Cell & Gene Therapies

US FDA approves first non-gene edited allogeneic CAR T-cell candidate for trials

15-Aug-2018 By Flora Southey

Unlike gene-edited CAR T-cell therapies, this approach does not cut the genome to knock out, or knock in, a gene, says Celyad.

DNA damage from CRISPR/Cas9 editing could lead to cancer, finds report

10-Aug-2018 By Maggie Lynch

Gene editing with CRISPR/Cas9 can cause genetic damage to cells leading to possible cancer risks, the study reported.

CRISPR research deal will attract new business, says Oxford Genetics

09-Aug-2018 By Flora Southey

Oxford Genetics has signed a supply and licensing agreement for CRISPR engineered mammalian cell line with an unnamed ecommerce firm.

Regeneron takes $100m stake in bluebird, with ‘long-view’ on drug discovery

07-Aug-2018 By Ben Hargreaves

Regeneron and bluebird bio have announced a partnership to develop six cell therapy drug candidates.

Science Exchange collaborates with AUM Biotech for time-saving R&D tech

07-Aug-2018 By Maggie Lynch

Science Exchange announces a collaboration with Philadelphia start-up, AUM Biotech, to provide users with access to R&D technologies.

PepVax creates 'Trojan Horse' drug delivery platform

07-Aug-2018 By Maggie Lynch

PepVax has developed SMARTmid, a “Trojan Horse” drug delivery platform for DNA and RNA based treatments.

Bluebird’s ‘one-time gene therapy’ receives EMA accelerated assessment

03-Aug-2018 By Ben Hargreaves

Bluebird bio’s LentiGlobin gene therapy has received an accelerated assessment from the EMA for the treatment of transfusion-dependent β-thalassemia.

Lonza: Out of spec Kymriah ‘not a Novartis issue…it’s an industry issue’

31-Jul-2018 By Flora Southey

The CDMO is working to resolve manufacturing concerns in the “extremely new” CAR T-cell space, says Lonza CEO.

US FDA puts gene-therapy on hold after DNA fragment found in plasmids

27-Jul-2018 By Maggie Lynch

Sarepta’s gene therapy clinical trial is put on hold by the US FDA after DNA fragment was found in plasmids during quality assurance testing.

CAR-T concerns for Novartis as Kymriah identified out of spec

25-Jul-2018 By Flora Southey

Novartis is working with the US FDA to solve a manufacturing issue affecting Kymriah, identified as ‘variability in its commercial specifications’, says CEO.

PTC to acquire Agilis Biotherapeutics advancing its commitment to gene therapy

25-Jul-2018 By Maggie Lynch

PTC Therapeutics will acquire Agilis Biotherapeutics to continue work on gene therapy for rare monogenic diseases affecting the central nervous system.