Events/Research Updates

In the spring of 2025 we have begun discussions with a pharmaceutical company who is utilizing lipid nano particles to deliver a good copy of DLG4 mRNA into the body.  This treatment path has the potential to reach clinical trials within 18 months.  As it's broken down by the body within 48 hours, it is deemed to be a safer approach, and it can be delivered via a port with good distribution throughout the body and shows efficacy that it can pass the blood brain barrier.  We are working with a lab on obtaining a DLG4 mouse model to start our efficacy and safety studies simultaneously, begin conversations with the FDA, and identify the treating hospital.  We hope to raise at least another $100,00 in the coming year to make this treatment path an option.  Please help us support this new technology, which could have a huge impact on the genetic space in the coming years for many diseases.  The time is now! Thank you for your ongoing support!

 As we approach two years as a nonprofit organization, I wanted to share with our donors and community the progress HOPE for Harvey has made over the past two years.

We have several research teams we have been coordinating with since our inception. Some paths have given us curveballs, but we will continue to work toward a treatment, because we have nothing but time for our sweet boy and so the many others impacted by this devastating disease for decades.

Despite the many setbacks we have experienced to create animal and cell models, which would be used to test various treatment modalities, we have had some recent success in the past couple of weeks. Jackson Labs in Maine is now working on several new constructs for a Harvey mouse, and hopefully, one of these mouse designs will be able to survive Harvey’s debilitating mutation. We also heard in the past two weeks, TWO LABS HAVE NEURONS!!! This is a huge milestone, and these neurons can now be used to potentially test readily available drugs to see if we can improve cellular functioning as well as test one of our novel gene or RNA therapies!

In addition, we have also spoken to some new therapeutic research developers recently to add to our team. One lab in San Diego also develops RNA-based ASOs, and they have Harvey’s genetic information. They are using their proprietary technology to take another pass at any potential ASO designs, which could help stop Harvey’s mutated copy from coding a mutated protein. For unique mutations, this company will often do this work philanthropically, and then pass along their ASO designs to partners like St. Jude or the nonprofit n-Lorem, which would be amazing for our small organization.

Our wonderful, 'newish' consultant from Vanderbilt is also looking into creating some plasmid lines to potentially measure what Harvey’s mutation is actually doing at the cellular level, which is a much harder than one would expect … but could help us better determine if existing drugs could positively impact Harvey’s mutated or healthy protein expression. 

In addition, we just heard from our Israeli team partners about some wonderful, but unfortunately confidential for now, progress in their lab toward an AAV gene therapy for DLG4, which sends in a good copy of the gene to make up for the mutated copy’s dysfunction.

Our consultant also introduced HOPE for Harvey to a new, and amazing researcher in Canada who is exploring gene therapies for other genetic disorders. She ever so kindly offered to do some exploratory work in her mouse lab on DLG4 as well! She is also interested in pitching some multidisciplinary grants to the NIH on our behalf in the coming months. In addition, her research is focused on using the Covid vaccine capsids/coatings to package gene therapies, as a cheaper and potentially safer (less toxic) way to deliver these life-changing therapies. We’re super excited to see where this pathway could take us!

Although we are having to delay trialing some base editing designs from a team at the Broad Institute, until we have a viable mouse model, we remain hopeful that this could be a life-changing strategy for Harvey longterm, as it would directly target his single-letter mutation through an enzyme delivered intrathecally. This is new wave technology, and is going to change lives!

Also, all six of our current iPSC lines are at the Broad Institute now, and they’re working on some potential designs for screening existing small molecules/drugs available today.

Harvey is also now enrolled in Jennifer Doudna’s INGENUITI study at UC Berkeley. We have shipped them blood samples, and they are currently making their own iPSCs /stem cells and doing whole genome sequencing. Jennifer Doudna was a co-recipient of the Nobel Prize in Chemistry in 2020 for her development of CRISPR, which allows researchers to cut and alter genes.

In addition, one of Harvey’s amazing Neurologists, who also conducts research, made a Harvey worm, and they are currently studying the worm and Harvey’s mutation, to determine if there is a path forward to study how the worm’s motor, social, GI, and neurological development could change/improve when introducing small molecules/drugs currently available.

Lots of projects, lots of steady progress, which we could not do without our generous and amazing donors, so THANK YOU ALL!

-The HOPE for Harvey Foundation