While most gene therapies to date focus on compensating for or fixing broken genes in typically small numbers of patients (Orphan Drug), we employ gene therapy as a tool to deliver novel protein therapeutics intracellularly, using local administration. Our pharmacologic gene therapy approach (as opposed to corrective gene therapy) has the potential to reach large numbers of patients in many disease categories. Of course, in order to realize such mass-utility potential, it is essential that innovative gene therapies are highly efficacious and deliver excellent safety with minimal side-effects (such severe immune responses, off-target effects or adverse events in non-target tissues).

Based on preclinical data, our gene therapy has the potential to address large-scale population with the potential to replace opioids or treat drug resistant neurological disorders. Our state-of-the-art gene therapy vector has been carefully bioengineered with precision to be ‘replication-defective’, making it disease-free capable of delivering a therapeutic peptide safely to the intracellular space. This gene therapy vector also evades immune detection, severe immune responses, and demonstrates tissue specificity with the vector traveling from local administration to hyper-excitable diseased neurons, to restoring balance, reducing excitability, and producing profound analgesia without opioids in patients with chronic pain.

By historic analogy, the innovation of humanizing antibody therapies has transformed the protein therapeutic space since 1990 from obscure niche therapies serving a few very unfortunate patients to mass-market applications like rheumatoid arthritis, psoriasis, cancer and numerous other diseases in large numbers of patients today. However, antibodies and other protein therapeutics have the fundamental limitation of working on the cell’s exterior and cannot penetrate cells to deliver the necessary treatment to intracellular targets.

There is a huge unmet need in medicine today to deliver therapeutic proteins inside the cell. In our opinion, the combination of our vector and therapeutic transgene-protein innovations represents this transformational therapeutic to advance our technology platform with the great potential to address significant unmet needs in medicine today by allowing novel protein therapeutics to be active inside target cells. Crossing this intracellular barrier has the potential, like antibodies did for the extracellular space, to be equally revolutionary for advancing an entire novel generation of intracellularly active protein pharmacotherapeutics for major inadequately addressed clinical needs, such as neurological disorders and various forms of chronic pain.

Scalable Manufacturing: Competitive Cost of Goods, Supply for Mass Markets

Overcoming Industry Challenges:

• • Avoids issues like “empty capsids” common in AAV-based vector production
  • Process in solid phase single use reactors
  • Conceptually simpler production (e.g., like vaccines) is efficient cost-effective in comparison to typical AAV gene therapies

Cost-Effective Proprietary Production:

• • Manufactured using proprietary cell lines carrying functionality of missing regulatory genes (licensed)
  • Scalable manufacturing process empowers competitive production costs, like mass-market biopharma medications

Gene therapy itself is not new; it has been around for a few decades now – the first gene therapy, Gendicine (recombinant human p53 adenovirus), was approved in China in 2003. As with Gendicine, most first-generation gene therapies are delivered with viral vectors derived from the adenovirus, adeno-associated-virus (AAV) or retrovirus. Gene therapy delivery with these types of vectors is far from optimal, often causing severe immune responses (hyper-inflammatory immune responses or anaphylaxis or even a cytokine storm) and/or they can expose the patient to the risk of liver-injury and acute liver-failure (often lethal), or death.

While the genetics of first-generation gene therapy vectors are relatively easy to manipulate and have proven to be useful early research tools, they have not been optimally developed for pharmacotherapeutic applications and have many manufacturing and patient treatment disadvantages. Therefore, we have made very deliberate and targeted choices for, and thoroughly engineered modifications of, our carefully selected viral vectors and the associated cell-production system. Our innovative gene therapies have been developed in close collaboration with the world’s leading viral vector development lab of Dr. Joseph Gloriosio (Adolore Scientific Co-Founder and Chief Scientific Officer) and his team at the University of Pittsburgh, and are highly differentiated on the basis of the key aspects as indicated in the diagram above.