Tag: Gene Therapy

Catalent’s Shopping List: Hinting Towards a One-Stop Cell & Gene Therapy CMO in Making

There appear to be no Monday blues for Catalent as the company announces the acquisition of MaSTherCell Global, a technology-focused cell and gene therapy contract development and manufacturing organization. Following its last year’s acquisition of Paragon Bioservices (a leading viral vector developer), Catalent’s recent acquisition has extended the company’s leadership in this already crowded space.

Party Like Celebrities: American Gene Technologies Hosts a Red Carpet Event to Celebrate IND Submission

On November 6, 2018, American Gene Technologies (AGT) announced that it will host a red carpet event to celebrate a significant milestone in the company’s 11-year history of scientific research in cell and gene therapy. Last month, the company successfully submitted its Investigational New Drug (IND) application to the Food & Drug Administration (FDA) to

Gene Therapy Pipeline Update: Orchard Therapeutics Presents Data on Metachromatic Leukodystrophy

Orchard Therapeutics, a commercial-stage biopharmaceutical company, announced initial results from a clinical trial (NCT03392987) with a cryopreserved formulation of OTL-200, gene therapy in development for the treatment of metachromatic leukodystrophy (MLD). Get the details of the results here. What is OTL-200: OTL-200 is autologous CD34+ cells transduced with a lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic

Gene Therapy Pipeline Update: Adverum Biotechnologies Doses First Patient for Wet AMD

Adverum Biotechnologies, a clinical-stage gene therapy company developing drugs to teated rare ocular diseases, announced that the first patient was dosed in the third cohort (n=9) of the ongoing OPTIC phase 1 clinical trial (NCT03748784) for ADVM-022 for the treatment of neovascular or wet age-related macular degeneration (wet AMD). What is ADVM-022: ADVM-022 utilizes a propriety

Gene Therapy Pipeline Update: AVROBIO Receives Orphan-Drug Designation

AVROBIO, a US-based company, developing lentiviral-based gene therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Company’s investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease. What is AVR-RD-02: AVR-RD-02 consists of the patient’s own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient

Gene Therapy Funding Update: With a Promise of $100 Million in Funding, US Government Bets Big on Gene Therapy

In a recent press announcement, The U.S. National Institutes of Health (NIH) has agreed to invest in the development of Gene Therapy in an attempt to cure HIV and sickle cell diseases. The program, for which NIH will partner with The Bill & Melinda Gates Foundation, will involve funding of $100 million each over the next

Cell and Gene Therapy Technology Update: ReNeuron Presents Positive Data

ReNeuron, the UK based, clinical-stage stem cell therapeutics company, announced that new data relating to its CTX stem cell platform will be presented today at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Dr. Steve Pells, Principal Investigator at ReNeuron, will present new data showing the phenotypic stability and scalability

Gene Therapy Pipeline Update: AGTC Presents Promising Data of its AAV Vectors for Ocular Gene Therapy

Applied Genetic Technologies Corporation (AGTC), a clinical-stage biotechnology company, developing transformational genetic therapies for patients suffering from rare and debilitating diseases, announced new data from non-clinical studies evaluating the effect of pre-existing anti-AAV antibodies on the transduction and expression efficiency of AAV vectors. According to the company’s CSO, Preexisting immunity to AAV remains a challenge for