Announcing Our Partnership with NeuCyte: Launching Patient-Derived Cell Models for 2q37 Deletion
We are excited to share an important milestone in the Rare Futures Foundation journey: Rare Futures has formally partnered with NeuCyte to initiate patient-derived cell work in support of our HDAC4 Rescue Feasibility Study.
This partnership marks a critical step forward in building the experimental foundation needed to evaluate therapeutic strategies for HDAC4 haploinsufficiency in 2q37 deletion syndrome.
Why Cell Models Matter
For ultra-rare neurodevelopmental disorders like 2q37 deletion, one of the biggest challenges is the lack of high-quality, disease-relevant model systems. Without them, it is nearly impossible to rigorously test therapeutic hypotheses or compare approaches head-to-head.
Our work with NeuCyte is designed to change that.
By turning patient samples into induced pluripotent stem cells (iPSCs) and then into the brain cells affected in 2q37 deletion, we can:
Directly study the consequences of HDAC4 haploinsufficiency in human cells
Establish reproducible, scalable platforms for therapeutic screening and rescue experiments
Create a shared resource that can support multiple downstream therapeutic modalities
This work lays the groundwork for rational, data-driven decision-making as we evaluate potential treatments.
About Our Collaboration with NeuCyte
NeuCyte is a specialized biotech with deep expertise in patient-derived iPSC generation, neuronal differentiation, and quality control. Through this partnership, we are launching a structured program of work that includes:
Turning patient samples into multiple stem cell lines that carry the 2q37 / HDAC4 deletion
Carefully checking that these cells are healthy, stable, and truly representative of the patient
Guiding the cells to become brain cells that are most relevant to HDAC4’s role in development
Studying how these 2q37 cells behave — how they grow, communicate, and function — so we can understand what’s going wrong and how different treatments may help correct it
Enabling Downstream Rescue Studies
The NeuCyte cell models are not an end in themselves. They are a launchpad.
These iPSC-derived neuronal systems will be used to support:
Testing antisense oligonucleotides (ASOs) to see if we can safely increase HDAC4 activity in cells that are missing one copy of the gene
Exploring CRISPR-based allele activation, a technique that aims to “turn up” the remaining working copy of HDAC4
Running drug repurposing screens to identify existing medicines that may improve how these cells function
Measuring meaningful changes in the cells — such as how they grow, signal, and behave — to understand whether a potential treatment is truly helping
By grounding our rescue efforts in patient-derived human cells, we can more confidently assess which approaches show real promise—and which should be deprioritized early.
A Foundation Built for Collaboration
A key goal of Rare Futures is to build infrastructure that enables collaboration across academia, CROs, and industry. Establishing robust, well-characterized cell models is a prerequisite for that vision.
This partnership with NeuCyte reflects our commitment to:
De-risking early therapeutic development
Using philanthropic dollars efficiently
Creating assets that can accelerate progress for the entire 2q37 / HDAC4 community
Looking Ahead
We are incredibly grateful to the NeuCyte team for their partnership and scientific leadership, which has made this work possible.
As the cell lines are generated and validated, we will share updates on progress and how these models are being used to inform the next phases of the HDAC4 Rescue Feasibility Study.
This is how progress happens in rare disease: step by step, model by model, with science leading the way.
With hope and persistence,
Vanessa
