Tag: Gene Therapy

Over the last decade, one of the major challenges faced by pharmaceutical companies across the globe is low drug solubility. In fact, it has been observed that around 40% of the pharmaceutical drugs approved by regulatory organizations exhibit poor bioavailability / solubility. Further, every year, poor bioavailability is one of the main causes of drug

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. Presently, over 250 companies are engaged in the gene therapy pipeline, the gene

The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. coli bacteria, as well as lambda bacteriophage, can adapt to the alterations in the composition of their nutrient medium. Molecular switch is a site on genes where regulatory molecules can bind to trigger transcription process,

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

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

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

There appear to be no Monday blues for Catalents 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), Recent acquisition by Catalent in Gene Therapy segment has extended the company’s leadership

As we enter 2020, one drug class that is set to take-off is gene therapy. With three approvals (Zolgensma® (US), Zynteglo™ (Europe) and Beperminogene Perplasmid (Japan), 2019 was a break out year for gene therapy developers. The $16 billion-plus investment by various investors finally seems to be paying off as we enter 2020 with a

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

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

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

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

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

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

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

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

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

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,

The gene therapy company, Exegenesis Bio has become one of the recent players to receive funding in the therapy space. According to a media statement, the company has completed fundraising of over 70 million yuan ($10 million), led by K2 Venture Partners and joined by Kaitai Capital and Legend Star. The company was founded by

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

From being a thing of science fiction to topping the priority list of every big pharma player, gene therapies have come a long way. With multiple approved products, the field of gene therapies has gained substantial momentum over the last couple of decades. The growing burden of genetic disorders and the rising evidence of gene

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,

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

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

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