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What new developments are worth paying attention to at the opening of the gene and cell therapy industry conference today?

 Last Update: 2022-08-15
 Source: Internet
 Author: User

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Today, the 25th annual meeting of the American Society for Gene and Cell Therapy (ASGCT) open.

AskBio Announces Results of First Clinical Trial of Gene Therapy for Heart Failure

Asklepios BioPharmaceutical has announced results from its first-in-human clinical trial of its gene therapy for heart failure based on the innovative adeno-associated viral vector AAV2i The AAV2i8 vector is an AAV vector that targets the heart but not the liv.

Preliminary published clinical trial results showed that no adverse events related to the investigational therapy were observed during the 12-month observation period in three heart failure patients treated with gene thera.

Image source: Reference [3]

Completing multiple gene editing steps in one step, non-viral gene editing platform shows potential

PACT Pharma has announced the latest data on its non-viral precision gene editing technology PACT^.

▲Introduction to PACT^NV technology platform (Image source: PACT Pharma's official website)

Data presented at the ASGCT Annual Meeting showed that this system not only knocked out the expression of endogenous TCRs that target patient neoantigens, but also knocked out the gene that expresses TGF-beta receptorThis means that the resulting T-cell therapy not only specifically recognizes the patient's tumor, but is also resistant to TGF-β-mediated immunosuppressive signali.

Rocket Pharmaceuticals' three gene therapies make positive progress

Rocket Pharmaceuticals announced that its gene therapy RP-L102 for the treatment of Fanconi Anemia has achieved positive results in a pivotal Phase 2 clinical tri.

RP-L102 is a gene therapy that uses lentiviral vector to deliver normal FANCA gene to transform hematopoietic stem cells in vit.

In addition, Rocket Pharmaceuticals' gene therapy for Danon Disease and pyruvate kinase deficiency has also shown a favorable safety profile in Phase 1 clinical tria.

Knock down the target protein expression by 98%, and the homing endonuclease genome editing technology shows its potential

Precision BioSciences presented results from multiple preclinical studies of its genome editing technology platform called ARCUS at the ASGCT Annual Meeti.

Image source: Precision BioSciences official website

In a non-human primate model, the company's optimized ARCUS nuclease can knock down the expression level of the target protein HAO1 by 98%, showing its efficient in vivo gene editing abili.

In addition, the company's gene editing therapy for HBV clearance effectively targets and degrades HBV covalently closed circular DNA (cccDNA) in primary human hepatocytes and reduces HBV surface antigen (HBsAg) expression by 7

Gene therapy expresses therapeutic antibody, gains proof-of-concept for brain metastases

Voyager Therapeutics announced preclinical proof-of-concept data for its anti-HER2 antibody-expressing gene therapy VCAP-10 The therapy uses its TRACER AAV9 vector to deliver a transgene encoding an anti-HER2 antibo.

In three different mouse models of HER2-positive breast cancer, a single VCAP-102 treatment significantly reduced CNS tumor burden and prolonged animal li.

References:

[1] AskBio and you shall receive as the Bayer-backed gene therapy maker touts early phase 1 da.

[2] AskBio to Present 11 Abstracts at Upcoming American Society of Gene and Cell Therapy's 25th Annual Meeti.

[3] A First-in-Human Phase 1 Clinical Gene Therapy Trial for the Treatment of Heart Failure Using a Novel Re-Engineered Adeno-Associated Vect.

Retrieved May 16, 2022, from https://annualmeeti.

asg.

org/abstracts /abstract-details?abstractId=6156

[4] PACT Pharma Highlights Capabilities and Versatility of Novel Non-Viral Gene Editing Technology in Presentation at American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeti.

Retrieved May 16, 2022, from https:// /news-releases/pact-pharma-highlights-capabilities-and-versatility-of-novel-non-viral-gene-editing-technology-in-presentation-at-american-society-of-gene--cell-therapy- asgct-25th-annual-meeting-30154727html

[5] A Versatile, Non-Viral Gene Editing Method for Directing Specificity and Enhancing Function of T Cel.

Retrieved May 16, 2022, from https://stora.

googleap.

com/pact-assets/ASGCT-2022-Lu/ASGCT -2022-.

pdf

[6] Precision BioSciences Announces Preclinical Data Showcasing Premier In Vivo Gene Editing Capabilities at American Society of Gene & Cell Therapy Annual Meeti.

Retrieved May 16, 2022, from https://invest.

precisionbioscienc.

com/news-releases/news-release -details/precision-biosciences-announces-preclinical-data-showcasing

[7] Voyager Therapeutics to Present Preclinical Data from its Vectorized anti-HER2 Antibody Program and a Novel AAV5-Derived TRACER™ Capsid at the 25th American Society of Gene and Cell Therapy Annual Meeti.

Retrieved May 16, 2022, from https:// .

voyagertherapeuti.

com/news-releases/news-release-details/voyager-therapeutics-present-preclinical-data-its-vectorized

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