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Disrupting Cell Engineering with Non-Viral Modalities

Jessica Schwaber, Scientific Director, Cell & Gene Therapy, Avectas, explores her company’s unique cell engineering platform, the recent shift in cell & gene therapy commercial needs, current barriers to realizing...

Impetus Digital

- PMLiVE

AbbVie signs gene therapy deal in eye care

AbbVie and REGENXBIO have teamed up on RGX-314, a potential one-time gene therapy against two leading causes of blindness, wet AMD and diabetic retinopathy.

Realizing the Potential of Genomics-Driven Precision Medicine

Jennifer Helfer, Senior Director, Patient Advocacy & Engagement at Encoded Therapeutics Inc., discusses genomics-driven precision medicine, gene therapy misconceptions and fears, involving patients and caregivers in designing gene therapy trials,...

Impetus Digital

- PMLiVE

Researchers publish promising results for ultra-rare disease gene therapy method

Gene therapy method evaluated in seven children with aromatic amino acid decarboxylase deficiency

- PMLiVE

bluebird bio lodges counterclaim against Spark Therapeutics

Spark previously accused bluebird of misusing trademarks relating to the word ‘spark’ in marketing/educational materials

- PMLiVE

New data shows benefits for presymptomatic SMA patients treated with Zolgensma

Zolgensma was initially approved in 2019 for the treatment of SMA, launching with a list price of $2.125m

- PMLiVE

J&J’s Darzalex shows ‘significant’ survival benefit in newly diagnosed multiple myeloma

Darzalex treatment regimen reduced risk of death by 32% after nearly five years follow-up

- PMLiVE

J&J exits $1.6bn blood cancer drug licensing deal with argenx

J&J and argenx signed a deal in 2018 to develop cusatuzumab to treat haematological cancers

- PMLiVE

J&J’s single-dose COVID-19 vaccine secures approval from the UK’s MHRA

Vaccine was found to be 67% effective in preventing COVID-19 infection in a phase 3 study

- PMLiVE

Sarepta Therapeutics’ DMD gene therapy shows early success

Most patients with Duchenne muscular dystrophy require full-time use of a wheelchair by their early teens

- PMLiVE

Biogen and Capsigen to collaborate on gene therapies

Biogen will gain exclusive right for undisclosed number of CNS and neuromuscular disease targets

- PMLiVE

Pfizer pauses enrolment for trial evaluating anti-BCMA bispecific antibody

Trial pause follows reports of three cases of peripheral neuropathy in phase 1 study

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