news
First patient treated with potentially curative HIV cell and gene therapy
The first-in-human study will evaluate the safety and efficacy AGT103-T, a cell and gene therapy product for HIV, in up to six participants.
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Gene therapy
webinar
Variable pathlength spectroscopy for dilution-free concentration measurement in GMP environments
27 May 2021 | By Repligen
Watch this on-demand webinar, where we reviewed VPT and how it can be used in various applications, from protein therapeutics to gene therapy.
news
Industry hustling to ensure supply of recombinant vector COVID-19 vaccines
A new report suggests pharma is working hard to combat worldwide viral vectors shortage that could threaten the COVID-19 vaccine rollout.
news
EMA’s human medicines committee (CHMP) meeting highlights, May 2021
In its latest meeting the CHMP recommended eight medications for approval, 17 medicines for indication extension and concluded reviews of two COVID-19 treatments.
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Curative cell and gene therapies and healthcare system disruption
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
news
First UK patient enrolled in Duchenne muscular dystrophy gene therapy trial
The Phase III trial will evaluate the safety and efficacy of Pfizer’s PF-06939926 gene therapy in 99 paediatric Duchenne muscular dystrophy patients across 15 countries.
news
Gene therapy successfully treats rare immunodeficiency in paediatric patients
In three Phase I/II trials an experimental gene therapy caused all but two patients with severe combined immunodeficiency to acquire and retain robust immune function for more than two years.
whitepaper
Whitepaper: Analytical testing considerations for ATMPs
This whitepaper discusses the analytical testing considerations for complex biological materials in ATMPs.
article
Cleanroom infrastructure innovation to fulfil the needs of cell and gene therapies
Biopharmaceuticals have transformed the patient care landscape, but alongside this step change in treatment capability comes the need for a similar paradigm shift in manufacturing capability. Here, Maik W Jornitz, President & CEO of G-CON Manufacturing Inc. highlights how cleanroom infrastructure must develop to keep pace with ever evolving industry needs.
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Bioprocessing & Bioproduction In-Depth Focus 2021
Featured in this in-depth focus, articles exploring why cleanroom infrastructure must transform to meet the needs of the cell and gene therapy industry and discussing a novel platform for discovering and developing genetically-targeted therapies for autoimmune diseases.
news
Novel blindness gene therapy shows promise in first-in-human trial
The lowest dose of the gene therapy was shown to be safe and somewhat improved vision in the first three patients treated in the trial.
news
Rocket releases positive results from Leukocyte Adhesion Deficiency-I trial
Interim Phase I/II trial results show Rocket’s ex vivo lentiviral gene therapy candidate, RP-L201, is safe and of benefit to paediatric patients with severe Leukocyte Adhesion Deficiency-I.
video
On-demand webinar: Optimising MALDI-TOF for filamentous fungi ID
Challenges and solutions to adopting MALDI technology for filamentous fungi ID with emphasis on sample preparation and database development.
webinar
Quality Risk Management tools to improve data integrity in micro QC
12 April 2021 | By Charles River Laboratories
Watch this on-demand webinar with Jordi Iglesias to learn how you can improve the data integrity of your microbiological QC assays.
whitepaper
Brochure: Integrated bioprocessing
The journey from large molecule to approved biotherapeutic is long, costly, complex, and fraught with risk – yet rewards for success are substantial.
