Cell and Gene Therapies Poised to Disrupt Health Care Status Quo with Wave of New Treatments

Washington, DC – March 31, 2022

Regenerative medicine takes aim at cancer and a wide range of other prevalent and rare diseases

The Alliance for Regenerative Medicine, the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies, today released its 2021 Annual Report, showcasing a year of significant advances and forecasting continued growth ahead.

The 2021 Report presents important milestones for the sector over the past year, including the approval of six new gene, cell, and tissue-based therapies, record sector investment, and promising early-stage clinical readouts. Highlights include the release of new clinical data supporting the use of gene editing directly in the body, powerful early evidence that cell and gene therapies can treat complex diseases with multiple genetic causes, and results that suggest these therapies may be able to reverse biological damage that has already occurred.

“The exciting advancements of 2021 demonstrate that regenerative medicine is here – and it’s not slowing down,” said Janet Lambert, CEO of ARM. “Regulatory decisions on ten new therapies are expected this year, five of which are gene therapies for rare diseases – making 2022 perhaps the most impactful year ever for gene therapy. More and more patients stand to benefit from the promise of these transformative treatments. But there is significant work ahead of us to ensure that health care systems are ready.”

There are 2,406 ongoing trials globally, including 222 in Phase 3. Of these, 1,171 are industry-sponsored trials and 1,235 are trials sponsored by academic and government institutions.

Cancer is the sector’s biggest target

The 2021 Annual Report shows that cancer, in both rare and prevalent forms, remains the largest target for regenerative medicine, representing 52% of trials globally. Cell-based immuno-oncology (cell-based IO) trials, a category that includes CAR-T therapies, make up the largest proportion of industry trials (41%), surpassing the number of cell therapy trials for the first time. The number of cell-based IO trials is likely to continue to grow, as more than half (57%) of phase 1 industry trials utilize this approach, compared to only about a quarter (22%) of phase 3 trials.

Solid tumors now make up nearly half (45%) of the cell-based IO therapies in the pipeline, with gastrointestinal cancer as the most common target, representing 23% of solid tumor trials.

Diseases of the Central Nervous System follow cancer as the second-largest target (6%) for regenerative medicines globally. The most targeted diseases in this category are Parkinson’s (21 ongoing trials), ALS (15), MS (12) and Alzheimer’s (10).

The pipeline is advancing for both prevalent and rare diseases

Today, nearly 60% of global regenerative medicine trials target prevalent conditions.

And the nature of prevalent diseases being targeted is evolving. Musculoskeletal disorders such as bone fractures, osteoarthritis, and sports injuries make up 23% of phase 3 trials targeting a prevalent disease, but only 7% of phase 1 trials. Other prevalent disease areas targeted include diabetes and related complications (54 ongoing trials) and central nervous system diseases such as Parkinson’s (21). With phase 3 programs in indications including critical limb ischemia and congestive heart failure, the first approval of a gene therapy for a prevalent disease could be just a few years out.

Rare diseases make up 41% of the pipeline globally. Sickle cell disease is the top rare disease target with 39 ongoing trials. Other top indications for rare disease trials include Hemophilia (28), Retinitis Pigmentosa (26), ALS and Thalassemia (15 each). ALS is the only complex, polygenic disease in the top five.

Gene editing continues its rise

Gene editing continues to be an exciting frontier for the sector, with 41 gene editing trials ongoing globally, 14 of which are in phase 1 and 27 of which are in phase 2. 80% of these trials use CRISPR gene-editing technology, which first entered the clinic in 2019. More than half (23) of the 41 trials target cancer; 20 are hematological malignancies, and 3 are solid tumor. The first CRISPR therapy (and first gene editing therapy overall) could be approved as early as 2023, for sickle-cell disease.

Making regenerative medicine accessible is key to serving patients

“The 2019 FDA prediction that it would approve 10-20 cell and gene therapies per year by 2025 is within reach,” said Lambert.  “With the promise of regenerative medicine rapidly becoming a reality for patients, it’s critical that we continue to work diligently to ensure that these therapies are accessible to as many patients as possible.”

Healthcare systems are accustomed to paying for conventional chronic care that treats symptoms and leads to the accrual of costs over long periods of time. Cell and gene therapies address the root causes of disease in a single dose, or a limited number of treatments, resulting in front-loaded costs that create accessibility barriers.  Paving the way for innovative payment models that recognize the unique value of these therapies is a priority for the regenerative medicine sector, and ARM will continue to advocate for removing barriers to such arrangements.

About Alliance for Regenerative Medicine (ARM)

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives 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 regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 425+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Media Contact

Stephen Majors, Alliance for Regenerative Medicine, smajors@alliancerm.org

Caitlin Hodes, Finsbury Glover Hering, caitlin.hodes@fgh.com