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The future of CNS drug development: signs of real progress

Drug Target Review

Due to off-target effects, tissue accumulation and immune responses, ASOs face significant challenges, especially for preclinical safety assessments. However, they face challenges in surviving the hostile CNS environment, integrating into existing neural circuits and avoiding immune responses; but they are rich in potential.

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A Visual Guide to Gene Delivery

Codon

And even if all these economic hurdles are overcome, there are still three main technical challenges to solve: cargo capacity, specificity, and immune response. The human immune system is designed to recognize and eliminate foreign material, which unfortunately includes common viral vectors for gene therapy.

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Roche shares Elevidys safety update in non-ambulatory Duchenne muscular dystrophy patients

The Pharma Data

Elevidys utilizes adeno-associated virus vector technology, which is a powerful delivery platform but carries inherent risks related to the delivery process and the patient’s own immune response.

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A Visual Guide to Genome Editors

Codon

” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. .” While crossing Trafalgar Square, Gray paused briefly to reflect on her experience.

DNA
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Why brain cancer is often resistant to immunotherapy

Broad Institute

So the researchers used a method developed at the Broad Institute in 2019 called consensus non-negative matrix factorization (cNMF) , which can define cells by their identity and activity independently. With this approach, the team identified four programs shaping the immune system.

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Lilly and Kumquat Biosciences Announce Collaboration to Discover and Develop Novel Small Molecules that Stimulate Tumor-Specific Immune Responses

The Pharma Data

Loxo Oncology at Lilly, a research and development group of Eli Lilly and Company (NYSE: LLY), and Kumquat Biosciences today announced an exclusive collaboration focused on the discovery, development and commercialization of potential novel small molecules that stimulate tumor-specific immune responses.

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Advancing vaccine design: potential of peptide mimicry

Drug Target Review

Since December 2019, SARS-CoV-2 (COVID-19) infection has become a worldwide urgent public health concern. 1 The study reveals a novel bioinformatic approach and tool that holds the potential to empower researchers in designing vaccines capable of inducing a stronger immune response.