Tag: Gene Therapy

Cell and Gene Therapy Manufacturing: COVID-19 Impact

With close to half-million infections and the economy showing signs of the 2008 financial crisis, COVID-19 has impacted several industries. Government institutes and industry players have announced various steps to deal with the ongoing situation. In this article, we will highlight the developments in the cell and gene therapy manufacturing industry amid the ongoing COVID-19

Gene Therapy Companies: Impact on COVID-19

In a recent press release, bluebird bio, one of the leading gene therapy companies, announced that COVID-19 has delayed the EU launch and US filing of the company’s gene therapy candidate Zynteglo (LentiGlobin). The company now expects to dose the first commercial patient with Zynteglo in Germany in the second half of 2020. Earlier, the company had

Cell Therapy Manufacturing: Here are the Key Innovations

Last week, during our weekly industry update sessions, our prime focus was to understand the recent innovations in the field of cell therapy manufacturing. We were also looking out for players that are driving these innovations. To start with, the first news that caught our attention was Novartis’ decision to sidestep Europe travel ban to

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: Rocket Pharmaceuticals Receives IND Clearance for Gene Therapy for Pyruvate Kinase Deficiency

Rocket Pharmaceuticals, a New York-based, clinical-stage company, developing first-in-class gene therapies for rare and devastating inherited diseases announced clearance of Investigational New Drug (IND) by the FDA for RP-L301 gene therapy. RP-L301 is a lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a genetic rare disease. Last month, the company received

Gene Therapy Pipeline Update: Homology Medicines Presents Data Demonstrating Preclinical Proof-of-Concept

Homology Medicines, a company using proprietary gene editing and gene therapy platform announced the presentation of preclinical data supporting its investigational gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics (ASHG) 2019 Meeting. The presented preclinical data showed the results from studies conducted in

Cell Therapy Manufacturing Update: Merck Becomes the First to Introduce Acoustic Technology

With its acquisition of FloDesign Sonics earlier this month, Merck/MilliporeSigma has become the first company to introduce acoustic technology in cell therapy manufacturing. The acquisition of FloDesign Sonics is a strategic fit for Merck, strengthening its ability alongside to manufacture cell-based therapies for patients. What is Acoustic Cell Processing: Acoustic cell processing is a disruptive technology that allows

Plasmid DNA Manufacturing Market Update: Cytovance®Introduces Integrated Single-Use Platform

In the ongoing shift of biologics manufacturers to single-use systems, Cytovance®, a leading biopharmaceutical CDMO, announced the addition of an integrated single-use platform for the manufacturing of plasmid DNA (pDNA) at their facilities in Oklahoma City, OK. According to the company, two grades are available (Critical Reagent Grade™, CGMP-grade) in lot sizes from 1g to 50g,

Gene Therapy Market Update: Researchers Deliver a Cure for Epidermolysis Bullosa (EB)

In a recent news update, a team of Irish gene therapy researchers announced a breakthrough in the search for a cure for one of the worst strands of the incredibly painful skin disease, EB (epidermolysis bullosa). According to the researchers, the incredibly painful genetic condition, which affects approximately 300 people in Ireland, is caused by a

Gene Therapy Market: Big Pharma Bets Big

Last week, I came across an interesting article by Andrew Dunn titled Sanofi investing in gene therapy as R&D focus turns toward rare disease. The article caught my attention because in June, this year, Sanofi scaled back its four-year-old gene therapy alliance with Voyager Therapeutics, for the second time. If we just look at the news titles,

Vector Manufacturing: Growing Cell and Gene Therapy Market

Cell and Gene Therapy CROs are the new entrants in the pharma Superstar Club. They are selling tickets faster than a Queen’s concert in the 1980s. In fact, as per the Alliance of Regenerative Medicine’s recent findings, there has been more than 75% year on year increment in funding to support the development of various cell and gene

The Rise of Cell and Gene Therapy CRO

Over 500 cell and gene therapy candidates are being investigated in different stages of clinical development across various geographies. In fact, in the past five years alone, more than 1,000 clinical trials, that are currently active, were initiated to evaluate the potential of these cell and gene therapies across multiple therapeutic indications. Due to several

Cancer Stem Cell: A Step Closer to the Cure

The discovery of cancer stem cell (CSC) may have revolutionized the field of cancer research in a manner similar to how the “germ theory of disease” altered the way medical science looked maladies in general.  Before the germ theory, disease and sicknesses were attributed to be manifestations of bad omens and evil spirits. The discovery of