Orchard Therapeutics

Orchard’s HSC gene therapy approach modifies a patient’s own blood stem cells outside the body to address serious genetic diseases.

Orchard focuses on treating rare, inherited disorders with limited or suboptimal therapeutic options, using groundbreaking science.

Orchard develops potential cures for genetic and severe diseases by modifying a patient’s own hematopoietic stem cells (HSCs) for permanent correction.

Orchard thanks participants in the MLD gene therapy clinical program and the broader MLD community for their support.

I’ve always felt strongly about the power in collaboration and connections to drive growth.

Orchard develops hematopoietic stem cell (HSC) gene therapies, which require specialized viral vectors, patient cell harvesting, gene modification, and reinsertion.

Orchard’s leadership team includes experts in hematopoietic stem cell (HSC) gene therapy, committed to advancing treatments for genetic and severe diseases.

“The reality that Livi could have been screened for MLD is just one of those things that I think about all the time. It shouldn’t be like that for families in the future.”

Orchard’s history spans early research in hematopoietic stem cell (HSC) gene therapy, contributing to its development as a potentially life-changing treatment.

Orchard has its global headquarters in London and U.S. headquarters in Boston.

HSC gene therapy aims to transform lives of people with severe and genetic diseases by addressing the underlying disease cause.

Orchard Therapeutics (Europe) Limited outlines its approach to handling personal data during and after the recruitment process.

Orchard Therapeutics maintains a compliance program to prevent legal violations, promote ethical interactions, and adhere to industry standards.

Orchard Therapeutics establishes a **$4.25 million** price tag for its gene therapy **Lenmeldy** in the US, following its recent regulatory approval. The therapy targets **metachromatic leukodystrophy (MLD)**, a rare genetic disorder. Orchard aims to make the treatment accessible while navigating commercialization challenges post-approval.

Mar 2024BioSpaceNews article

Orchard Therapeutics receives FDA approval for **Lenmeldy (atidarsagene autotemcel)**, the first gene therapy for pediatric **metachromatic leukodystrophy (MLD)**. The therapy targets children with the rare neurodegenerative disorder. **AGC Biologics** is selected as the manufacturer for production.

Mar 2024BioPharm InternationalNews article

Kyowa Kirin acquires Orchard Therapeutics for **$477 million** to gain access to its therapy for **early-onset metachromatic leukodystrophy**, a rare genetic disorder. The transaction expands Kyowa Kirin’s pipeline of advanced therapies targeting neurological diseases. Orchard Therapeutics develops gene therapies for inherited metabolic disorders.

Oct 2023PharmaTimesNews article

England’s National Institute for Health and Care Excellence (NICE) rejects Orchard Therapeutics’ **Libmeldy**, a stem cell gene therapy for **metachromatic leukodystrophy (MLD)**, citing concerns over its cost-effectiveness. The therapy, targeting a rare and fatal genetic disorder with no existing cures, was deemed too expensive during initial negotiations. Orchard’s treatment, approved in the EU, remains unavailable in England pending further review.

Jul 2021Fierce PharmaNews article