Orchard Therapeutics - U.S.’s Post

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We are thrilled to announce that the FDA has approved Lenmeldy, the only therapy for eligible children with early-onset metachromatic leukodystrophy in the U.S. Read more in our press release: https://bit.ly/4ceTmzr.

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Kristen Czech

Senior Manager, Clinical Execution at DevPro Biopharma

4mo

My nephew passed away two years ago from this devastating disease. The entire MLD community was anxiously awaiting this news yesterday. There are not enough words to express how thankful we all are to everyone who made this happen. 

Rob Goldstein-Mahon

Patient Advocacy at Ionis | Helping People and Organizations Change the World (posts are my own, not on behalf of Ionis)

4mo

Such an important milestone moment for the MLD community. 🎉

Angela C.

Healthcare Expert & Leader/Patient Engagement/ Communication/Public Affairs/Rare Diseases

4mo

Congratulations!!!! I have a personal history with this terrible disease, and I´m thrilled about the lives this achievement will save! I wrote a short post about it : https://www.linkedin.com/posts/angelapublic_rarediseaseday-raredisease-rarediseases-activity-7175589970395451392-8BdY?utm_source=share&utm_medium=member_desktop

This is fantastic news! The approval of Lenmeldy by the FDA marks a significant milestone in the treatment of early-onset metachromatic leukodystrophy for children in the U.S. It's reassuring to see advancements in medical therapies that can potentially improve the lives of those affected by this condition. Congratulations on this achievement!

Erin Hugger DiFilippo

Vice President of Business Development

4mo

So happy to see this cell/ gene therapy make it to market! The initial clinical data from HSR-TIGET with MLD patients back in ~2012 time frame was so impressive. I'm happy to see that Orchard has taken this study forward and secured FDA approval. Very important outcome for patients with MLD.

Christine Waggoner

Senior Software Engineer, Technical Director, Award-winning Simulation Supervisor at Pixar Animation Studios, Cure GM1 Foundation Founder and President, Award-winning Patient Advocate

4mo

A great day for rare diseases and leukodystrophies!

Juan Miguel Sanchez Nieto, PhD

Bioassay Manager @ Moderna | Analytical Development - Cell & Gene Therapies - Vaccines

4mo

Bravo! Congratulations!

Anny Bedard

Orphan Drug Market Entry & Commercialization Strategy | Gene Therapy | Strategic Alliances | International

4mo

Congratulations! Huge achievement

Christopher Armah MSc. PhD

CEO and Founder of a Global Medical Writing and Biostats Consultancy Spearheading Innovation in the Pharmaceutical Industry

4mo

Huge congratulations to Orchard Therapeutics on the remarkable milestone. This approval marks a significant leap forward in the realm of paediatric diseases, offering hope and a lifeline to children and families in need.

Ian Johnston

Project Lead Translational TCR Drug Development, R&D at Miltenyi Biotec

4mo

Wonderful news. It has been a long and challenging journey. Congratulations to Bobby Gaspar and adrian thrasher, the Orchard team and the various academic consortia members from Milan, Paris and the US that have worked so hard to develop these technologies and move these therapies forward.

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