100 top cell and gene therapy companies to watch in 2023

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The cell and gene therapy sector is poised to deliver a wave of new therapies that could potentially cure rare and common diseases. As many as 13 new cell or gene therapies could be approved for use in the U.S., Europe, or both by the end of 2023.

While manufacturing and regulatory challenges remain, the cell and gene therapy industry continues to offer significant therapeutic potential and commercial opportunities.

This year, we have beefed up our list of leading cell and gene therapy companies, doubling the number of organizations from 50 to 100.

In selecting the companies for this list, we weighed public companies’ degree of involvement in cell and gene therapy, including drug developers, equipment vendors and CDMOs specializing in the area. For private companies, we factored in their funding levels, pipeline and degree of history for the list.


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Genascence believes gene therapy can transform the treatment of knee osteoarthritis

Osteoarthritis (OA), a degenerative joint disease affecting the knee and other joints, affects hundreds of millions of people worldwide.

Genascence (Palo Alto, California) is developing a gene therapy known as GNSC-001 for OA of the knee. A potent inhibitor of interleukin-1 (IL-1) signaling, GNSC-001 is a recombinant adeno-associated virus vector with a coding sequence for interleukin-1 receptor antagonist (IL-1Ra).

The startup believes GNSC-001 could offer durable inhibition of IL-1 after a single injection.

“Gene therapy has been a big passion of mine,” Genascence CEO Tom Chalberg said. “And osteoarthritis is one of the few areas of medicine that hasn’t enjoyed any benefit of the biologics revolution.”

Tom Chalberg

Traditional knee OA treatment includes non-surgical strategies such as weight management, physical therapy and medications to reduce pain and i…

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Genanscence raises $10.5 million in Series A financing

The clinical-stage biotech Genascence has closed a Series A financing led by Pacira BioSciences, generating $10.5 million.

The funding round included investors Polymerase Capital, DeepWork Capital and University of Florida Research Foundation.

The Palo Alto, California–based company is focused on treating musculoskeletal diseases with gene therapy.

The company plans on using the funding for continued development of GNSC-001, an adeno-associated vector with a coding sequence for interleukin-1 receptor antagonist (IL-1Ra), which inhibits interleukin-1 (IL-1) signaling.

GNSC-001 is Genascence’s lead program in osteoarthritis. Inhibiting IL-1 is an attractive target in osteoarthritis because IL-1 is involved in cartilage degeneration and inflammation.

Osteoarthritis affects approximately 13% of adults according to data from CDC.

“Osteoarthritis affects more than 30 million Americans and is the leading cause of disability and chronic pain…

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