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3rd In Vivo Cell Engineering & Gene Editing Summit

pharmaphorum

Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic

In-Vivo 52
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Gene editing: beyond the hype

pharmaphorum

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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Aevitas to sell sCFH asset rights to 4D Molecular

Pharmaceutical Technology

sCFH is an engineered and improved version of complement factor H (CFH) that can fit into adeno-associated virus (AAV) vectors with solid expression and applications confirmed in cultured human cells in vitro, in addition to various preclinical animal models in vivo. The firm will also receive single-digit royalties on net sales.

In-Vivo 130
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Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

Type of Gene Regulation Based on the type of transcription factor ( activators / repressors ), gene regulation has been categorized into positive and negative gene regulation. Negative Gene Regulation: Repressor is a protein molecule that initiates negative regulation.

Gene 40
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Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

WCG Clinical

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 40
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Global Roundup: Overland Pharma Launches in China to Provide Access to Breakthrough Therapies

The Pharma Data

With the launch of Overland, we are creating a robust research and development engine in parallel with the significant growth and opportunity presented by Asia’s evolving biotech industry. A solution could be at hand with Overland Pharmaceuticals. Below is a roundup of some of this week’s interesting news.

In-Vivo 52
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Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions

FDA Law Blog

Lenz, Principal Medical Device Regulation Expert — On December 23, 2021, CDRH released as a draft guidance, Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions (Draft Guidance). By Adrienne R. Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.

In-Vivo 52