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Orchard Therapeutics develops autologous ex vivo gene therapies to treat rare, life-threatening genetic diseases. Its approach collects a patient’s hematopoietic stem cells, edits a functional gene outside the body, and reinserts the corrected cells to rebuild the blood and immune system. The company differentiates itself by delivering single-dose, patient-specific cures that aim to address the disease at its source, with Libmeldy/Lenmeldy as its approved product and a clinical-stage pipeline for other conditions. Orchard’s goal is to provide safe, one-time therapies for children with severe genetic disorders and to expand a portfolio of approved and investigational gene therapies through its Kyowa Kirin ownership.
Industries
Biotechnology
Healthcare
Company Size
201-500
Company Stage
IPO
Headquarters
London, United Kingdom
Founded
2015
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Total Funding
$691.7M
Above
Industry Average
Funded Over
8 Rounds
Hybrid Work Options
Orchard Therapeutics announces new gene therapy manufacturing facility. A new facility in California will expand Orchard Therapeutic's capacity to develop and deliver lentiviral vector and gene-corrected hematopoetic stem cells. Orchard Therapeutics announced in a Dec. 13, 2018 press release that it signed a long-term lease agreement to build out a gene therapy manufacturing facility in Fremont, CA. The new 150,000-ft[2] facility adds to the Foster City and Menlo Park, CA sites, which oversee the ongoing development and validation of the manufacture of Orchard's ex vivo gene therapy product candidates. Once operational, the new site will provide cGMP manufacturing capacity for both lentiviral vector and cryopreserved cell therapy products, enhancing Orchard's ability to manufacture and deliver gene-corrected hematopoetic stem cells for a range of diseases on a global basis. In addition to this expanded capacity, the company also plans to continue its collaborations with its contract manufacturing partners. "The expansion of our California operations to now include a manufacturing facility is a critical step in advancing Orchard's capabilities to supply products for our ex vivo gene therapy programs," said Stewart Craig, chief manufacturing officer of Orchard Therapeutics, in the press release. "We believe that this new facility, as an early investment in our own manufacturing, will not only drive efficiencies and scalability in terms of lentiviral vector and drug product development, it will also complement the capabilities of our existing vector and drug product manufacturing partners to support the potential launch of our gene therapy clinical product candidates." The build-out of Orchard's new manufacturing facility is expected to begin in 2019, and the company expects to hire more than 100 full-time employees over the next few years to support in-house manufacturing efforts. Stay at the forefront of biopharmaceutical innovation - subscribe to BioPharm International for expert insights on drug development, manufacturing, compliance, and more.
Er-Kim will collaborate with Orchard Therapeutics to refer eligible children with early-onset MLD identified in these countries to an established European-based qualified treatment center.
Believe "embodies a model of success and resistance to the worrying phenomenon of consolidation at work in the sector that has seen the successive acquisition of many significant distributors", it adds, highlighting Sony Music's acquisition of The Orchard and AWAL.
Pictured: FDA signage at its headquarters in Maryland/iStock, hapabapaThe FDA on Monday approved Orchard Therapeutics’ autologous gene therapy atidarsagene autotemcel, which will now be marketed as Lenmeldy, for the treatment of metachromatic leukodystrophy in pediatric patients.Lenmeldy is the first authorized gene therapy for children with pre-symptomatic late infantile or early juvenile metachromatic leukodystrophy (MLD), according to the FDA’s press release on the approval. Lenmeldy is also the first approved therapy for early juvenile or early symptomatic forms of the disease.Orchard CEO Bobby Gaspar in a statement said that Lenmeldy’s approval “opens up tremendous new possibilities” for children suffering from these early-onset forms of MLD, “who previously had no treatment options beyond supportive and end-of-life care.”The company on Monday did not provide specific launch details for Lenmeldy but promises to do so within the week.MLD is a rare and heritable metabolic disease that affects one in every 100,000 live births. It is characterized by a pathological build-up of sulfatides in the brain, liver, kidneys, spleen and other body parts, which over time can cause organ damage and neurological issues. Those with MLD often suffer from motor, behavioral and cognitive problems, as well as seizures. Patients also slowly lose the ability to move, talk, swallow and eat.Children with MLD often degenerate to a vegetative state and require round-the-clock care. Most patients die within five years of disease onset.MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene, which under healthy circumstances produces an enzyme that breaks down sulfatides
Orchard Therapeutics enters agreement with Beneluxa consortium to facilitate reimbursed access to Libmeldy.
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Industries
Biotechnology
Healthcare
Company Size
201-500
Company Stage
IPO
Headquarters
London, United Kingdom
Founded
2015
Find jobs on Simplify and start your career today