Cell therapy weekly: Clinical Trial Application approval for muscular dystrophy gene therapy

Written by Megan Giboney

This week: Slingshot Bio (CA, USA) launched a portfolio of products to tackle quality control challenges in cell therapy development and manufacturing, Aaron Ondrey has been appointed Chief Financial Officer of Rocket Pharmaceuticals (NY, USA) and Atamyo Therapeutics (Evry, France) has received approval to initiate a clinical trial investigating its gene therapy for limb-girdle muscular dystrophy.

The news highlights:


Slingshot Bio launches synthetic cells to improve analytical methodologies for cell therapies

Slingshot Bio has introduced a portfolio of products to tackle quality control challenges in cell therapy development and manufacturing. One key challenge addressed is the dependence on controls that are frequently unavailable or flawed. These controls are crucial for validating the accuracy and consistency of analytical methods used to evaluate cell therapy products.

The portfolio includes synthetic cells expressing precise levels of markers like B-cell maturation antigen and CD19, along with a product featuring lymphocyte subsets with defined cell populations relevant to standardizing patient characterization

CEO of Slingshot Biosciences, Jeff Kim, states: “These synthetic cells are tailored for cell therapy developers. They provide a dependable means for characterizing patient starting materials and CAR product characterization, addressing a previously unmet need. Our commitment lies in empowering the cell therapy community with the necessary tools for making informed, data-driven decisions. We are dedicated to enhancing cell therapy research and manufacturing precision through our commitment to excellence and strategic global partnerships.”

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Rocket Pharmaceuticals appoints new Chief Financial Officer

Aaron Ondrey has been appointed Chief Financial Officer of Rocket Pharmaceuticals. Most recently, Ondrey served as Chief Financial Officer of Mirati Therapeutics (CA, USA) and he brings over 20 years of experience in financial management, strategic planning, commercial finance, capital allocation and mergers and acquisitions.

“Today’s leadership announcements underscore Rocket’s preparedness as we transform from a clinical organization to a commercial stage gene therapy company, notably by expanding our financial expertise with the addition of seasoned finance leader, Aaron Ondrey as our Chief Financial Officer,” said Gaurav Shah, Rocket’s CEO.

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Clinical trial to commence for limb-girdle muscular dystrophy gene therapy

The Italian Medicines Agency and the French Medicines Agency have approved a Clinical Trial Application for Atamyo Therapeutics’ gene therapy candidate ATA-200, aimed at treating limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5) caused by mutations in the gamma–sarcoglycan gene.

This regulatory authorization allows Ataymo to initiate a Phase Ib, dose escalation study (NCT05973630) to evaluate the safety and efficacy of ATA-200 in children.

“We are thrilled to obtain our [Clinical Trial Application] approval in France and Italy for the devastating LGMD2C/R5 disease affecting primarily a pediatric population and for which there is no approved treatment,” said Sophie Olivier, Chief Medical Officer of Atamyo. “Atamyo plans to initiate dosing in patients for ATA-200 in third quarter 2024.”

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