FAQs

Why do you favor a small molecule strategy instead of a gene therapy approach?

There are several reasons for this:

1. Control. Small molecules allow telomerase activation to be controllable and reversible, which is a key safety consideration. Most gene therapies, once applied, are effectively always “on.” While inducible gene therapy systems exist, they are not widely used clinically.

2. Expression profile. Gene therapies often produce high expression in a limited number of cells, resulting in a mosaic effect. This is likely not preferable for a telomerase therapy. Our view is that a telomerase therapy should instead aim for broader distribution across cells, with more moderated levels of expression within each cell.

3. Cost and accessibility. Gene therapies are currently very expensive and are likely to remain so in the near term. Even with reductions in cost, they are unlikely to be broadly accessible. A small molecule approach offers the potential for significantly lower cost and wider availability.  We want a telomerase therapy to be financially accessible.

4. Convenience. An oral drug is far more practical than an IV or lumbar puncture-based delivery.

5. Safety considerations. Gene therapies still carry unresolved safety concerns, including hepatic effects, which continue to be an area of active evaluation.

What stage of development are you currently in?

Our work is currently at the in vitro stage. We have demonstrated increased hTERT expression and telomerase activity using multiple small molecule compounds, with reproducible results and appropriate controls. Our next phase focuses on safety characterization, telomere length analysis, and expansion into organoid and in vivo models.

Have you patented these molecules yet?

Provisional patent applications have been filed to establish early protection of our work. We intend to convert these into non-provisional filings as part of our next stage of development. The timing of full patent disclosure is being managed carefully to balance intellectual property protection with ongoing research progress. In the meantime, key aspects of our work are maintained as confidential know-how.

What evidence do you have that your approach works?

We have observed increased hTERT expression via qPCR and increased telomerase activity using TRAP assays across multiple compounds and experimental replicates. Telomere length effects are currently under investigation. Our focus at this stage is to establish a clear and reproducible efficacy signal alongside a strong safety profile.

How is your approach different from other telomerase-based therapies?

Most telomerase-based approaches focus on gene therapy, which can result in permanent or difficult-to-control expression. Our approach uses small molecules designed to enable controlled and reversible activation of hTERT. We believe this provides a more practical path toward safety, scalability, and clinical accessibility.

What are the biggest risks to this approach?

The primary risks include:

– Potential off-target biological effects
– Overactivation of telomerase in certain cellular contexts
– Unintended impacts on gene expression due to epigenetic modulation

Our development strategy is specifically designed to address these risks through controlled activation, dose optimization, and comprehensive safety evaluation across multiple biological pathways.

How do you evaluate safety?

We evaluate safety across multiple biological pathways, including genotoxicity, DNA damage, oxidative stress, apoptosis, inflammation, transformation, and cell cycle regulation. We are also planning RNA sequencing studies to assess broader transcriptional effects and identify any unintended pathway activation.

Why does your research approach operate on a shoestring budget?

Historically few people have been willing to fund telomerase research. There seems to be an unjustified bias against telomerase therapies in the research and investment communities. We believe this is beginning to change as the field matures and new data emerges. If investors interested in longevity science would like to put their research funds to excellent use, we would be glad to speak with them.

What is your opinion of senolytic drugs?

We believe senolytic drugs will provide short to medium term benefit (by reducing SASP signaling and inflammation, and potentially cancer rates), but that they may also accelerate aging in the longer term by stimulating non-senescent cells to divide more frequently, thus accelerating the rate at which their telomeres shorten (and thus senesce). Or to state it more aphoristically, the longer term problem with senolytic drugs is that eventually you run out of cells to eliminate. A better solution would be treat senescent cells with a therapy that will actually reverse their senescence – assuming they are not otherwise damaged.

Why do adult stem cells become senescent?

The research evidence is mixed regarding the degree to which adult stem cells produce telomerase to maintain their telomeres. Our review of the literature indicates that adult stem cells produce very little or no telomerase, or not to a sufficient degree to offset the telomere loss that results from replacing cell populations. Thus they too become senescent. This obviously does not apply to embryonic stem cells which do express telomerase. In addition, the evidence also supports that iPSC stem cells do express telomerase.

Is there some concern about telomerase and cancer?

This is a common and important question. The relationship between telomerase, aging, and cancer is complex, but several key points are well supported by current evidence. hTERT expression alone is not sufficient to drive cancer. In fact, critically short telomeres themselves can contribute to genomic instability, which is a known driver of cancer and other disease processes.

A more fruitful question is not simply whether telomerase can be activated, but whether it can be used safely as a therapeutic. Our approach focuses on controlled and reversible activation, which we believe is central to addressing this question. This is one of the primary reasons we are pursuing a small molecule approach.

We explore this topic in more detail in a dedicated article here.

Do you view telomerase therapy as a 'cure all' for age related pathologies?

Likely not. While we think that telomerase therapies will likely resolve many age related pathologies as a result of solving senescence and resetting gene expression, it is likely that some problems will need to be treated separately. If we look at the model of the lobster, whose cells do express telomerase, they still develop lipofuscin deposits which would need to be treated independently. Alternately, we think it is likely that certain types of damage may need to be repaired separately from a telomerase therapy.

So while we see telomerase therapy as a necessary first step toward substantially extending maximal human lifespan, we do not see it as being fully sufficient by itself.

However the reality is that no one truly knows which problems will be corrected by telomerase therapies and which ones will not (at the organismal level) – until we actually try it (at the organismal level) and find out. Until we actually do that, the discussion is largely (educated) speculation and hand waving. The best answer is, let’s try it and find out.

What does success look like in the next 12–18 months?

Success in the near term would include:

– Demonstrating reproducible telomerase activation alongside a favorable safety profile
– Validating telomere length effects in vitro
– Optimizing lead compounds
– Establishing early in vivo or organoid signals of efficacy and safety

These milestones would provide a clear foundation for further preclinical development.

Why focus on telomerase instead of other longevity approaches?

Many approaches target downstream effects of aging, such as inflammation or senescent cell accumulation. Our focus is on addressing an upstream driver – loss of cellular replicative capacity and healthy cellular function – while recognizing that multiple approaches may ultimately be needed in combination.

Why has telomerase therapy not already been widely developed? Why now?

Great question. There are several reasons, including historical concerns around cancer risk, technical challenges with gene delivery, and limitations in controlling innate telomerase activation. Advances in molecular biology and a growing body of epigenetic research are now enabling more precise and controlled approaches, which we believe creates an opportunity to revisit this area with improved tools and understanding.

Are you open to partnerships or collaboration?

We are open to discussions regarding research collaboration, licensing, and strategic partnerships, particularly with groups interested in longevity science and early-stage therapeutic development.