New Report Highlights Key Industry Trends in Pharmaceutical R&D
NEW YORK, April 28, 2022, the global business intelligence provider for the biopharma industry, and The American Society of Gene & Cell Therapy. The Q1 2022 report assesses industry trends by examining approved gene, cell and RNA therapies, the pharmaceutical pipeline by company, most targeted therapeutic areas, and an overview of recent dealmaking and start-up funding.
Notable findings from the first quarterly report of the year include:
- FDA Approvals - Since Q4 2021, one new genetically modified cell therapy has been approved and one new gene therapy has filed for approval. Carvykti, a CAR-T cell therapy developed by Legend Biotech and Johnson & Johnson, was approved for multiple myeloma in the U.S. and EtranaDez (etranacogene dezaparvovec), an AAV5 gene therapy developed by uniQure, was filed for approval in the EU and UK for hemophilia B.
- Gene Therapy Pipeline Growth - Since Q1 2021, the gene therapy pipeline has increased by 16% with 3,579 therapies in development, ranging from preclinical through preregistration. A total of 1,986 gene therapies (including genetically modified cell therapies such as CAR T-cell therapies) are in development, accounting for 55% of gene, cell and RNA therapies; and 816 non-genetically modified cell therapies are in development, accounting for 22% of gene, cell and RNA therapies.
- Most Targeted Therapeutic Areas - Oncology and rare diseases continue to be the top areas of gene therapy development in both the overall pipeline (preclinical to preregistration) and in the clinic (Phase I to preregistration). Development for rare diseases most commonly occurs in the oncology space, representing a majority of 52% compared to non-oncology rare disease gene therapy pipeline development.
- Deal Making - A total of 123 deals were signed, a 15% decrease in volume from Q4 2021. Q1 2022 was the lowest quarter total within the last year, and acquisition and alliance volume quarter by quarter remained flat, while financings continue to trend down. Acquisitions were the only type of deal to increase in Q1 2022.
- Start-up Financing - Start-up financing by gene, cell and RNA therapeutic companies remained steady in Q1 2022. A total of 15 companies raised seed or Series A financing, totaling an aggregate $507.8M. The overall deal-making total across alliance, acquisitions and financings saw a 15% decline. In the largest start-up financing, Cellino Biotech, a Harvard University spin-out, raised $80M to support its large-scale production of autologous and allogeneic cell therapies.
"Since we started putting these quarterly reports together in Q1 2021, we have seen the gene therapy pipeline steadily increase. Oncology and rare disease remain the most targeted therapeutic areas, and we continue to see a strong pipeline for CAR-T cell therapies for cancer indications," said Ly Nguyen-Jatkoe, PhD, Executive Director, Americas, Informa Pharma Intelligence. "Rare diseases continue to be a focus for RNA therapies."
"The continued approvals of gene and cell therapies has turned into one of the brightest spots in releasing this report each quarter," said David Barrett, JD, CEO, the American Society of Gene & Cell Therapy (ASGCT). "With the approval of Carvykti in Q1 2022 and the ever-expanding pipeline, we're exceptionally optimistic about the future of the field."
All pipeline and trial insights were compiled using data from Pharma Intelligence's Pharmaprojects, and Trialtrove, part of Citeline, which has been tracking global drug development since 1980. Deal, financing and catalyst data were derived from Biomedtracker. For more information, or to view the current and past reports, visit Informa Pharma Intelligenceor contact pharma@informa.com.
About The American Society of Gene & Cell Therapy (ASGCT)
The American Society of Gene & Cell Therapy (ASGCT) is the primary professional membership organization for scientists, physicians, patient advocates and other professionals with interest in gene and cell therapy.
Our members work in a wide range of settings including universities, hospitals, government agencies, foundations, biotechnology and pharmaceutical companies. ASGCT advances knowledge, awareness and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease to benefit patients and society.
About Informa Pharma Intelligence
Informa Pharma Intelligence powers a full suite of analysis products - Datamonitor Healthcare, Sitetrove, Trialtrove, Pharmaprojects, Biomedtracker, Scrip, Pink Sheet and In Vivo - to deliver the data needed by the pharmaceutical and biomedical industry to make decisions and create real-world opportunities for growth.
With more than 400 analysts keeping their fingers on the pulse of the industry, no key disease, clinical trial, drug approval or R&D project isn't covered through the breadth and depth of data available to customers. For more information, visit pharmaintelligence.informa.com
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Alex Wendland for The American Society of Gene & Cell Therapy
