Category REGENERATIVE MEDICINE

bluebird bio Announces EU Conditional Marketing Authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype

ZYNTEGLO is the first gene therapy approved for transfusion-dependent β-thalassemia (TDT) European marketing authorization for ZYNTEGLO follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program ZYNTEGLO is bluebird bio’s first gene therapy to gain regulatory approval bluebird bio, Inc. announced today that the European Commission (EC)

Read More


Research sheds light on new ways to target autoimmune disorders

Novel molecules developed by researchers at the Rockefeller University (NY, USA) have the potential to provide treatments for the millions of people who suffer from an autoimmune disorder. Reported recently in Nature Communications, Thomas Tuschl (Professor at Rockefeller University; NY, USA) and his fellow researchers from have developed small molecules that can bind to and inhibit one

Read More


Regulatory intelligence: Update on regenerative medicine advanced therapies designations

This article discusses the scope and purpose of the special designation for Regenerative Medicine Advanced Therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT, such as keeping the US globally competitive in the field. They provide a tally of products receiving the special

Read More


Using CAR T-cell Therapy in Older Patients With Aggressive Lymphoma

Article Highlights CAR T-cell therapy is gaining traction in relapsed/refractory lymphoma in older patients with aggressive disease. As a result of prospective trials investigating CAR T-cell therapy, there is potential that older patients with lymphoma can be spared multiple lines of chemotherapy and its resultant toxicities while still having long-term clinical benefit. Toxicities are still

Read More


Advanced Therapies News

Source ARM Organovo Collaborates With MCRI and Leiden University Medical Center to Develop Stem Cell-Based Bioprinted Tissue Treatments for Kidney DiseaseMay 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

Read More


Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele Announce Exclusive Worldwide License Agreement for the Treatment of MPS-I

Encouraging Clinical Data Generated from Ongoing Proof-of-Concept Study Conducted by the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget); Enrollment Expected to Complete in the First Half of 2020 Leverages Orchard’s Expertise in Treating Neurometabolic Diseases Using its Investigational Autologous Hematopoietic Stem Cell Gene Therapy Builds Upon Orchard / SR-Tiget Partnership to Develop Novel Gene Therapy Candidates Orchard Therapeutics, Fondazione

Read More


Dr. Preti Discusses Three-Tier Strategy for Cell Therapy with Global Business Reports

Dr. Robert A. Preti, theCEO and President of Hitachi Chemical Advanced Therapeutics Solutions (HCATS), was recently interviewed by Global Business Reports (GBR) for their 2019 US Biopharmaceuticals report. The GBR report covers a range of topics, such as the state of the biopharma industry, new developments in biopharmaceutical manufacturing, the investment climate for cell and gene

Read More