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Orchard Therapeutics, Pharming Ink Development Pact For Gene Therapy

  • Orchard Therapeutics plc (NASDAQ: ORTX) and Pharming Group NV (NASDAQ: PHAR) have collaborated to research, develop, manufacture and commercialize OTL-105 for hereditary angioedema (HAE).

  • OTL-105 is an investigational autologous hematopoietic stem cell (HSC) gene therapy designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks.

  • OTL-105 inserts one or more functional copies of the SERPING1 gene into patients' HSCs ex vivo, which are then transplanted back into the patient for potential durable C1-INH production.

  • Under the terms of the collaboration, Pharming has worldwide rights to OTL-105 and will be responsible for clinical development, regulatory filings, and commercialization.

  • Orchard will lead the IND-enabling activities and oversee the manufacturing of OTL-105 during pre-clinical and clinical development, which Pharming will fund.

  • In addition, both companies will explore the application of a non-toxic conditioning regimen for use with OTL-105 administration.

  • Orchard will receive an upfront payment of $17.5 million comprising $10 million in cash and a $7.5-million equity investment.

  • Orchard is also eligible to receive up to $189.5 million in milestone payments and royalty payments on future worldwide sales.

  • ORTX, PHAR Price Action: ORTX shares were down 2.16% at $4.30 at last check Thursday, while PHAR shares were up 2.34% at $11.53.

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