Capsida Biotherap News - LARVOL Sigma

|||||||||| SAR444836 / Sanofi
PHASE 1/2 OPEN-LABEL STUDY DESIGN TO EVALUATE SAFETY, TOLERABILITY, AND EFFICACY OF SAR444836, AN AAV-MEDIATED GENE TRANSFER IN PKU PATIENTS (Poster Room | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_914;
P1/2
Discussion/Conclusion Targeting the unmet need in the treatment of PKU, this innovative trial will investigate whether AAV gene therapy can provide a substantial treatment advantage over existing therapy. NCT number: NCT05972629 Palavras-chave : Phenylketonuria, Safety, Efficacy, Study design, AAV-mediated gene transfer

|||||||||| AAV gene therapy / Capsida Biotherap, Eli Lilly
Journal: Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques. (Pubmed Central) - Mar 6, 2024
In this nondeficient model, we did not find downregulation of endogenous AAT. However, the dual-function vector did serve as a potentially "liver-sparing" alternative for high-dose liver-mediated AAT gene replacement in the context of underlying liver disease.

|||||||||| AAV gene therapy / Capsida Biotherap, Eli Lilly
Journal: Scientific and Regulatory Policy Committee Points to Consider: Sampling, Processing, Evaluation, Interpretation, and Reporting of Test Article-Related Ganglion Pathology for Nonclinical Toxicity Studies. (Pubmed Central) - Jul 25, 2023
Reports should describe findings at these sites, any TA relationship, and the criteria used for assigning severity grades. Contextualizing adversity of ganglia findings can require a weight-of-evidence approach because morphologic changes of variable severity occur in ganglia but often are not accompanied by observable overt in-life functional alterations detectable by conventional behavioral and neurological testing techniques.

|||||||||| AAV gene therapy / Capsida Biotherap, Eli Lilly
Prediction of Adeno-Associated Virus Manufacturability by Machine Learning (Board No. 431) - May 3, 2023 - Abstract #ASGCT2023ASGCT_2433;
In the future, this model could be used as an in-silico screen for AAV capsids to reduce the time necessary for in-vitro experiments. With this work, we aim to establish an ML-based platform to increase manufacturability of clinical AAV capsids to make AAV gene therapies economically viable for patients.

|||||||||| AAV gene therapy / Capsida Biotherap, Eli Lilly
Specific Pharmacological Blockade of IRAK4 Suppresses AAV Induced Immunogenicity (Board No. 725) - Apr 21, 2023 - Abstract #ASGCT2023ASGCT_1307;
To further extend these novel findings we are testing different doses of both IRAK4 inhibitor/degrader delivered one time either as pre-treatment (prior to AAV infusion) in an in-vivo mouse model of AAV immunogenicity. Applicability of this strategy has the promise to be pan-indication, such as Huntington disease, metachromatic leukodystrophy, amyotrophic lateral sclerosis, hemophilia A, and enable successful clinical translation of AAV gene therapy.

|||||||||| AAV gene therapy / Capsida Biotherap, Eli Lilly
A Computational Framework to Detect ITR Deletion for rAAV Vectors (Board No. 889) - Apr 6, 2023 - Abstract #ASGCT2023ASGCT_728;
We also establish the need to triangulate analyses from multiple variant calling tools to conclusively confirm deletions in the ITR sequences. Identifying mutations in complex genomic regions can save substantial costs down the line and lead to more reproducible results in manufacturing AAV gene therapies.

|||||||||| AAV gene therapy / Capsida Biotherap, Eli Lilly, human Rhodopsin / Kubota
P1/2 data, Preclinical, Journal: Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis. (Pubmed Central) - Jan 20, 2023
Good laboratory practice (GLP) studies evaluated systemic biodistribution in rats and toxicology in non-human primates (NHPs). These results expanded our knowledge of dose response for an AAV5-vectored transgene under control of the human rhodopsin kinase (hGRK1) promoter in NHPs with respect to photoreceptor transduction and safety and, in combination with the rat biodistribution and mouse efficacy studies, informed the design of a first-in-human clinical study in patients with LCA1.



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