RegenixBio

Therapeutic Programs:

https://www.regenxbio.com/therapeutic-programs/

 

Sobi

Therapeutic Programs:

https://www.sobi.com/en/Our-Focus-Areas/Research-and-Development

 

Orchard

Therapeutic Programs:

http://orchard-tx.com/programmes/#

 

Sangamo

Therapeutic Programs:

https://www.sangamo.com/product-pipeline

 

Armagen

Therapeutic Programs:

Pipeline Chart

 

BioMarin

Therapeutic Programs:

http://www.biomarin.com/products/pipeline

 

Ultragenyx

Therapeutic Programs:

http://www.ultragenyx.com/pipeline/

 

Lysogene

Therapeutic Programs:

http://www.lysogene.com/patients-and-families/mps-iiia/

 

Abeona

Therapeutic Programs:

R&D

 

Esteve

Therapeutic Programs:

https://www.esteve.com/en/research-development

 

Shire

Therapeutic Programs:

https://www.shire.com/research-and-development/pipeline

 

Phoenix Nest:

Therapeutic Programs

http://www.phoenixnestbiotech.com/about-sanfilippo-syndrome/