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Organovo Collaborates With MCRI and Leiden University Medical Center to Develop Stem Cell-Based Bioprinted Tissue Treatments for Kidney Disease
May 30, 2019 – (Organovo) – This multi-organizational effort integrates Organovo’s leading bioprinting platform with MCRI’s advanced stem cell differentiation technology and LUMC’s cell lines and clinical expertise.  The collaboration has been made possible through generous funding from Stem Cells Australia and CSL Limited.
Atara Biotherapeutics Appoints Pascal Touchon President, Chief Executive Officer and Member of the Board of Directors
May 28, 2019 – (Atara Biotherapeutics) – Dr. Touchon has over 30 years of global biopharmaceutical leadership experience, most recently at Novartis Oncology serving as Global Head, Cell & Gene and member of the Oncology Executive Committee. Notably in this role he was accountable for the Oncology Cell & Gene unit financial performance and responsible for all activities including the global launch of KYMRIAH.
Ziopharm Oncology Announces Exclusive License with National Cancer Institute to Identify and Use T-Cell Receptors Targeting Neoantigens for Cancer with Sleeping Beauty Platform
May 28, 2019 – (Ziopharm Oncology) – Pursuant to the terms of the license agreement, NIH will receive from Ziopharm an upfront payment and certain clinical, regulatory, and sales milestone payments, as well as royalties on net sales of products covered by the license.
Amicus Therapeutics and the University of Pennsylvania Announce Major Expansion of Gene Therapy Collaboration
May 29, 2019 – (Amicus Therapeutics) – Amicus Therapeutics and UPenn announced anexpansion to their collaboration, which has been expanded from three to six programs for rare genetic diseases and now includes: Pompe disease, Fabry disease, CDKL5 deficiency disorder (CDD), Niemann-Pick Type C (NPC), and MPS Types IIIA and IIIB. Terms of the agreement include a $10M annual investment from Amicus, each year for five years.
Iovance Announces New Facility to Support U.S. Production of Tumor Infiltrating Lymphocyte Cell Therapy Products
May 29, 2019 – (Iovance) – Iovance expects to invest approximately $75M over three years for equipment and construction of the manufacturing suites. The facility is expected to be completed in approximately two years.
Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele Announce Exclusive Worldwide License Agreement for the Treatment of MPS-I
May 28, 2019 – (Orchard Therapeutics) – The clinical-stage program is currently enrolling patients in an ongoing proof-of-concept study. The terms of the deal include an upfront payment in cash as well as contingent payments on the achievement of future development, regulatory and sales milestones, as well as royalty payments on net sales.
Oxford Biomedica Announces Strategic Investment by Novo Holdings A/S
May 28, 2019 – (Oxford Biomedica) – Oxford Biomedica announced that Novo Holdings agreed to invest up to £53.5M in the company in return for new ordinary shares representing up to 10.1% of the outstanding shares after the capital increase. The proceeds from the Transaction will be used to repay the existing debt facility and to further develop the LentiVector platform and the proprietary product portfolio. 
Rexgenero Successfully Expands Manufacturing of REX-001 Through Partnership with German Red Cross Blood Donor Service
May 28, 2019 – (Rexgenero) – Under a manufacturing partnership, the German Red Cross Blood Donor Service will manufacture REX-001, principally for use at additional trial sites in Germany, Poland, Czech Republic, Hungary, The Netherlands, the U.K. and Austria.
Technology Startup Kytopen Raises $3.6M Seed Round
May 28, 2019 – (Kytopen) – Kytopen, a technology startup accelerating the discovery and manufacturing of gene-modified cell therapies, today announced it has raised $3.6M in seed financing from The Engine, Horizons Ventures and angel investors.
Sensorion Announces the Signature of a Partnership Framework Agreement with Institut Pasteur on Gene Therapy Programs Targeting Hearing Loss
May 27, 2019 – (Sensorion) – This agreement defines an exclusive option to obtain licences for joint programs including Otoferlin & Usher Type 1 and preference rights on the research pipeline in the field of genetic disorders of the inner ear in order to set up future collaborations.
Medigene Announces Clinical Trial Agreement for HA-1 TCR Immunotherapy MDG1021 with University of Leiden
May 22, 2019 – (Medigene) – Medigene announced that it entered into a Clinical Trial Agreement with the Leiden University Medical Center to conduct a Phase I clinical trial with Medigene’s TCR modified T cell therapy MDG1021 targeting the HA-1 antigen. The study is scheduled to start in 2020.
BrainStorm Announces Second Clinical Site to Enroll Patients in Its Progressive MS Phase 2 Study
May 30, 2019 – (BrainStorm) – BrainStorm announced that the Stanford University School of Medicine will serve as the second contracted clinical study site for the Company’s Phase 2 trial of repeated administration of autologous MSC-NTF cells in participants with progressive MS.
Mesoblast Initiates Rolling Submission of Biologics License Application (BLA) to FDA for Remestemcel-L in the Treatment of Acute Graft Versus Host Disease
May 30, 2019 – (Mesoblast) – Remestemcel-L has received Fast Track designation for aGVHD and under this designation Mesoblast intends to request a priority review once its BLA filing is completed and accepted by the FDA. In Mesoblast’s Phase 3 trial of 55 children with aGVHD, treatment with remestemcel-L resulted in a six-month survival of 69%.
REGENXBIO Announces Completion of Dosing for Phase I/IIA Clinical Trial of RGX-314 in Wet AMD
May 30, 2019 – (REGENXBIO) – The trial includes 42 dosed subjects across five escalating dose cohorts. Topline data is expected by the end of the year.
Fibrocell Receives FDA Regenerative Medicine Advanced Therapy Designation for FCX-007 Gene Therapy for the Treatment of RDEB
May 29, 2019 – (Fibrocell) – The company expects to initiate the Phase 3 clinical trial for FCX-007 in Q2 2019, with enrollment and dosing of patients to be completed Q3 2020 and data collection for the primary endpoint to be completed in Q4 2020. Fibrocell expects to file a Biologics License Application for FCX-007 in 2021.
InVivo Therapeutics Announces Enrollment of First Two Patients into the INSPIRE 2.0 Study for the Treatment of Acute Spinal Cord Injury
May 29, 2019 – (InVivo Therapeutics) – The INSPIRE 2.0 Study is a two-arm 20-patient (10 subjects in each study arm), randomized, controlled trial designed to provide clinical data that will supplement the existing clinical results from the company’s previous single-arm study.
BioMarin Announces that Ph 3 Trial of Gene Therapy in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the U.S. and Europe
May 28, 2019 – (BioMarin) – At week 26, the estimated median Annual Bleed Rate (ABR) was zero and the estimated mean ABR was 1.5, representing a reduction of 85% from baseline levels where all patients were on standard of care prophylaxis. Seven of 16 study participants reached or exceeded the pre-specified Factor VIII levels of 40 international units per deciliter using the chromogenic substrate assay.BioMarin also announced three-year clinical data from its ongoing Phase 1/2 trial of its gene therapy for severe hemophilia A. The three-year update demonstrated that bleed rate control with valoctocogene roxaparvovec 6e13 vg/kg dose was maintained for a third year with a median Annualized Bleed Rate (ABR) of 0 and mean ABR of 0.7 in that year.
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