Functional Skills
Software Skills
Sector Experience
Experience
-Focus areas include delivery and gene editing approaches to develop cell therapies, methods to differentiate iPS cells to specific target cells to treat a number of diseases with high unmet medical need, reviewing non-confidential PowerPoint decks containing pitches for a new biotech startup.
-Developed and established a novel beta cell differentiation process that generates 99%+ pure endocrine fate and scaled up the process from spinner vessels to 1L and 3L STR bioreactors. Signed inventor disclosures to file two patents.
-Implemented DoE approaches across the company to rapidly optimize the differentiation of hPSCs into functional beta cells (SC451 program) and other target cell types.
-Partnered cross-functionally to establish an RNA sequence platform within the team for a more robust QC of the developing drug product.
-Established a process development/scale-up lab from the ground up, including room design, equipment selection, and hiring of FTEs.
-Increased beta-cell yields up to 4-fold, determining the probability of failure at each stage of differentiation and providing proof of function in vitro and in vivo.
-Effectively devised approaches to increase current CRISPR/Cas9 HDR-mediated knock-in efficiencies in hPSCs up to 150-fold.
-Collaborated with the Duchenne Muscular Dystrophy team to help them gene edit hPSCs and differentiate them into cardiomyocytes for disease modeling purposes.
●Developed a novel transdifferentiation vector to generate cortical neurons rapidly and efficiently from hPSCs for the Neuro team.
-Spearheaded scaling up the differentiation of hPSCs into pancreatic progenitors from 100 ml to 3L bioreactor systems.
-Led effective team meetings with counterparts from our CRISPRTx-Viacyte collaboration (VCTX210) to find target areas of synergy to move our program forward faster. VCTX210 is now in clinical trials.
-Effectively devised approaches to increase current CRISPR/Cas9 HDR-mediated knock-in efficiencies in hPSCs up to 150-fold.
-Collaborated with the Duchenne Muscular Dystrophy team to help them gene edit hPSCs and differentiate them into cardiomyocytes for disease modeling purposes.
●Developed a novel transdifferentiation vector to generate cortical neurons rapidly and efficiently from hPSCs for the Neuro team.
-Spearheaded scaling up the differentiation of hPSCs into pancreatic progenitors from 100 ml to 3L bioreactor systems.
-Led effective team meetings with counterparts from our CRISPRTx-Viacyte collaboration (VCTX210) to find target areas of synergy to move our program forward faster. VCTX210 is now in clinical trials.
•Established bioreactor-based retinal and brain organoid differentiation protocols for the disease modeling of Spinocerebellar Ataxia 7 and Parkinson's disease.
•Implemented a novel transdifferentiation process to rapidly and efficiently generate cortical neurons from hPSCs.
•Supervised two research associates, one senior and one junior, to ensure optimum work performance and results.
-Established a novel 3D culture organoid differentiation protocol using bioreactors that proved to dramatically improve photoreceptor outer-segment formation and increase rod photoreceptor yield by 6-fold compared to static differentiation protocols.
-Designed and 3D printed in biocompatible material a scaffold for the co-culture of human RPE and photoreceptors in a bioreactor environment.
-Generated RPGR mutant patient derived retinal organoids to test the efficacy of a AAV2/5-hRKp.RPGR clinical vector to rescue the disease in vitro. Here, AAV transduction led to the restoration of full length RPGR protein and poly-glutamylated tubulin at the photoreceptor connecting cilia in a dose-dependent manner. This vector is now in clinical trials in partnership with MeiraGTx.