Atamyo Seeking Approval for LGMD Gene Therapy Trial in Europe

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a type of limb-girdle muscular dystrophy (LGMD).

The company, a spin-off from Genethon, a Paris-based genetics research laboratory, has submitted an application asking three European nations for permission to begin clinical testing of ATA-100 for LGMD type 2I/R9, known as LGMD2I/R9.

“This is a first step in our mission to bring to patients suffering from limb-girdle muscular dystrophies (LGMD) a new generation of safe and effective gene therapies,” Stephane Degove, Atamyo’s CEO and co-founder, said in a press release.

If the clinical trial application (CTA) is approved, Atamyo expects to begin dosing patients in the first half of next year.

“We are thrilled to file our first CTA in France, Denmark and the U.K. for the devastating LGMD2I/R9 disease,” Degove said.

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LGMD2I/R9 is caused by mutations in the gene FKRP, which provides instructions for making a protein with the same name. This form of muscular dystrophy is estimated to affect 5,000 people in the U.S. and Europe, according to Atamyo. Symptoms generally begin in late childhood or early adulthood, with progressive muscle weakness that eventually makes it impossible to walk. Problems with breathing and heart function are common.

The aim of the ATA-100 gene therapy is to deliver a healthy version of the FKRP gene to the body’s cells, allowing them to produce a functional form of the FKRP protein. The therapy delivers its genetic cargo using a specifically engineered viral vector, called an adeno-associated virus (AAV). This modified virus is often used in gene therapies because it’s safe for patients and easy to manipulate in a laboratory.

ATA-100 has been well-tolerated in preclinical mouse models, and has shown the potential to reduce symptoms and biological markers of disease “at unprecedented low doses” for AAV-based gene therapies for muscle disease, according to Atamyo.

Atamyo was launched by Genethon, which has expertise in AAV gene therapies and muscular dystrophies.

“This CTA filing is the achievement of 30 years of research at Genethon, first on the search for genes responsible [for] LGMDs, then on the development of gene therapies for these complex diseases,” said Isabelle Richard, PhD, co-founder and chief scientific officer of Atamyo.

In addition to ATA-100, Atamyo is developing gene therapies for two other types of LGMD: LGMD2C/R5 and LGMD2A/R1. Both are related to protein deficiencies.

“The creation of Atamyo by Genethon stems from its ambition to create a leader of gene therapy applied to LGMDs,” said Frédéric Revah, PhD, CEO of Genethon.

“Its mission will be to develop these treatments for the benefit of the largest number of patients who have no therapeutic options,” Revah said, adding that Atamyo will “raise the funds necessary to develop its LGMD programs up to the market.”

The company recently announced the appointment of Sophie Olivier, MD, as its chief medical officer, overseeing clinical development and medical affairs.

“Knowing that our work has the potential to improve the life of patients with LGMDs is very motivating,” Olivier said.