news
Experimental gene therapy reverses sickle cell disease for three years
The gene therapy corrected the shape of some sickle cell patients' red blood cells and eliminated episodes of severe pain.
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Gene therapy
news
Gene therapy sustains bleeding control in haemophilia A trial
Giroctocogene fitelparvovec had a mean annualised bleeding rate (ABR) of just 1.4 percent over two years, with no bleeding events in the first year post infusion.
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Five marketing strategies for novel therapeutics
Long before the advent of Coronavirus, next-generation therapies were making headlines, but the successful development of COVID-19 vaccines, including mRNA vaccines, has now shone a spotlight on next-generation therapies. This offers a boost to the long-term growth projections for the advanced therapy medicinal products (ATMPs) sector. But although ATMPs hold…
news
Astellas and Dyno Therapeutics to develop AAV gene therapy vectors
Astellas and Dyno Therapeutics will develop adeno-associated virus (AAV) vectors for gene therapy directed to skeletal and cardiac muscle.
whitepaper
Whitepaper: Sterility testing – difficult products
This whitepaper considers sterility testing to support pharmacopoeial monographs with an emphasis on products that are difficult to test or validate.
whitepaper
Whitepaper: Accelerate biologics development via innovation
This whitepaper provides a roadmap for assessing and selecting the right partner to get promising biological therapies to patients quickly.
whitepaper
Whitepaper: Drug repurposing strategies to shorten timelines
This whitepaper explores drug repurposing, where known clinical candidates or commercially approved molecules are evaluated for new targets and indications.
whitepaper
Future of QA/QC for Complex Biologics Whitepaper
Download this whitepaper to discover the latest developments and research in QA/QC for biologic therapies via exclusive articles, interviews and application notes.
video
On-demand webinar: Water systems – microbial monitoring
This webinar considers the fundamental importance of water system control within pharma processing and the best control and monitoring practices.
whitepaper
Whitepaper: Impact of a pandemic outbreak on vaccine development approach
This whitepaper explores vaccine development during a pandemic, a response to complex challenges that traditional manufacturing methods will not solve.
news
Gates biomanufacturing facility announces agreement with Nkarta
The Gates Biomanufacturing Facility announced a strategic agreement with Nkarta to manufacture Nkarta's investigational cell therapy NKX101.
news
FDA joins Bespoke Gene Therapy Consortium (BGTC) for rare diseases
The consortium also consists of 10 pharmaceutical companies and five organisations to accelerate gene therapies to treat rare diseases.
whitepaper
Case study: Delivering a world class cell and gene therapy suite
RoslinCT and Connect 2 Cleanrooms have worked in partnership to deliver a world-class cell and gene therapy facility.
webinar
Simplifying oligonucleotide characterisation with Microchip CE-MS
18 October 2021 | By 908Devices
In this on-demand webinar, 908 Devices present a microfluidic capillary zone electrophoresis-based separation coupled with HRAM mass-spectrometry for rapid characterisation of oligos in positive ionisation mode.
video
On-demand webinar: Spores in my cleanroom
This webinar considers the problems bacterial and fungal spores present for contamination control and remediation and disinfection strategies.
