Global Cell & Gene Therapy Sector on Pace for Annual Records in Product Approvals and Financings, Says Alliance for Regenerative Medicine

GLOBAL CELL & GENE THERAPY SECTOR ON PACE FOR ANNUAL RECORDS IN PRODUCT APPROVALS AND FINANCINGS, SAYS ALLIANCE FOR REGENERATIVE MEDICINE

ARM’s H1 2021 Report Chronicles ‘A Year of Firsts’ For The Advanced Therapies Sector

BRUSSELS, Belgium – August 18, 2021
The regenerative medicine and advanced therapies sector completed its best-ever six-month period of financing, raising $14.1B from January through June and reaching 71% of the record amount raised in all of 2020, according to the Alliance for Regenerative Medicine’s (ARM) H1 2021 Report released today.

Four new Advanced Therapy Medicinal Products (ATMPs) – cell, gene, and tissue-based therapies – have already received regulatory approval in 2021. With decisions still expected on six others, the number of total approvals could surpass the record of nine set in 2016.

Gene therapies/gene-modified cell therapies are especially positioned for a record-breaking year. Three of the four new products approved so far in 2021 and four of the six remaining regulatory decisions this year are in this category, making it almost certain it will exceed the record of three approvals set in 2017.

ARM’s report chronicles a year of “firsts” so far for the advanced therapies sector. In scientific advancements, Intellia Therapeutics reported promising initial data from the first-ever use of CRISPR gene-editing directly in the human body, to treat ATTR amyloidosis. On the regulatory front, the FDA approved Bristol Myers Squibb’s/bluebird bio’s Abecma, the first CAR-T therapy to treat multiple myeloma and to target BCMA; China approved its first CAR-T therapy, Kite’s Yescarta. And in financing, 20 companies have issued initial public offerings (IPOs) so far this year, exceeding the record number of IPOs raised in all of 2020.

“The regenerative medicine sector is accelerating across all fronts simultaneously,” said Janet Lambert, CEO of ARM. “Scientific progress is driving intense interest among investors. Companies are forming, maturing, and increasingly moving promising therapies toward approval. And we’re seeing rising global competition to cultivate this sector and deliver access for patients.”

Highlights from ARM’s H1 report and data include:

• Financing for cell-based immuno-oncology ($6.6B) for the first time surpassed funding for gene therapy ($6.4B); public equity performance for cell-based IO companies has exceeded that of gene therapy companies since mid-2020
• There are 1,195 regenerative medicine and advanced therapy developers active worldwide, up from 1,085 at the end of 2020
• There are 1,320 industry-sponsored clinical trials ongoing worldwide with 158 trials in Phase 3, up from 1,220 and 152 at the end of 2020; there are an additional 1,328 trials sponsored by government and academia, with 85 in Phase 3
• There are 558 industry-sponsored trials with sites in the US and 238 in Europe. Since 2018, more than twice the number of trials have started in the US and Asia-Pacific than in Europe
• The number of developers in Europe since 2018 has declined by 4%, while increasing by 23% in the US and 119% in Asia-Pacific

As the global voice of the sector, ARM surpassed 400 member organizations for the first time. Thirteen of these member organizations, and ARM itself, welcomed 17 interns to the inaugural class of the GROW RegenMed Internship Program, created to address the underrepresentation of Black employees in the regenerative medicine workforce. ARM expects the program to double in size in its second year in 2022.

ARM continues to advocate for patient access through policymaker engagement at the US federal and state levels, and in Brussels and key European Union Member States. ARM’s advocacy achieved an important outcome for patients and the sector in Germany by reducing a bureaucratic reimbursement hurdle for hospitals that provide advanced therapies. In the US, ARM is working with congressional sponsors on ‘Cures 2.0’ legislation and advocated for increased funding for FDA’s Center for Biologics Evaluation and Research (CBER) through congressional appropriations and the PDUFA process.

Media inquiries
For more information or for media requests, please contact Stephen Majors, Director of Public Affairs for ARM, at smajors@alliancerm.org.

About the Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organisation dedicated to realizing the promise of advanced therapy medicinal products (ATMPs). ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives in Europe and internationally to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing ATMPs. In its 12-year history, ARM has become the global voice of the sector, representing the interests of 400+ members worldwide and 80+ members across 15 European countries, including small and large companies, academic research institutions, major medical centres and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.