White Paper

Cell And Gene Therapy Field Quiet In Q2

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The Q2 2024 landscape report highlights ongoing growth in the advanced therapy sector despite encountering some challenges. Notably, two new RNA therapies were approved in the US: Rytelo for myelodysplastic syndrome and mRESVIA for RSV prophylaxis. However, no new gene or cell therapies received approval this quarter. The development pipeline remains strong, with 4,047 therapies in progress across various modalities. Gene therapies, including genetically modified cell therapies, constitute the largest portion at 51%, followed by RNA therapies at 27% and non-genetically modified cell therapies at 22%.

Oncology and rare diseases continue to dominate as the primary focus areas, especially in gene and cell therapy development. For RNA therapies, rare diseases are the top target, with anti-infective indications coming in second. The report also notes some headwinds in deal-making and financing activities, with a 20% drop in total deals from Q1, largely due to a 39% decrease in financing transactions. While early-stage financing volume declined, the total amount raised saw a slight increase to $266.3 million across six deals.

In summary, despite near-term challenges, particularly in financing, the advanced therapy sector remains robust, with a strong and diverse clinical pipeline and continued progress in the RNA space.

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