When the U.S. Food and Drug Administration issued new guidance earlier this month on how companies who handle human cells, tissues and cellular and tissue-based products should report problems to the agency, it was a recognition that the medical industry is heading deeper into uncharted territory.

But FDA Commissioner Scott Gottlieb recognized that it was still early-going, calling the policy "another step forward in our development of a comprehensive framework for regulating cell and tissue products – one that provides clear rules and balances patient protection with the need for efficient oversight of these technologies."

[Also: Genome editing tools set to bring monumental change to healthcare]

As research into novel treatments for cancer and rare disease continues, regenerative medicine is becoming big business, with companies focused on cellular products having raised some $25 billion in financing, according to a recent study from Informa Pharma Intelligence.

"Previously regenerative medicine focused on devices, but now we’re seeing a pipeline shift centered on therapies," said Amanda Micklus, principal analyst of Informa Pharma Intelligence. "This is an exciting time for the field, with new innovations spanning diseases and patients that were previously underserved by currently available treatments. Considering the large size of the pipeline we’re going to see several new discoveries and advances in the space over the next few years.”

[Also: In a precision medicine landmark, FDA approves first gene therapy]

That pipeline shift has seen an uptick in large federal and private sector investment since President Barack Obama signed the 21st Century Cures Act, which created a special status pathway for regenerative medicines through the FDA’s regenerative medicine advanced therapies designation.

There were more than 100 regenerative medicine mergers and acquisitions announced since 2016, with at a total value of $68 billion, according to Informa Pharma Intelligence, with nearly half of such deals involved smaller biotech companies partnering with each other to advance their respective technologies

"Growth in regenerative medicine is being driven by a friendly federal government and enormous market interest, especially from Johnson & Johnson, Celgene and AstraZeneca, the most active large pharma players in the space in terms of number of partnerships," said Micklus. "We’re going to continue seeing major breakthroughs in this space, that can help people where no other help is currently available."

Still controversial, but gene editing is making advancements

Another report from Informa Pharma Intelligence examines the expansive new frontier of gene editing, which is fast evolving thanks to growth in the CRISPR-Cas9 tools. The study spotlights the fact that more than 100 inventors have been granted patents so far.

The technology -- the two halves of the acronym stand for "clustered regularly interspaced short palindromic repeats" and "CRISPR associated protein 9" -- represents just the latest (but so far most hugely promising) generation of gene editing technologies, which center around the use of an RNA molecule to guide the Cas9 endonuclease to a target area editing.

[Also: Start-up working on breakthrough in 3-D printing of human organs]

Scientists such as Yoshizumi Ishino at Osaka University, Emmanuelle Charpentier at University of Vienna, Jennifer Doudna of the University of California and Feng Zhang of Harvard and MIT's Broad Institute have been instrumental in the field's research, and "intellectual property around the foundational US patents in CRISPR/Cas9 has become a contentious issue in the gene editing field," according to Informa Pharma Intelligence.

There are other controversies and hurdles that need to be ironed out for advancements in gene editing to truly take hold, according to the report. For instance, the practice is currently limited to ex vivo applications, but many of the human diseases that could be treated or prevented would need to be targeted in vivo. Unwanted (and often unpredictable) immune reactions and off-target mutations are big concern. And several groups have issued policy statements expressing concern with the ethics of gene editing and recommending caution.

That said, "many companies, such as Bluebird Bio and Homology Medicines, are improving on existing gene-editing technologies to produce more efficient systems," said Informa Pharma Intelligence researchers, and the existing field of gene editing companies is "advancing their assets through several key deals. Several large pharmaceutical companies have become quite active, including Novartis, which has signed agreements with Caribou Biosciences, Intellia Therapeutics, and Celyad."

Nonetheless, "gene editing for the treatment of human disease is still in early stages and represents a very small proportion, just 3 percent of the overall regenerative medicine landscape in comparison to more active treatment modalities within regenerative medicine," according to the report.

Across the gene editing landscape, the most active companies span discovery-stage biotechs to large pharma firms. Most are using CRISPR-Cas9, including Editas Medicine and CRISPR Therapeutics, "which each have ten candidates in development (either as an originator or licensee)," according to the report. Big names such as Shire, Pfizer and Bayer have also staked their claims to the gene editing field.

Twitter: @MikeMiliardHITN
Email the writer: mike.miliard@himssmedia.com