Month: January 2021

Orchard Therapeutics Announces OTL-200 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation by FDA for the Treatment of Metachromatic Leukodystrophy (MLD)

News / By

Interactions with FDA anticipated by mid-2021 to determine the BLA filing strategy for OTL-200 BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) — Orchard Therapeutics, a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200, an investigational ex vivo autologous hematopoietic stem …

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In Memorial or Honorarium – December 2020

In Memory or In Honor Donation / By

The ULF received donations in memorial or honorarium for the following during the month of December 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org or by calling 815.748.3211. IN HONOR OF Aayaansh ModiAnonymous  Ainsley CunninghamAnonymous Cheryl Malden  Alex Hiles Cynthia Powell  Amari BallDiane Mobeck Ken Mobeck  Ben …

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FDA Clearance of Investigational New Drug for Patients with Krabbe Disease

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January 4, 2021 by Dan Salvo Forge Biologics has received IND, IBC, and IRB clearance for FBX-101, a gene therapy for patients with Krabbe disease, a rare and fatal genetic disorder FBX-101 is a first-in-human potential treatment utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene to cells in the CNS and …

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