Telomerase Gene Therapy

Sierra Sciences has invented a patented Telomerase Gene Therapy for humans, dogs, cats, and horses designed to be a “Cure for Aging”. We also created one for pigs though we aren’t certain there is a market for one. Our gene therapies result in cells producing 30 times more telomerase than is needed to simply stop telomere shortening (see The Science of Telomere Biology and Aging). So, using this technology the telomeres actually get longer.

Gene Therapy is the science/medicine of delivering genes to cells to produce medically beneficial activities to the cells that were lacking or deficient before the therapy. Genes can be delivered to cells using viruses, liposomes, nanoparticles, etc. Dr. Andrews has been using gene therapy to deliver genes to cells since the early 1980’s. In the early 1970’s Dr. Andrews first wrote a paper on using retroviruses to deliver the “Hexosaminidase-A gene” to cure patients suffering from Tay Sachs Disease and, surprisingly, this is still in discussion almost 50 years later today (http://www.curetay-sachs.org/genetherapy.jsp). Dr. Andrews’ 40 years of experience with gene therapy has shown that viruses are the most effective method of delivering genes to cells. He has hands-on experience with almost every known gene therapy delivery system there is. Dr. Andrews believes that the safest viral delivery system at the present time is Adeno-Associated Virus (AAV) and Sierra Sciences has been using this system to successfully deliver genes, including the telomerase gene, to cells in vitro since 2008.

Sierra Sciences’ AAV Telomerase Gene Therapy is ready for immediate clinical testing and regulatory approval. It is also considered ready for immediate marketing because of new FDA rulings that allow patients to pay to be in clinical studies under the following terms:

FDA regulations permit such trials in “extraordinary circumstances,” such as when the drug being tested has a price tag so high that the trial couldn’t otherwise be run.

-Title 21 of the Code of Federal Regulations (FDA) – June 2016 21 CFR 312.8(b)(1)(iii)

As a result, Entrepreneurs and Investors can start generating revenue as soon as the testing begins.

Also, in the U.S. the FDA has recently allowed special provisions for gene therapy studies that will allow quicker and easier approval under the new Regenerative Medicine Advanced Therapy (RMAT) designation.

-Section 3033 of the 21st Century Cures Act

However, Entrepreneurs and Investors can conduct the studies under the jurisdictions of any countries in which they choose to conduct the studies.

Over 200 clinical studies are presently underway using AAV gene therapy to safely deliver a variety of genes to humans. And numerous studies have been published showing successful and safe delivery of genes, including telomerase, to animals using AAV gene therapy.

Sierra Sciences has written complete clinical protocols to use AAV telomerase gene therapy to treat many diseases, called Telomeropathies, associated with shortened telomeres. These include Alzheimer’s Disease, Clinical Limb Ischemia, Cardiomyopathy, and Aging itself. These are listed in ClinicalTrials.gov at https://clinicaltrials.gov/ct2/results?recrs=&cond=&term=libella&cntry=&state=&city=&dist=.

Other protocols are presently being written for other telomeropathies including, Idiopathic Pulmonary Fibrosis, Degenerative Disc Disease, AIDS, Demyelinating Disorders (e.g., MS and CIDP), and many more. In fact, there are no diseases anymore that have not been shown in scientific peer reviewed journals to be associated with telomere length. To see this, go to www.pubmed.gov and type “Telomer* AND [disease of choice]” in the search window. Lengthening telomeres could be the best discovery ever to provide health benefits to humans and pets.

Considerable cost savings could be achieved with the disease target of Degenerative Disc Disease because:

1. Injection straight into spinal discs will minimize the amount of gene therapy needed.
2. Results can be easily monitored by X-ray, CAT Scan, or MRI to view Disc thickness
3. Immune system responses should be minimal if at all.
4. Injections into spinal discs are already a very regular routine procedure.
5. Dr. Andrews Presented the idea as the Keynote Speaker at a Spine Disorder Conference in New Zealand in 2018 and it was very well received.
6. The therapy has already been shown to work in dogs when treated ex vivo [3]

Pets

Assembly Research and in vitro studies needed.

Estimated Time: 6 Months

Employees:

1 Molecular Biologist (Scientist) that is skilled in vector construction.

1 Cell Biologist (Scientist) that is skilled in transduction protocols of mammalian cells

1 QA/QC Scientist skilled in Telomerase Assays

Lab Space: 600 sf ($130/sf)

Office Space: 150 sf ($130/sf)

An additional Licensing Fee of $300,000 will be required if the Entrepreneur or Investor wants exclusivity.

Regarding pets, Telomerases are slightly different from species to species. So, to prevent immune responses it would be best to treat each pet with their own species-specific telomerase. In addition, binding of the protein component of telomerase to the RNA component of telomerase appears to be species specific so using Human TERT in dogs may not result in as much telomerase activity in dogs as using Dog TERT in dogs, for example. Sierra Sciences has already created AAV telomerase gene therapies for Dog, Cats, and Horses. We have also created one for Pigs since pigs are often used in efficacy studies for human drug approval.

At the time of this writing, Sierra Sciences has not tested any of our animal gene therapies in vitro.