Waskyra™, a Gene Therapy Manufactured Using RetroNectin®,
Receives U.S. and EU Marketing Authorization Approvals

Takara Bio Inc. announces that Fondazione Telethon ETS ⁽¹⁾ (headquartered in Milan, Italy), has received marketing authorization from the U.S. Food and Drug Administration (FDA) ⁽²⁾ and the European Medicinal Agency (EMA) ⁽³⁾ for Waskyra™, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS) developed by Fondazione Telethon itself. Takara Bio supplies RetroNectin^® and grants a commercial-use license to Fondazione Telethon, and RetroNectin^® is used in the manufacturing process of Waskyra™.

 

RetroNectin^® is a recombinant protein uniquely developed by Takara Bio. It significantly enhances the efficiency of gene transduction into cells using viral vectors and enables highly efficient proliferation of cells after transduction. In the manufacturing of ex vivo gene therapy products such as Waskyra™, RetroNectin^® is used during the gene transduction step.

 

In September 2023 ⁽⁴⁾, we entered into an agreement with Fondazione Telethon covering the commercial-use licensing and supply of RetroNectin^®. Under this agreement, we have supplied RetroNection^® for the commercial production of Strimvelis™ ⁽⁵⁾, which is a Fondazione Telethon EU-approved ex vivo gene therapy for adenosine deaminase–deficient severe combined immunodeficiency (ADA‑SCID). Going forward, in addition to Strimvelis™ we will also supply RetroNectin^® for the commercial production of Waskyra™.

 

Takara Bio will continue to promote the supply and commercial licensing of RetroNectin^® for the expanding field of gene and cell therapy as a new modality, contributing to addressing unmet medical needs in gene and cell therapy.

 

 

Glossary / Notes

 

RetroNectin^®

RetroNectin^® is a recombinant protein derived from human fibronectin, a cell-adhesive protein. The method of gene transduction into cells using RetroNectin^® is known as the RetroNectin^® method. RetroNectin^® is a registered trademark of Takara Bio Inc.
 

Waskyra^TM

Waskyra (etuvetidigene autotemcel) is an ex vivo gene therapy, indicated for the treatment of patients aged 6 months and older with Wiskott-Aldrich syndrome (WAS). Waskyra is used to treat patients for whom a haematopoietic stem cell transplantation (HSCT) is appropriate, but for whom no suitable stem cell donor is available. Wiskott-Aldrich syndrome is a rare, inherited disease, seen almost exclusively in males, that affects blood cells and cells of the immune system (the body's natural defenses)

 

Strimvelis^TM

Strimvelis is an ex vivo gene therapy, indicated for the treatment of patient with severe combined immunodeficiency caused by adenosine deaminase (ADA‑SCID). Strimvelis is used to treat patients who cannot be treated by a bone-marrow transplant because they do not have a suitable, matched, related donor. ADA‑SCID is an extremely rare congenital immunodeficiency, in which there is a change in the gene needed to make an enzyme called adenosine deaminase (ADA). As a result, patients lack the ADA are defenseless against infections and typically fatal without treatment.

 

⁽¹⁾ https://www.fondazionetelethon.it/en/

⁽²⁾ https://www.fda.gov/vaccines-blood-biologics/waskyra

⁽³⁾ https://www.ema.europa.eu/en/medicines/human/EPAR/waskyra

⁽⁴⁾ News Release (dated September 25, 2023) Takara Bio enters into a license agreement of RetroNectin® with Fondazione Telethon ETS

⁽⁵⁾ https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis