Latest Headlines

  1. Orchard Therapeutics Announces Encouraging Update from Proof-of-Concept Study of OTL-203 for the Treatment of Mucopolysaccharidosis Type I (MPS-I)

    Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, a gene therapy for the treatment of mucopolysaccharidosis type I (MPS-I) developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan

  2. Orchard Therapeutics Presents an Integrated Data Analysis Demonstrating Sustained Clinical Benefit of OTL-200 for the Treatment of Metachromatic Leukodystrophy

    Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced results from an integrated data analysis of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The results, which demonstrate positive clinical effects of OTL-200 in the treatment of MLD and a consistent safety profile, were featured today in an oral presentation at the Society for the Study of Inborn Errors of Metabolism (SSIEM) symposium in Rotterdam, the Netherlands. MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms, patients will not survive beyond their first few years of life.

  3. Biologics Modular Announces Issuance Of US Patent For Modular Cleanroom Facility

    Biologics Modular today announced a significant milestone with respect to intellectual property, demonstrating its ongoing commitment to innovation and leadership of the modular cleanroom industry. The patent award encompasses the area where cleanroom modules are preassembled with air filtration systems already in place.

  4. Alliance For Regenerative Medicine (ARM) Releases Therapeutic Developers’ Statement Of Principles

    The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, has released a Therapeutic Developers’ Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications.

    The statement, developed by ARM’s Gene Editing Task Force and signed by 13 therapeutic developers using gene editing technologies, specifies five key principles for the ethical use of gene editing and genetic modification.

  5. Cognate BioServices Secures Capital Investment To Expand Commercial Manufacturing Capacity And Services For Cell Therapy Production

    Cognate BioServices, a leading contract development and manufacturing organization ("CDMO") in the global cellular therapies industry, announced today it has secured a financial sponsor to support its short-term and long-term growth as it closed on a round of growth capital from EW Healthcare Partners ("EW Healthcare").

  6. New $500 Million Gene Therapy Drug Manufacturing Facility Announced By Pfizer (Sanford NC)

    Pfizer announced today an additional half billion dollar investment for the construction of its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. This facility is anticipated to support Pfizer’s continuing investment in gene therapy research and development, similar to Pfizer’s Chapel Hill and Kit Creek, North Carolina research and development sites. This facility would expand the company’s presence in North Carolina, where there are currently more than 3,600 Pfizer colleagues, including 650 in

  7. Gemini Bio-Products To Distribute Nordmark Cell Therapy Reagent Products In US

    Gemini Bioproducts, LLC (“Gemini”), a portfolio company of BelHealth Investment Partners, LLC (“BelHealth”), a New York-based healthcare private equity firm, recently announced a distribution agreement with Nordmark Biochemicals, a division of Nordmark Arzneimittel GmbH & Co. KG (“Nordmark”). Launching this week, customers in the United States can now purchase Nordmark’s line of collagenase and neutral protease products directly from Gemini.

  8. SIRION Biotech Soars To New Heights: Announces 100 Percent Record Growth In 2019 And Expands US Presence

    SIRION Biotech GmbH (“SIRION”), a world leader in viral vector-based gene delivery technologies for gene and cell therapy, announced financial results for the first half of 2019.  SIRION’s viral vector technologies make breakthrough advances in cell and gene therapy possible.

  9. Facility Design Takeaways From Novartis And University Of Pennsylvania Cell Therapy Facility

    In August the Novartis Institutes for Bio-Medical Research, based in Cambridge, Massachusetts, and the University of Pennsylvania’s Perelman School of Medicine (Penn Medicine) announced they had entered into an exclusive global research and licensing agreement. Under the alliance, Penn Medicine granted Novartis an exclusive worldwide license to the technologies used in an ongoing trial of patients with chronic lymphocytic leukemia (CLL) as well as future therapies based on chimeric antigen receptor (CAR) technology developed through the collaboration.

  10. Bayer Increases Cell Therapy Presence With Acquisition Of BlueRock Therapeutics

    Bayer AG and BlueRock Therapeutics recently announced an agreement under which Bayer will fully acquire BlueRock Therapeutics, a privately held US-headquartered biotechnology company focused on developing engineered cell therapies in the fields of neurology, cardiology and immunology, using a proprietary induced pluripotent stem cell (iPSC) platform.