ASH 2022 | Delivery of mRNA-loaded LNPs to the HSC niche to treat BMF disorders

Stefano Rivella • 11 Dec 2022

ASH 2022

General

Stefano Rivella, PhD, Children’s Hospital of Philadelphia, Philadelphia, PA, discusses the advantages of using mRNA-loaded lipid nanoparticles (LNPs) that are directly delivered to the hematopoietic stem cell (HSC) niche to treat bone marrow failure (BMF) disorders. Dr Rivella explains that this strategy allows avoiding myeloablation and bone marrow transplantation (BMT) which are toxic and costly. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

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Disclosures

BVF Partners L.P: Consultancy; Cambridge Healthcare Res: Consultancy; Catenion: Consultancy; Celgene: Consultancy; Disc Medicine: Current holder of stock options in a privately-held company, Membership on an entity’s Board of Directors or advisory committees; First Manhattan Co: Consultancy; FORMA: Consultancy; Ghost Tree Capita: Consultancy; Incyte: Membership on an entity’s Board of Directors or advisory committees; Ionis Pharmaceuticals: Membership on an entity’s Board of Directors or advisory committees; Keros Therapeutics: Consultancy; MeiraGTx: Consultancy; Noble insight: Consultancy; Protagonist Therapeutics: Consultancy; Rallybio, LLC: Consultancy; Sanofi Aventis U.S: Consultancy; Slingshot Insight: Consultancy; Techspert.io: Consultancy; venBio Select LLC: Consultancy; Vifor: Membership on an entity’s Board of Directors or advisory committees.