[1/10/2025] Gene and Cell Therapy- weekly digest from PackGene

Jan 10 , 2025
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Featured

Jan 08 First-ever gene edit of infant boy leads to apparent cure
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Jan 07 Novartis gene therapy poised to expand SMA treatment landscape
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Jan 07 Vertex pays Orna $65M to work on next-gen gene therapies for blood disorders
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News

Jan 08 Resilience cuts 120 employees at a former bluebird gene therapy site
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Jan 08 Dx&Vx, From mRNA Vaccines to Nanoparticle Vaccines era of Next-Generation Vaccines
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Jan 07 Global Market for Cell and Gene Therapy Tools and Reagents Projected to Grow at a 10.8% CAGR
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Jan 03 Seven Biopharma Trends to Watch in 2025
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Jan 02 Regeneron snaps up eye biotech, snaring science but not staff
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Jan 02 A surge in deals: GSK bets on ADC, neuro and fibrotic diseases to bolster drug development
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Articles

Jan 08 Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo
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Jan 08 A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD
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Jan 07 High frequency CCR5 editing in human hematopoietic stem progenitor cells protects xenograft mice from HIV infection
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Jan 07 DNA targeting by compact Cas9d and its resurrected ancestor
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Jan 06 The N1‐methyladenosine methyltransferase TRMT61A promotes bladder cancer progression and is targetable by small molecule compounds
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Jan 02 Rational engineering of minimally immunogenic nucleases for gene therapy
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Jan 01 A Novel Competing Endogenous RNA Network Reveals Potential Mechanisms and Biomarkers of Chemoresistance in Lung Adenocarcinoma
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About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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