- UK-based Orchard Therapeutics has entered into a deal with Dutch company PharmaCell to manufacture the former's ex vivo gene therapy products for clinical trials and the market.
- Orchard's therapies use stem cells taken from a patient's body and equip them with a lentiviral vector — a retrovirus with a talent for integrating new genetic material into a variety of cells' DNA. The modified cells are then transplanted back into the patient in an effort to treat disorders such as severe combined immunodeficiencies (SCIDs) and Sanfilippo syndrome.
- No financial details were disclosed in a Jan. 9 statement.
Manufacturing gene and cell therapies for clinical trials and for the market is a complex process. Many companies, especially smaller biotechs, choose to partner with with contract manufacturing organizations (CMOs) like PharmaCell to lessen the burden.
This is the second manufacturing Orchard has signed in recent months. The first came in November when the company partnered with Oxford BioMedica for the development of its lentiviral programs.
Orchard isn't the only gene-focused company getting help from outside sources, either. Spark Therapeutics is working on an in vivo therapy for hemophilia B, and turned to Selecta Biosciences its the biotech's synthetic vaccine particles delivery technology to advance the drug's tolerability.
Meanwhile, CAR-T specialist Kite Pharma is strengthening its in-house production capabilities. Kite Pharma opened a commercial manufacturing facility earlier this year that it says will be able to as many as 5,000 patient therapies annually.
Orchard Therapeutics has two main drugs under investigation. The lead is a treatment for a type of SCID called adenosine deaminase (ADA). Children born with this are at risk of death from infection, and may also have a wide range of developmental abnormalities. Current treatments for the disease are limited to stem cell transplants or weekly enzyme injections. Orchard's drug is in clinical trials with more than 30 patients that have seen 100% survival rates in follow up periods ranging from 2 months to more than 4 years.
The company's other drug is preclinical and aims to help patients with Sanfilippo syndrome, which has no approved treatment. The disease leads to a loss of motor and behavioral functions and affects around one to two people per 100,000.