Big Pharma partnerships, record $22.7B investment raise profile of regenerative medicine in 2021

Regenerative medicine couldn't stop making news in 2021. The year was marked by Intellia's first results in humans for gene editing, massive Big Pharma partnerships and record investment of $22.7 billion.

This past year saw six new cell, gene and tissue-based therapies approved, according to an annual report from the Alliance for Regenerative Medicine (ARM). Meanwhile, CAR-T therapies put up the data to justify moving up a line in cancer treatment, and venture capital went all-in on the field. 

Some major milestones for the field are yet to come in 2022, ARM said. About nine therapies are awaiting FDA nods, and another seven are before EU regulators. They include Gamida Cell’s cell therapy omidubicel and BioMarin’s ValRox for hemophilia A. For 2023, two sickle cell disease treatments, bluebird bio’s gene therapy bb1111 and CRISPR Tx and Vertex Pharmaceuticals’ CTX001, will be considered in the U.S.

There was much debate over delivery mechanisms for gene therapies, which ARM said weighed down the field in 2021. But the group, an advocacy organization with more than 425 members, is optimistic that the field is making progress in finding new ways to optimize the leading mechanism, adeno-associated viruses. ARM also noted that the FDA has indicated it will not impose a universal dose cap on AAV-based gene therapies.

AAVs were used in 145, or 46% of all gene therapy clinical trials. Other mechanisms included lentivirus, which had 24 clinical trials, or 8%, and adenovirus, with 22, or 7% of trials.

Gene editing had 41 trials underway this past year, with one-third in phase 1 and the rest in phase 2. About 80% of these use CRISPR technology. That includes Intellia’s transthyretin amyloidosis treatment that had a much buzzed about readout in summer 2021. The company rolled out some more data from the study in February that showed a dose-dependent response and began to answer the durability question associated with gene editing.

More than half of all gene editing trials are in cancer. 2021 also saw the first gene editing therapy in a more common disease, when CRISPR Tx and ViaCyte launched a study in Type 1 diabetes.

As for investment, gene therapy and cell-based companies nabbed the majority of funds at $10.2 billion and $10.1 billion, respectively. Cell therapy followed with $2 billion and tissue engineering biotechs garnered $341 million.

The majority of the cash came from venture capital, which contributed $9.8 billion—a 75% increase over the year before. IPOs helped companies raise $4.8 billion, an increase of 30%.

There’s been a rush of Big Pharma partnerships across biotech, and these deals helped raise $2.3 billion through upfront payments in 2021, a 23% increase year over year. ARM noted the $900 million upfront payment Vertex offered to CRISPR Tx to work on CTX001 and AbbVie’s $370 million check for REGENXBIO for an eye disease gene therapy.

Cancer is easily the busiest indication for regenerative medicine, with a whopping 1,246 trials underway right now across oncology, or 52% of all studies in the field globally.

The sector has long been working on hematological cancers, which saw 685 trials, or 55%. But ARM noted a shift over the past year toward solid tumors, which climbed to 561, or 45% of trials. Gastrointestinal cancers were the leading indication here, with 130 trials, or 23% of all solid tumor trials underway, followed by brain and spinal cord cancers with 71 or 13%.

CAR-T therapies also moved up a treatment line this past year, with both Gilead Sciences and Bristol Myers Squibb reporting data showing their therapies performed well against second-line standard of care in relapsed or refractory large B cell lymphoma.