Advanced gene editing platform for the treatment of type 1A muscular dystrophy

Summary

In 2017, we developed Uni-Large, a new generation of gene editing technologies that combines the precision of CRISPR with the effectiveness of vectors established in the clinic. We are currently developing the first treatment for congenital type 1A muscular dystrophy (MDC1A), although we expect that Uni-Large will be able to used in the treatment of other diseases.

MDC1A is a condition produced by mutations in LAMA2, a protein that exceeds the size limit of the adeno-associated virus (AAV) vectors, therefore, alternative therapies are needed. Uni-Large will provide a safe therapeutic opportunity.

It solves several challenges that impede a wider deployment of gene editing technologies, such as dependence on mutation and homologous recombination, and safety issues related to double-strand breaks and insertion mutagenesis.

Achievements

  • In 2017, we developed the first prototype of Uni-Large
  • In 2018, we took part in CaixaImpulse Validate
  • In June 2019, we filed the provisional patent application in the US.
  • During 2019, we validated the technology and performed a proof of principle in vivo.
  • During 2019, we constructed an early regulatory draft.
  • In 2020 we raised an additional €140,000 in non-dilutive funding.

Next Steps

Thanks to the support of CaixaImpulse Consolidate, we will advance on the preclinical phase, obtaining further validation with a larger mice cohort and we will produce a pilot GLP batch. Moreover, we will submit the provisional American patent application, and prepare a second batch of IP covering more specific aspects.

We also plan to generate a regulatory roadmap and we will perform an early consultation to FDA/EMA. We will explore new indications suitable for our technological platform and we expect to create a Spin-off and close a Series A during this year.

Team

Project leader

Marc Güell Cargol

UPF - Universitat Pompeu Fabra

Project leader

Senior postdoctoral scientist

Avencia Sánchez-Mejías

UPF - Universitat Pompeu Fabra

Partners

Validate-18
Consolidate-19

Scientific Area

Clinical sciences

Business area

Therapeutic

Research center

Universitat Pompeu Fabra