Register today for the upcoming RMIP Research Spotlight on Thursday, March 20th at 12:00-1:00 PM ET. This meeting will feature Dr. Graça Almeida-Porada of Wake Forest University and the Wake Forest Institute for Regenerative Medicine.
Abstract: Current hemophilia A (HA) treatments, relying on frequent FVIII infusions, face challenges including high cost, limited global access, and inhibitor development. Prenatal therapy (PNTx) offers a potential cure by circumventing immune barriers and inducing FVIII tolerance. Large animal studies have demonstrated sustained FVIII expression using human placental cells (PLCs) transduced with a lentiviral vector encoding a bioengineered FVIII transgene (mcoET3). In this research spotlight we present results from a preclinical study to manufacture, characterize, and authenticate a gene-modified PLC-based hemophilia A therapeutic product, and confirm therapeutic potential and safety in mouse and large animal PNTx models.
We hope that you will be able to join us for this spotlight.
Recent Publications
Congratulations to the following study teams for their recent publications or preprints:
N.A. Hyams, … Y. Mei. Improving human cardiac organoid design using transcriptomics. Sci Rep. 2024 Aug 30;14(1):20147. doi:10.1038/s41598-024-61554-w
S.S. Lee and L.A. Garza Volar Fibroblasts and their Potential Therapeutic Use. J Invest Dermatol. 2024 Dec 24:S0022-202X(24)02965-8. doi:10.1016/j.jid.2024.10.614.
Meet with us
BDC now has office hours each Wednesday from 2:00 to 3:00 PM ET to assist investigators with aggregating and harmonizing RMIP study data on the ecosystem.
We continue to coordinate one-on-one meetings with study teams to discuss a data linkage strategy. This strategy will ensure that the cell characterization data is prepared and formatted appropriately for seamless sharing with the study team. The goal is to facilitate efficient data transfer and analysis.
Mike Enger
March 2025 RMIP Updates
RMIP Research Spotlight
Register today for the upcoming RMIP Research Spotlight on Thursday, March 20th at 12:00-1:00 PM ET. This meeting will feature Dr. Graça Almeida-Porada of Wake Forest University and the Wake Forest Institute for Regenerative Medicine.
Abstract: Current hemophilia A (HA) treatments, relying on frequent FVIII infusions, face challenges including high cost, limited global access, and inhibitor development. Prenatal therapy (PNTx) offers a potential cure by circumventing immune barriers and inducing FVIII tolerance. Large animal studies have demonstrated sustained FVIII expression using human placental cells (PLCs) transduced with a lentiviral vector encoding a bioengineered FVIII transgene (mcoET3). In this research spotlight we present results from a preclinical study to manufacture, characterize, and authenticate a gene-modified PLC-based hemophilia A therapeutic product, and confirm therapeutic potential and safety in mouse and large animal PNTx models.
We hope that you will be able to join us for this spotlight.
Recent Publications
Congratulations to the following study teams for their recent publications or preprints:
Meet with us
BDC now has office hours each Wednesday from 2:00 to 3:00 PM ET to assist investigators with aggregating and harmonizing RMIP study data on the ecosystem.
We continue to coordinate one-on-one meetings with study teams to discuss a data linkage strategy. This strategy will ensure that the cell characterization data is prepared and formatted appropriately for seamless sharing with the study team. The goal is to facilitate efficient data transfer and analysis.
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