STRATEGY & ROADMAP

Care4ASH1L believes that research is at the core and integral for ASH1L community. We not only solicit researchers for ideas but also actively identify projects which focus on novel therapy development. We constantly look for experts and hope to coordinate efforts to deliver results for the benefit of ASH1L community. As a rare disease foundation, we realize the importance of funding and appreciate how precious donations are. We, therefore, hope to only invest in critical projects that will leverage the funds significantly towards delivering a cure and enable additional projects essential for therapy development. Our ASH1L Patient community matters the most to us! 

Roadmap to therapies image.png
Image by Silje Roseneng

BUILD HUMANIZED MICE AND OTHER MODELS LIKE iPSCs FOR ASH1L

  • Build models that are most relevant to the ASH1L human mutation and replicate the symptoms

  • Study, examine and analyze the phenotypes exhibited by the models

  • Design a wide range of therapeutics and observe the effect on the models

CHARACTERIZE THE MODEL

  • Understand and correlate the humanized model based on the phenotypes and symptoms

  • Meticulous examination of the symptoms on the model

  • Develop measurable biomarkers for testing therapies

Scientist Using Microscope
Image by Nastya Dulhiier

TEST AND OPTIMIZE THERAPIES ON THE MODEL

  • Using different platforms like Basket/Umbrella/Adaptive Trials, test and optimize which therapeutic model can be delivered for ASH1L community 

    • Gene Editing, Base Editing, Prime Editing Therapies

    • AAVs

    • Antisense Oligonucleotide (ASO) and other RNA Therapies

    • Small Molecules and Repurposed Drugs 

SAFETY AND EFFICACY STUDIES

  • The models give the opportunity of testing the safety and efficacy of therapies without real human interventions. 

  • Once a drug or a therapy has been proven to be safe and optimal on the models, then the next step is to prepare for phases of clinical trials. 

Image by Sincerely Media
Doctor High Five

ASH1L PATIENT CLINICAL TRIALS

  • Approval from FDA and other international regulatory agencies is pivotal.

  • Proving the safety and efficacy of a drug or a therapy in a model to the FDA can facilitate the clinical trials in human patients. 

GOAL - CURE ASH1L

The end goal is to have a cure through drug discovery or therapy which will help ameliorate the symptoms in ASH1L patients so that the patients can lead a good quality of life. 

Image by Ronnie Overgoor