Month: October 2019

Cell Therapy Manufacturing Update: United Therapeutics Receives Permit

As per recent news coverage, United Therapeutics has received a permit for $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinic’s Discovery and Innovation Building. What is the area of the facility:  The facility will have a total of 21,843-square-foot space. What are the capabilities at the facility:  The facility will house

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

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

Alzheimer’s Pipeline Update: Biogen Takes a U-Turn on Failed Drug; Approval in Sight

In one of the most drastic shifts in fortune in the pharmaceutical industry, Biogen has decided to submit its previously failed Alzheimer’s drug for approval. According to the company, The Phase 3 EMERGE (NCT02484547) study met its primary endpoint showing a significant reduction in clinical decline, and Biogen believes that results from a subset of patients

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,