Cell & gene therapies

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Urgent action needed to reduce high costs of gene therapies

By Jane Byrne

The extremely high costs of gene therapies are unsustainable, and a global commitment to affordable, equitable access to these treatments is urgently needed, concluded the organising committee of a conference on human genome editing.

Pic:getty/antoniosolano

Syena sets out to pioneer TCR NK cell therapy

By Rachel Arthur

New company Syena says it has the potential to create the next generation of cell therapy: combining the safety, potency and scalability of natural killer (NK) cells with the ability of T cell receptors (TCR) to target intracellular tumor antigens.

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Lonza: Robust industry fundamentals are supportive of growth

By Jane Byrne

Despite an uncertain macroeconomic environment, Lonza says its ‘resilient’ business model and sustained market demand delivered a strong financial performance in 2022, in line with the Swizterland based CDMO’s outlook.

© GettyImages/AlexanderFord

Opus acquires two compounds for inherited retinal diseases

By Jane Byrne

Last week saw Opus Genetics announce it had acquired the rights to two preclinical-stage adeno-associated virus (AAV)-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio.

Pic:getty/markrubens

BioNTech acquires GMP manufacturing site in Singapore

By Rachel Arthur

BioNTech will create an mRNA facility in Singapore through the acquisition of a Novartis site: which will become its Regional Headquarters and production center for the Asia Pacific region.

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Lonza expands team, labs for cell and gene development

By Jane Byrne

Lonza is to increase the size of the cell and gene therapy (CGT) development space at its Houston facility; the expansion will include viral vector-based process development and analytical development laboratories.

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Syncona to acquire Applied Genetic Technologies Corporation

By Rachel Arthur

London investment fund Syncona will acquire retinal gene therapy company Applied Genetic Technologies Corporation (AGTC): with AGTC noting it had been facing ‘significant challenges’ in funding operations beyond 2022.

Melbourne wants to become an mRNA hub, with investments from both BioNTech & Moderna. Pic: getty/walterbibikow

BioNTech to build mRNA center in Australia

By Rachel Arthur

BioNTech will establish an mRNA research and innovation center in Victoria, alongside clinical scale manufacturing capabilities. Meanwhile, it has also pledged to step up clinical development of oncology therapies in the country.

Pic:getty/MG

EMA pilot to provide regulatory support to selected ATMP developers

By Rachel Arthur

The European Medicines Agency (EMA) will provide up to five selected advanced therapy medicinal products (ATMPs) with enhanced regulatory support - and in the process hopes to learn how to improve the regulatory processes for other ATMP drugs in the future.

Pic:getty/sewcream

Rocket Pharmaceuticals to acquire Renovacor

By Rachel Arthur

Rocket Pharmaceuticals, a late-stage, clinical biotech advancing a pipeline of genetic therapies for rare childhood disorders, will acquire Renovacor: helping it strengthen its position in AAV-based cardiac gene therapy.

Pic:getty/minadelao

bluebird bio gets green light for Skysona gene therapy in the US

By Rachel Arthur

The US Food and Drug Administration (FDA) has granted Accelerated Approval for bluebird bio’s Skysona (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral...