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Novartis warns of two patients deaths after dosing with gene therapy Zolgensma

MedCity News

Liver complications are a known side effect risk of Zolgensma and other genetic medicines that are delivered by engineered viruses. The two deaths reported following treatment with Novartis’s Zolgensma mark the first fatal cases of acute liver failure associated with the gene therapy.

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“Universal” CRISPR-edited T cells improve resistant leukaemia

European Pharmaceutical Review

Researchers at Great Ormond Street Hospital for Children (GOSH) and University College London (UCL) Great Ormond Street Institute of Child Health (UCL GOS ICH) have used “universal” CRISPR-edited cells in humans for the first time to treat B-cell acute lymphoblastic leukaemia (B-ALL) child patients with engineered donor T cells.

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Tackling immune-mediated disease with CAR Tregs

European Pharmaceutical Review

In this article, Sangamo Therapeutics’ Chief Scientific Officer, Jason Fortenot, explains why CAR Tregs may become transformational therapeutics for immune-mediated diseases, highlighting the challenges his company has overcome to get the first ever engineered Treg therapy into clinical trials. How do CAR Tregs differ from T cells?

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Bottlebrush Nanoparticles Deliver Immunostimulatory Drugs

Medgadget

These drugs are intended to stimulate immune cells in the vicinity of tumors to begin destroying tumor cells, but when delivered systemically they cause unacceptable dose limiting side-effects such as systemic inflammation. These particles are bottle-brush shaped, with the inactive drug being conjugated to the “brush backbone”.

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Soft Bioresorbable Implant Controls Pain by Cooling Nerves

Medgadget

A team of engineers at Northwestern University led by John Rogers, the person responsible for many advances in flexible electronics, created a drug-free implant that can control pain by cooling nerves. As a drug-free technology, the implant could help to avoid the addiction and side-effects that opioids frequently cause.

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Synlogic preps phenylketonuria drug for phase 3 after mid-stage win

pharmaphorum

Drug treatment is an option for some patients, with BioMarin’s enzyme-based therapies Kuvan (sapropterin) or Palynziq (pegvaliase) both approved to reduce levels of the amino acid, although they only work in a minority of patients and, in the case of Palynziq, can have serious side effects.

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Maximizing ROI in Pharma: The Power of Display Advertising for Targeted Campaigns

Pharma Marketing Network

Include required disclaimers and side effect warnings. Display ads are visually based and appear on websites, while search ads are text-based and appear in search engine results. Are display ads effective for promoting branded drugs? Regulatory Challenges and Solutions Pharma marketers face strict advertising regulations.

Pharma 52