Resumption of the clinical trial for RNA therapy for USH2a has been terminated

UPDATE September 27, 2023

In mid-August, we reported that ProQR Therapeutics, which had previously been forced to halt its two promising clinical trials, had found a new partner in Laboratoires Théa (Théa) to continue development. Unfortunately, this collaboration has been terminated prematurely.

One of the conditions for finalizing the agreement was that several key ophthalmic employees of ProQR would enter into employment with Théa. Regrettably, some employees have decided not to proceed with this, making it impossible to meet this closing condition. As a result, Théa has terminated the agreement.

The immediate consequence is that the clinical trial testing RNA therapy for individuals with Usher syndrome 2A will not be resumed at this time.

The full press release can be read here.

 

LEES ARTIKEL IN NEDERLANDS

ProQR has announced an agreement with Laboratoires Théa to acquire Ultevursen  programs.

In August 2022 ProQR decided to focus exclusively on the development of the Axiomer® RNA editing technology platform. ProQR Therapeutics was forced to stop promising clinical trials unless they found a new strategic partner to continue.
Read about this: http://www.ushersyndroom.nl/klinische-trials-celeste-en-serius-worden-noodgedwongen-stopgezet/

The new partner
Laboratoires Théa (Théa)
has signed an agreement with ProQR to continue the further development of the IRD Sepofurses and Ulteverses programs. Théa is European pharmaceutical company specialized in the research, development, and commercialization of eye care products. This family-owned is based in Clermont-Ferrand, France.

Within Théa, a fully dedicated team specializing in inherited retinal disorders and a new organization are currently being set up to manage these projects. More information on the next steps for these programs will be available in the coming weeks from Théa.

About Ultevursen
Ultevursen (formerly QR-421a) is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. Ultevursen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA.

The transaction is expected to close in the third quarter of 2023.

For further information or enquiries about the announcement today, please email: patientinfo@proqr.com.

The press release from ProQR can be found via this link.

Read more about RNA therapy on our Knowledge Portal:
http://www.ushersyndroom.nl/en/knowledge-portal/research/rna-therapy/