Around the Helix: Cell and Gene Therapy Company Updates – July 10, 2024

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. Myrtelle’s Canavan Disease Gene Therapy Selected for FDA START Pilot Program

Myrtelle Inc has announced that the FDA selected its investigational gene therapy, rAAV-Olig001-ASPA (previously known as MYR-101), for inclusion in its Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.

2. Spur Therapeutics Continues to Make Progress in Evaluation of Gaucher Disease Gene Therapy FLT201

Spur Therapeutics (formerly known as Freeline Therapeutics) has announced newly updated results from its now fully-enrolled phase 1/2 GALILEO-1 clinical trial (NCT05324943) evaluating FLT201, an adeno-associated virus (AAV) vector-based gene therapy intended to treat Gaucher disease, along with updated plans for a new phase 3 clinical trial for FLT201.

3. Nkarta Launches Trial for CAR-NK Therapy NKX019 in Lupus and Gains Clearance for Separate Trial in Other Autoimmune Diseases

The first patient has entered screening for Nkarta’s Ntrust-1 clinical trial (NCT identifier pending) evaluating NKX019, an investigational allogeneic chimeric antigen receptor (CAR) natural killer (NK) cell therapy, for the treatment of lupus nephritis (LN); the company has also received clearance from the FDA for a separate clinical trial (Ntrust-2) for NKX019 in systemic sclerosis (SSc, scleroderma), idiopathic inflammatory myopathy (myositis), and ANCA-associated vasculitis.

4. Ocugen Doses First Patient With Retinitis Pigmentosa Gene Therapy in Phase 3 Trial

Ocugen has dosed the first patient with retinitis pigmentosa (RP) in its phase 3liMeliGhT trial (NCT06388200) of OCU400 gene therapy. The phase 3 trial will last 1 year and have a sample size of 150 participants, at least 8 years of age, half with RHO gene mutations and half that have gene agnostic RP.

5. Codexis Hands Off Engineered Enzymes to Crosswalk Therapeutics

The 2 companies have finalized a deal for Crosswalk’s purchase of Codexis’s investigational engineered enzymes that target Pompe disease and Fabry disease. Crosswalk intends to use the assets along with its protein biodistribution platform to deliver the enzymes to hard to reach tissues. "Patients with lysosomal storage diseases often face burdensome and frequent enzyme replacement infusions as part of current therapeutic options," Rizwana Islam, the SVP and head of metabolism at Crosswalk, said in a statement. "While these treatments save lives, they often leave residual disease burden in tissues they don't reach, leading to poor prognoses over time."

6. SmartCella Provides XyloCor Therapeutics Rights to Use Its Catheter System for Gene Therapy Delivery

Under a new license agreement, XyloCor will use SmartCella’s Extroducer® Infusion Catheter System for the delivery of its refractory angina gene therapy XC001 directly into the heart. Notably, SmartCella also recently garnered EUR 50 million via a share issue, which it intends to put towards commercialization of Extroducer and its cell and mRNA therapies.

7. Andelyn to Shoulder Vector Manufacturing for UMass Chan Medical School’s Cockayne Syndrome Gene Therapy

UMass Chan Medical School has selected the contract development and manufacturing organization Andelyn Biosciences to manufacture the AAV9-CSA vector that will be used in its investigational gene therapy program aimed at treating Cockayne syndrome. “Our dream of making a difference for Cockayne patients is coming closer every day, and signing the contract with Andelyn Biosciences to make the clinical material is a major step in that direction,” Miguel Sena-Esteves, PhD, an associate professor of neurology and director of the Translational Institute for Molecular Therapeutics at UMass, said in a statement.

8. uniQure's Shares Spike After Promising Gene Therapy Data Update

The company's shares shot up in value by 70% following it's announcement of updated interim data regarding its Huntington disease gene therapy AMT-130 from 29 patients treated across 2 phase 1/2 clinical trials. “We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of neurofilament light chain in the cerebrospinal fluid at 24 months,” Walid Abi-Saab, MD, the chief medical officer of uniQure, said in a statement.