July 9, 2020

The Niche

Knoepfler lab stem cell blog

Regenerative medicine recommended reads including $191 million to researcher

It’s notable how the FDA now considers gene-editing a kind of regenerative medicine. This means that various gene therapy products in development technically qualify as regenerative therapies.

Ever since the agency began its rapidly growing regenerative medicine advanced therapy (RMAT) designation program, we’ve seen an increasing number of gene therapy biotechs and products qualify as RMATs. As a result, the RMAT-qualified gene therapies get to go into a speedier lane of FDA oversight.

Today’s post is a mix of recommended reads in the regenerative medicine space, including on stem cells and gene editing such as CRISPR.

Biotech Forty Seven, which has a unique approach to immunotherapy for cancer, is going to be purchased by Gilead for $4.9 billion. This is a huge deal on a number of levels including for cancer patients.

A huge beneficiary of the deal is pioneering stem cell biologist Irv Weissman of Stanford, whom the LA Times reports could receive $191 million as a result.

Holy crap.

This is also good news for the California stem cell agency, CIRM, which helped Forty Seven with substantial funding. CIRM had a celebratory blog post about this and you can read some more over at California Stem Cell Report. However, what I don’t know yet and haven’t heard back yet from CIRM about, is how much CIRM and/or the State of California will benefit financially. I hope CIRM gets hundreds of millions given their big investment in the company.

Until we have that info it’s hard to say just how much this helps CIRM concretely, but it surely helps the campaign for another round of CIRM funding from us California voters this fall.

IPS cell review pub on disease modeling and drug discovery. Ever since the first few years of the IPS cell field’s history went by, a fun debate and topic of discussion has been whether IPS cells will have more impact directly via producing cellular therapies or if IPS cells would have more impact from disease modeling, drug discovery, and other indirect uses. What do you think? This new review pub from Cell Stem Cell digs into the latter kind of therapeutic impact. Multi-lineage Human iPSC-Derived Platforms for Disease Modeling and Drug Discovery.

The Alliance for Regenerative Medicine (ARM) released its annual report for 2019 and it’s full of useful and encouraging info. Key take-homes:alliance for regenerative medicine (ARM)

  •  “Globally, companies active in gene and cell therapies and other regenerative medicines raised nearly $10 billion in 2019, the second highest year on record. Venture financings were particularly strong, making up more than $4 billion in global financings – a 33% increase over 2018.
  •  There were 1,066 clinical trials underway worldwide by year-end 2019. 10+ product candidates are poised for approval, and the number of approved gene therapies will likely double in the next one to two years.
  •  Companies headquartered in Europe raised $3 billion, the strongest year on record, and were sponsoring 260 trials by the end of the year.
  •  There is a supportive policy environment for regenerative medicines, with policymakers showing a strong interest in promoting the development of, and patient access to, these innovative therapies.”

    Mark Pennesi, CRISPR regenerative medicine eye
    Mark Pennesi, using CRISPR for regenerative medicine in the eye.

I’m continuing to keep a tally of stem cell trials for COVID-19, the disease caused by the novel coronavirus. My impression so far is that these trials are unlikely to be helpful, but we’ll see. They are definitely being hyped, sometimes as a cure.

CRISPR treatment inserted directly into the body for first time. From a Nature News piece on this by Heidi Ledford, we have the story of using CRISPR In this new way to treat a hereditary blindness disorder called Leber’s congenital amaurosis 10 (LCA10). From Ledford’s piece:

“For the latest trial, the components of the gene-editing system – encoded in the genome of a virus — are injected directly into the eye, near photoreceptor cells. By contrast, previous CRISPR–Cas9 clinical trials have used the technique to edit the genomes of cells that have been removed from the body. The material is then infused back into the patient.

“It’s an exciting time,” says Mark Pennesi, a specialist in inherited retinal diseases at Oregon Health & Science University in Portland. Pennesi is collaborating with the pharmaceutical companies Editas Medicine of Cambridge, Massachusetts, and Allergan of Dublin to conduct the trial, which has been named BRILLIANCE.”

 

%d bloggers like this: