• Regulatory

FDA accepts Drug Master File, signifying a new milestone for Touchlight’s enzymatic doggybone DNA technology

Touchlight today announced a ground-breaking new milestone for its enzymatic doggybone DNA (dbDNA™), following the FDA acceptance of the Drug Master File (DMF) for doggybone DNA technology. doggybone DNA is the first enzymatic DNA platform with a DMF, representing a significant step towards regulatory adoption. The DMF simplifies the U.S. regulatory process for utilising doggybone DNA and accelerates regulatory filings.

CEO Update | 30 January 2023

The end of January is always incredibly busy at the BIA, with our first Board meeting of the year welcoming newly elected members, the publication of our annual report into the financing of the sector and of course our Gala Dinner which was a great evening last week. Read more on R&D tax credit campaign at centre of finance work, the Chancellors’ growth speech and more.

CEO Update | 23 January 2023

This week is one of the busiest in the BIA calendar as we host our much looked-forward-to Gala Dinner, get ready to publish last year's finance figures for the sector and gather our new Board for the first time in 2023. More on all this in next week’s Newscast. I look forward to seeing over 600 of you on Thursday night at the Brewery. Read on for AMR national action plan and how CGI blockbuster, Avatar, is in fact important life science work.

CEO Update | 9 January 2023

I write this week from San Francisco where the JPM conference is now well underway – with full participation from the UK ecosystem. Folk are already talking about  the announcement of BioNtech’s MoU with the UK Government to develop next generation cancer vaccines in partnership. Also demonstrating the UK’s capability in accelerated innovation is the news from RQ Bio and AstraZeneca that the first of RQ’s monoclonal antibodies to prevent COVID-19 in vulnerable patients, which originates from work of the BIA Antibody Taskforce, has entered clinical trials less than 12 months after discovery, with the start of AZ’s SUPERNOVA Phase I/III trial.

CEO Update | 19 December 2022

Reflections on 2022 and look forward to 2023. Our life science sector is continuing to deliver world-changing products and services at a pace and scale that will transform lives in the years to come. And whether the breakthrough highlighted be next-generation products for type 2 diabetes, novel malaria vaccines, new developments in mRNA vaccinology or cell and gene therapy for the early diagnosis of cancer, I see the UK innovation ecosystem engaged and involved in world-leading programmes.

Role of patient advocacy organisations in regulatory best practice

The most recent FDA guidance on the use of real-world data in regulatory decision-making is fundamental for patient advocacy organizations, ensuring that the FDA endorses drugs or biological products that lead to proper health outcomes without the risk of life-threatening side effects.

Boyds bolsters regulatory affairs team with key appointment

Leading global drug development consultancy Boyds has appointed experienced regulatory affairs professional and cell and gene therapy expert Dr Patrick Ginty.

MHRA Innovation Passport for Complement Therapeutics Geographic Atrophy Gene Therapy

Complement Therapeutics Ltd (CTx), a biotechnology company developing novel therapeutics for complement-related diseases, today announced its lead asset CTx001, an AAV-based gene therapy for the treatment of Geographic Atrophy (GA), has been awarded an Innovation Passport by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA).

Cross-country HTA of gene therapy Libmeldy calls for price cut

Joint price negotiations for Orchard’s gene therapy Libmeldy could follow outcome of multi-country health technology assessment.

SiSaf’s innovative RNA therapeutic for rare genetic skeletal disorders begins the U.S. regulatory process

SiSaf Ltd, an RNA delivery and therapeutics company, announces that it is initiating the U.S. FDA Regulatory process to obtain an Orphan Drug Designation for SIS-101-ADO, a siRNA therapeutic for patients with Autosomal Dominant Osteopetrosis Type 2 (ADO2), a rare genetic skeletal disorder.