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

News / January 22, 2021

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 …

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

In Memorial or Honorarium – December 2020

In Memory or In Honor Donation / February 6, 2021

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

uncategorized / January 5, 2021

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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Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)

News / December 21, 2020

Press Release: Orchard Therapeutics BOSTON/LONDON/MILAN, December 21, 2020 – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy …

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In Memorial or Honorarium – November 2020

In Memory or In Honor Donation / December 21, 2020

The ULF received donations in memorial or honorarium for the following during the month of November 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 Alex Bouley Bernard Levine, M.D.  Aryabella HaegerShannon Wright  Daxton WallaceSandy Wallace  Emily CullenLisa Cullen …

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In Memorial or Honorarium – October 2020

In Memory or In Honor Donation, News / February 6, 2021

The ULF received donations in memorial or honorarium for the following during the month of October 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 C. Michael Jaynes: Angela Pontius, C. Michael Jaynes – All those fighting leukodystrophies and …

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In Memorial or Honorarium – September 2020

In Memory or In Honor Donation / October 4, 2020

The ULF received donations in memorial or honorarium for the following during the month of September 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 Ella McKee: Heather McKee – For our brave daughter who continues to fit her …

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In Memorial or Honorarium – August 2020

In Memory or In Honor Donation, uncategorized / October 4, 2020

The ULF received donations in memorial or honorarium for the following during the month of August 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 Amie Matlon: Marianne Matlon  Aveline Brendel: Pauline Naudin, William Brendel, Amy McKenzie, Ryan McKenzie, …

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In Memorial or Honorarium – July 2020

In Memory or In Honor Donation, uncategorized / October 4, 2020

The ULF received donations in memorial or honorarium for the following during the month of July 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 David Stocki: Melissa Scier – For my son who has Alexander Disease Leukodystrophy  Dax …

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In Memorial or Honorarium – June 2020

In Memory or In Honor Donation / July 14, 2020

The ULF received donations in memorial or honorarium for the following during the month of June 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 Angelica Gallegos: Rozzana Vasquez Cobos  Anna Walentowski: Anonymous  Briana Singh: Stephanie Singh  Dax Wallace: …

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