Around the Helix: Cell and Gene Therapy Company Updates – April 29, 2026

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. Lonvo-z Advances Toward Approval as First In Vivo CRISPR Therapy for Hereditary Angioedema

Intellia Therapeutics has initiated a rolling biologics license application (BLA) submission to the FDA for lonvoguran ziclumeran (lonvo-z), an investigational one-time CRISPR-based gene editing therapy for hereditary angioedema (HAE), following phase 3 data showing substantial reductions in attack rates and treatment burden.

2. Amanda Piquet, MD, FAAN, on Assessing Miv-Cel in Stiff Person Syndrome

Data from a phase 2 single-arm registrational trial evaluating Kyverna Therapeutics’ mivocabtagene autoleucel (miv-cel; also known as KYV-101), an autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy for stiff person syndrome (SPS), were presented at the 2026 American Academy of Neurology (AAN) Annual Meeting, held April 18 to 22 in Chicago, Illinois. Amanda Piquet, MD, director of the Autoimmune Neurology Program and holder of the Celine Dion Foundation Endowed Chair in Autoimmune Neurology at the University of Colorado, reported the findings and subsequently spoke with CGTLive®’s sister site NeurologyLive® on the conference floor.

3. Bad T-cells Lead to Failed Cancer Treatments

In the context of a discussion of the potential of CAR-T in neuroblastoma, Kristopher R. Bosse, MD, told the ImmunoLogic cohosts about how collecting and storing T-cells from patients early in the treatment journey may allow for better treatment options later on.

4. Emerging Therapies in DMD

This panel discussion, featuring experts including John F. Brandsema, MD, and Aravindhan Veerapandiyan, MD, surveys the Duchenne muscular dystrophy (DMD) clinical trial landscape. Next-generation microdystrophin gene therapies are highlighted as "second-generation" approaches that refine vector design, transgene composition, promoters, and tissue targeting. It remains too early to determine which will prove superior, but the panel is enthusiastic.

5. Orchard’s HSC Gene Therapy for MPS-IIIA Receives Innovation Passport Designation Under UK's Refreshed ILAP

Orchard Therapeutics has received Innovation Passport designation for OTL-201, an investigational ex vivo autologous hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIA (MPS-IIIA; Sanfilippo syndrome type A), under the United Kingdom's refreshed Innovative Licensing and Access Pathway (ILAP). The designation provides a structured framework for early, sustained engagement with the MHRA, NICE, and NHS. OTL-201, which uses a lentiviral vector to deliver a functional SGSH gene, is currently under evaluation in an investigator-sponsored proof-of-concept trial (NCT04201405) and holds FDA orphan drug and rare pediatric disease designations.

6. Rocket Pharmaceuticals Sells Rare Pediatric Disease Priority Review Voucher Gained From Kresladi Approval

Rocket Pharmaceuticals has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $180 million. The PRV was awarded following the FDA's accelerated approval of KRESLADI (marnetegragene autotemcel), the company's gene therapy for leukocyte adhesion deficiency-I. Proceeds from the sale, which the company characterizes as nondilutive capital, are intended to support advancement of Rocket's clinical-stage cardiovascular gene therapy programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, with cash runway now projected to extend into the second quarter of 2028.