Around the Helix: Cell and Gene Therapy Company Updates – April 16, 2025

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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social (@CGT_Live on X, or CGTLive on LinkedIn) and use #AroundTheHelix!

1. Lexeo’s Gene Therapy LX2006 Reduces Abnormal LVMI in Patients With Friedreich Ataxia Cardiomyopathy

Lexeo Therapeutics’ LX2006, an adeno-associated virus (AAV) vector-based gene therapy, has demonstrated the ability to reduce left ventricular mass index (LVMI) in patients with Friedreich ataxia (FA) cardiomyopathy who had a normal LVMI at baseline.

2. World Parkinson's Day 2025: Looking Back at Progress for Cell and Gene Therapy

In honor of World Parkinson's Day, observed annually on April 11 by the patient and clinician communities, CGTLive® took a look back at the progress that has been made in cell and gene therapy for Parkinson disease over the past year.

3. Fate Therapeutics’ iPSC-derived CAR-T FT819 Nabs FDA RMAT Designation for Lupus

Fate Therapeutics’ FT819, an investigational allogeneic induced pluripotent stem cell (iPSC)-derived chimeric antigen receptor T-cell (CAR-T) therapy, has garnered regenerative medicine advanced therapy (RMAT) designation from the FDA for active moderate to severe systemic lupus erythematosus, including lupus nephritis.

4. Verve Therapeutics’ Base Editing Therapy VERVE-102 Reduces LDL-C in Patients With HeFH and CAD

Verve Therapeutics’ VERVE-102, an investigational in vivo base editing therapy that uses a lipid nanoparticle (LNP) delivery system, has demonstrated the ability to reduce low-density lipoprotein cholesterol (LDL-C) and PCSK9 protein levels in patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) being treated in the phase 1b HEART-2 clinical trial (NCT06164730).

5. CGT Catapult and INmune Bio Team Up

Cell therapy developer INmune Bio has formed a partnership with innovation and technology organization CGT Catapult that will focus on providing commercial manufacturing readiness for INmune's cell therapy platforms. The first aim of the partnership will be the scaling up of manufacturing for INmune's recessive dystrophic epidermolysis bullosa cell therapy CORDStrom, which is currently in clinical development.

6. First Patient in Austria Receives UniQure and CSL Behring’s Hemophilia B Gene Therapy

The first patient in Austria has been treated with UniQure and CSL Behring’s hemophilia B gene therapy etranacogene dezaparvovec (marketed as Hemgenix). The patient's treatment with Hemgenix, which received conditional marketing authorization in the European Union in February 2023, took place at the Comprehensive Care Center of the University Hospital of Medicine Vienna.