Hola,
¿eres nuevo aquí?

Regístrate gratis y conecta tu empresa con financiación pública, partners y proyectos.

Tengo cuenta

Regístrate

Ver video

GetRadi

Financiado

Cerrado

Gene Therapy of Rare Diseases

More than 300 million people worldwide are suffering from more than 6000 rare diseases. Nearly all of these rare diseases are caused by a single inherited mutation and cannot be treated effectively. Repair of the defective gene by... ver más

31/08/2026

UCPH

Presupuesto desconocido

Líder del proyecto

KOBENHAVNS UNIVERSITET No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2022-09-01

HORIZON-MSCA-2021-DN-01-01: MSCA Doctoral Networks 2021 ExpectedOutcome:

I+D

Cerrada hace 3 años

0% 100%

Información adicional privada

No hay información privada compartida para este proyecto. Habla con el coordinador.

Participantes

UCPH

Lider

Conecta tu I+D

¿Tienes un proyecto y buscas un partner? Gracias a nuestro motor inteligente podemos recomendarte los mejores socios y ponerte en contacto con ellos. Te lo explicamos en este video

Proyectos interesantes

PRIME Prime editing to Repair Inherited Metabolic Errors: in vivo... 1M€ Cerrado

Genome Biologics Reducing the cost and increasing the speed of preclinical re... 71K€ Cerrado

CCedit Coiled coil mediated exonuclease tethering technology for th... 150K€ Cerrado

ImmunoStem Dissecting and Overcoming Innate Immune Barriers for Therape... 2M€ Cerrado

EnhancerDesign AI driven enhancer design for gene therapy 150K€ Cerrado

JDC2023-051690-I Synthetic CRISPR/Cas based tools: Enhancing precision medici... 72K€ Cerrado

Duración del proyecto: 47 meses Fecha Inicio: 2022-09-01
Fecha Fin: 2026-08-31

Líder del proyecto

KOBENHAVNS UNIVERSITET No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto More than 300 million people worldwide are suffering from more than 6000 rare diseases. Nearly all of these rare diseases are caused by a single inherited mutation and cannot be treated effectively. Repair of the defective gene by gene editing is the only possible curative therapy. However, only for very few rare diseases such gene editing therapy has reached the clinic. The “Gene therapy of Rare Diseases” (GetRadi) consortium aims now to contribute strongly to the establishment of more gene therapies for rare diseases. This will be accomplished by training of future leaders in gene therapy of rare diseases preforming ambitious research projects, with the following objectives: (i) Improving transfer of genome editing tools to target cells, (ii) improving gene editing efficiency, and (iii) improving safety of gene therapy. Strong participation of the pharmaceutical industry to research projects and training, development of novel in vitro and in vivo models for rare diseases to test gene therapies in relevant settings, and application of several unique genome editing tools developed by the applicants to the treatment of rare diseases are hallmarks of the network. GetRadi brings together strong industrial beneficiaries (AstraZeneca, Miltenyi Biotec) supervising 3 ESR, highly innovative academic beneficiaries supervising 7 ESR, 3 additional industrial partners, and 4 student enrolling universities. Professional outreach training with access to widely used social media channels will be provided by the partner “European Consortium for Communicating Gene and Cell Therapy Information”. Complementary unique expertise that is spread efficiently by network-wide training and secondments, excellent quality of supervision and intersectoral interactions, innovative transferable skill training, and efficient consortium management will result in an exceptional training of future leaders in gene therapy of rare diseases.

Conecta tu I+D

Entra hoy

¿Olvidé mi contraseña?

Financiación

Empresas

CTIs/Universidades

Proyectos

Investigadores