Gene Therapy, Regenerative Medicine Markets Booming Worldwide

Globally, companies manufacturing gene therapy, cell therapy, and other regenerative medicines raised nearly $10 billion in 2019, the second-highest year on record, according to a recent analysis from the Alliance for Regenerative Medicine (ARM).

ARM released its 2019 Annual Report and Sector Year in Review last week, highlighting the organization’s key priorities and initiatives, as well as offering a deeper look into trends and metrics for cell therapy, gene therapy, and tissue engineering sectors.

Using data sourced from ARM’s data partner, Informa, researchers uncovered that venture financing made up more than $4 billion in global financings, which is a 33 percent increase over 2018.

Gene and gene-modified cell therapy raised $7.6 billion, while cell therapy raised $5.1 billion and tissue engineering raised $441 million in 2019. 

“What’s been so impressive is the uptake of these new therapies, given all of these pressures and questions about access,” said Matthew Kane, CEO & co-founder of Precision Bioscience. “Why is that? It’s the response rates you’re seeing for with late-stage cancers that have progressed despite trying all the other therapies. That’s what makes this field exciting. I think that’s why we’re all here today and why we’re so optimistic.”

“Going into 2020, we have a new set of questions to start to answer and unravel in terms of how we can make these products more accessible,” he continued. 

Additionally, the report found that companies headquartered in Europe saw the best year on record, raising $3 billion. These companies were sponsoring 260 trials by the end of 2019 and more than ten candidates were assured clinical trial approval. 

There were 1,066 clinical trials underway worldwide by the end of 2019, including 352 gene therapies, 452 gene-modified and cell-based IO, 216 cell therapy, and 46 tissue engineering. Researchers expect the number of approved gene therapies to double in the next one to two years.

Important takeaways from the report also included total financings for the sector, partnerships and other deals, clinical trial information, anticipated near-term product approvals and regulatory filings, and expert commentary from industry representatives in the US and Europe. 

Researchers highlighted the discovery of a new form of gene editing called prime editing, which could expand the number of diseases addressed worldwide. Two patients have received the CRISPR gene editing so far and have benefited from the treatment. 

Just last week, Oregon Health & Science University (OHSU) held (LINK) the first-ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10. 

BRILLIANCE is one of 14 different clinical trials looking at new genetic treatments for ophthalmic conditions. The procedure uses CRISPR, which has never been previously used. 

“Being able to edit genes inside the human body is incredibly profound,” said Mark Pennesi, MD, PhD, the Kenneth C. Swan associate professor of ophthalmology at the OHSU School of Medicine. “Beyond potentially offering treatment for a previously untreatable form of blindness, in vivo gene editing could also enable treatments for a much wider range of diseases.” 

The clinical trial was a success for one patient, which is promising as gene therapy becomes increasingly more popular throughout the US. 

ARM voiced that they will continue to update information through additional reports at the close of each quarter, tracking sector performance, key financial information, clinical trial numbers, and clinical data events. 

“We need to try to figure out how to enable greater access to cell and gene therapies into other indications,” said Tim Lu, co-founder and CEO of Senti Bio, an ARM member. “How do we go beyond making single changes, single modifications? I think it’s pretty clear now from the basic research that it’s possible. The design cycle for modifying and making these sort of therapies is only going to accelerate over the next decade and it’s a matter of how do we then take that matter, match that with the right indications, and really drive those into the clinic.”