Around the Helix: Cell and Gene Therapy Company Updates – July 19, 2023

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive™’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. Companion Diagnostic Immunoassay for Roctavian Receives FDA Approval

The FDA has approved ARUP Laboratories’ AAV5 DetectCDx, a companion diagnostic immunoassay intended to detect the presence of antibodies that bind to the adeno-associated virus serotype 5 (AAV5), for the purpose of determining the eligibility of patients with severe hemophilia A who are considering treatment with BioMarin’s gene therapy valoctocogene roxaparvovec (val-rox; Roctavian). In the United States, ARUP Laboratories will provide all laboratory testing services for AAV5 DetectCDx, which is notably the first FDA-approved companion diagnostic immunoassay for a gene therapy.

2. FDA Releases Guidance on CGT Manufacturing Changes, Good Cause for PMR Noncompliance

The FDA has released new draft guidance on manufacturing cell and gene therapy (CGT) products as well as on noncompliance with postmarketing requirements (PMRs). Specifically, the manufacturing guidance focuses on the management and reporting of manufacturing changes for investigational and licensed CGT products and comparability studies to assess these changes.

3. ICER Rates Exa-Cel and Lovo-Cel Cost-Effective up to $2 Million

The Institute for Clinical and Economic Review (ICER) has posted a finalized version of its evidence report assessing the cost effectiveness of gene therapy for sickle cell disease (SCD), namely exagamglogene autotemcel (exa-cel; Vertex Pharmaceuticals and CRISPR Therapeutics) and lovotibeglogene autotemcel (lovo-cel; bluebird bio). The report, an updated version of a May 2023 draft, includes revisions made based on comments from patient groups, clinicians, drug manufacturers, and other stakeholders and will be reviewed at a virtual public meeting of the California Technology Assessment Forum on July 27, 2023. While much of the report remains the same, ICER did update the calculated health-benefit price benchmark for lovo-cel and exa-cel to be between $1.35 million to $2.05 million.

4. PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy Nets Fast Track, Orphan Drug Designations

The FDA has granted fast track and orphan drug designations to Rocket Pharmaceuticals’ RP-A601 gene therapy for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). RP-A601 is an adeno-associated virus vector (AAV.rh74) gene therapy. Rocket announced that its investigational new drug application (IND) for the therapy was cleared by the FDA in May 2023 based on preclinical studies that demonstrated decreased arrhythmias and increased survival.

5. Frequency Therapeutics Merges With Korro Bio

Korro Bio noted that it anticipates the resulting new company will operate under the Korro Bio, Inc. name and will focus its initial activities on furthering the development of Korro’s existing RNA editing programs, including its lead program for alpha-1 antitrypsin deficiency. “Following comprehensive review and consideration of our strategic options, management and our Board of Directors believe the merger with Korro Bio provides the best opportunity for the company and its stockholders,” David. L. Lucchino, the CEO of Frequency Therapeutics, said in a statement.

6. Eli Lilly Subsidiary Prevail Therapeutics Enters AAV Capsid Option Agreement With Sangamo

Under the terms of the agreement, Prevail may evaluate and license AAV capsids that Sangamo Therapeutics has developed with its Selecting In vivo For Transduction and Expression of RNA (SIFTER) platform for the purpose of targeting specific neurological indications. The SIFTER capsids were designed to be administered to the cerebrospinal fluid with the intention of efficiently delivering gene therapy products to the central nervous system.

7. Michael J. Fox Foundation Provides NysnoBio With Funding for Parkin Gene Therapy 

The funding will be used to support manufacturing of product for IND-enabling studies for NB001, NysnoBio’s investigational gene therapy that is intended to treat patients with Parkin gene-associated Parkinson disease (PD). “Parkin is a highly validated genetic target relevant to many in the PD community with younger disease onset, for whom new treatments could offer significant benefit,” Shalini Padmanabhan, PhD, the vice president of Michael J. Fox Foundation, said in a statement.

8. Genprex Licenses More Intellectual Property from University of Pittsburgh 

As a result of the new agreement with the University of Pittsburgh, which regards a gene therapy intended to treat both type 1 and type 2 diabetes, Genprex has garnered an exclusive license to a patent application and related technology and a non-exclusive license to additional intellectual property. “Using these technologies, Genprex’s product candidates are currently being evaluated and optimized in preclinical studies at the University of Pittsburgh,” Mark Berger, MD, the chief medical officer of Genprex, said in a statement.

9. MilliporeSigma to Adds Space for Cell Culture Media Production to Its Kansas Facility

MilliporeSigma has put $25 million towards expanding its facility, located in Lenexa, Kansas, with an addition of 98,000 square feet of lab and production space intended to help meet the current demand for dry powder media in the Americas. Dry powder media is used in the production of gene therapies and other advanced therapeutics.

10. CoJourney Completes Series A Financing and Expands Operations 

CoJourney, a contract development and manufacturing organization focused on cell therapy and genetic medicines, announced that it has received $30 million in funding from a round of financing led by Legend Holdings. The company also reported that it has expanded its manufacturing capabilities in the United States.