiple therapy areas. A high number of trials are currently at Phase II, where safety and efficacy is assessed.
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This paper also includes a section on the regulatory pathway for the development of cell and gene therapies, written by Scendea, a leading product development and regulatory consulting practice serving the pharmaceutical and biotechnology industry.
We have worked with businesses at every stage of the business life-cycle and understand the challenges and opportunities they face. We also have a particular interest in next-generation therapies, backed up by experience and knowledge in the space within the team. We have drawn on publicly available sources, as well as conversations with industry leaders and key opinion leaders, to produce this report.
If you would like to discuss anything you have read please contact Gurpal Ahluwalia or Anand Ashok.