“Landmark” genetic blindness CRISPR trial outcomes released
A Phase I/II trial has shown that a CRISPR-based gene editing therapy can be safely delivered to the retina and provide clinically meaningful outcomes.
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Alternative methods for mycobacterial testing in biological products
Considering the limited available information on implementing mycobacterial testing for quality control of biologicals, researchers have highlighted a suitable potential alternative detection method.
Combination treatment could provide new standard-of-care for aggressive lymphoma
AstraZeneca has released new clinical data demonstrating the first BTK inhibitor to show a favourable trend in overall survival versus standard-of-care chemoimmunotherapy in adults with untreated mantle cell lymphoma (MCL).
NICE recommends new haemolytic anaemia treatment
The National Institute For Health and Care Excellence (NICE)'s decision follows a reduction in the price of Pfizer’s small molecule treatment for sickle cell disease.
Novel PAT tool could improve pharmaceutical QC
The at-line PAT method utilised machine-vision and was able to predict API content of tablets containing amlodipine and valsartan with low relative errors.
MHRA publishes strategy on AI regulation
Incorporating artificial intelligence (AI) into the MHRA’s regulatory processes could help the agency focus on key priorities such as innovation.
Long-acting antibody demonstrates promising efficacy in infant RSV
New real-world evidence from a long-term follow up study adds to evidence of the high efficacy of Beyfortus in infant RSV.
AbbVie breaks ground on new R&D centre
The company’s €150 million investment in its new research and development facility will enable “cutting-edge” research to be conducted at its Ludwigshafen site in Germany long term.
Enhancing medicine manufacturing through digitalisation
Under a new initiative, manufacturers could increase productivity via innovative technologies, enabling faster patient access to new medicines.
CHMP meeting highlights: April 2024
In its April meeting, the EMA’s human medicines committee recommended eight new medicines, including treatments for cancer and autoimmune diseases.
Accelerating spectroscopic analysis for data acquisition
A study has reported the first experimental evidence of time-domain compressed sensing, which could accelerate data acquisition in ultrafast spectroscopy.
WHO proposes recommendations for preventing nitrosamine contamination
The World Health Organization (WHO)’s draft guidance on nitrosamines is applicable to manufacturers of excipients, active pharmaceutical ingredients (APIs) and finished pharmaceutical products.
Novel haemophilia B gene therapy approved
Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
Rapid method for whole-genome fungal ID
The novel technique for whole-genome analysis of fungal species is “highly suitable” for quality and safety monitoring of medicinal materials, the paper suggests.
CGT Catapult appoints new Non-Executive Directors
The new members of the Cell and Gene Therapy Catapult’s board hold expertise across therapy development, digitalisation and leadership.
