Cell and gene therapies (CGTs) are coming of age, with most healthcare leaders expecting them to be ready for widespread delivery within the next 3 to 10 years. They can be considered the new “magic bullet”, which is reminiscent of monoclonal antibodies (mAbs) some 20 to 25 years ago. At the time, mAbs represented a breakthrough: large proteins with capacity to target specific antigens in a world of small molecules. The mAbs brought with them much hope and significant activity amongst biotech and pharma companies worldwide, in terms of mergers and acquisitions (M&As), collaborative deals, and licensing opportunities. They have since delivered (and are still delivering) great progress in the treatment armamentarium of autoimmune diseases, cancers, and more.
But what does the deal landscape look like today for CGTs as these technologies begin to reach their potential? Will the same trends in licensing or acquisitions manifest in the CGT space as they did for mAbs? Or, given the lack of commercial benchmarks and uncertainties in long-term valuation for these therapies, can we expect new trends to emerge? To help answer these questions, we have looked at recent M&A and collaborative activity in the space, with an eye toward size, pace and focus of companies involved, deal structure and value, and the number and clinical stage of assets, to draw similarities and parallels between mAbs and CGTs.
Evidence requirements for orphan drugs
CRA’s Life Sciences Practice focuses on global policy, commercialization, and market access issues dedicated to orphan drug issues, including orphan evidence...