CorriXR: How This Company Is Using CRISPR/Cas Gene Editing To Develop New Medicines

By Amit Chowdhry • Apr 26, 2024

CorriXR Therapeutics is on the clinical frontier of gene editing as a transformative pre-clinical stage biotechnology company with a ground-breaking in vivo gene-editing therapeutic platform technology. Pulse 2.0 interviewed CorriXR Therapeutics CEO Deborah Moorad to learn more about the company.

Deborah Moorad’s Background

Moorad’s original education track was medical school. And Moorad said:

“From a young age I really enjoyed science and research – ultimately, doing something that impacted others, that made a difference. During my medical school track, I was introduced to the world of venture capital and how patents (intellectual property)/research is advanced through development. This story really resonated with me, I knew this path would provide a large opportunity to continuously engage in cutting edge research and make a difference in the word of medicine. Instead of completing medical school, I opted to obtain my MBA. It has been non-stop ever since.”

“Most notable was my tenure as CEO of Nature Technology Corp., whose technology (HyperGRO, Nanoplasmid, and REVIVER) is used by leading pharmaceutical/biotechnology including Genentech and Poseida Therapeutics – as well as nearly every contract development and manufacturing organization (CDMO) in the gene therapy/genomic medicine space. Nature Technology Corp. was acquired by Aldevron a Danaher Company. Other career highlights include managing and leading the post-merger and management of the global KOL’s for Dentsply Sirona. The Dentsply International and Sirona Dental Systems merger notched $5.6 billion. I also led a repurposed drug (OKN-007) from animal studies to clinical trial for glioblastoma multiforme, which was later acquired, and patients are still being treated using this therapy.”

Formation Of CorriXR

How did the idea for the company come together? Moorad shared:

“Our Founder Eric Kmiec, Ph.D. has been in the gene editing field for over 25 years. After joining the Helen F. Graham Cancer Center & Research Institute at ChristianaCare, he began holding regular discussions with surgical oncologists and developed CRISPR-directed gene editing for solid tumors as an augmented therapy for cancer care. The original idea came together in 2016 with the choice of NRF2 as the target gene with the goal of overcoming cancer drug resistance to enable standard-of-care (e.g., chemotherapy) to work better at lower dosages, thereby reducing side effects and patient suffering. In some ways, the idea has been incubating for 25 years, but formation of CorriXR Therapeutics was based on clinical advice and support from ChristianaCare.”

“I was introduced to CorriXR/Eric Kmiec by a longtime friend in the biotech industry Christopher Yochim. Coming off a large acquisition – I knew I was not done and desired to find a meaningful project with a large impact. When I met Eric at CorriXR it was an immediate fit – and I knew, this was my next company/adventure. CorriXR allows me to use the experience I have gained career wise and connections I have made throughout the years – as well as do something innovative in the world of research. I am truly thankful – Bringing this research to my home state – (Oklahoma) and sharing this journey with individuals I have worked with over the years (Matt Gibson, Mike Moradi, Dr. James Battiste, Jill Castilla). The greatest joy is sharing this adventure and having the support of others and the opportunity to be part of a pivotal time in research.”

Favorite Memory

What has been Moorad’s favorite memory working for the company so far? Moorad reflected:

“I recently onboarded CorriXR and I was in Delaware meeting with the Gene Editing Institute (GEI) and Christiana Care – where the CorriXR technology was spun out of. Eric (scientific founder) told me we would have a casual day in the lab – and I should dress comfy encouraging me to wear my hoodie and sneakers. The next day I showed up to the lab in a Taylor Swift hoodie and adidas and met the entire GEI lab. So, my first introduction to the lab as CEO – was in a Taylor Swift hoodie and adidas.”

“Now when we have big milestones or achieve something big – Eric always makes comments about wearing a Malibu Okie sweatshirt and we have a good laugh.”

Core Products

What are CorriXR’s core products and features? Moorad explained:

“Our therapeutic is a CRISPR/Cas9/LNP complex that disables genes in tumor cells that confer cancer drug resistance. By knocking out these genes, augmentative cancer therapies can be more efficacious. In animal studies, our therapeutic molecules demonstrate very low off-target effects and minimal biodistribution to other cells in the body.”

Challenges Faced

What challenges has Moorad faced in building the company? Moorad acknowledged:

“The biggest challenge is the lack of understanding of research/medicine in this field – It does sound a lot like rocket science or putting a man on the moon. When in reality there have been many advancements in this space – the recent FDA approval of Vertex CRISPR-based gene-editing therapy in the U.S. , and we will start to see more of these wins in the years to come (including CorriXR).”

Evolution Of CorriXR’s Technology

How has the company’s technology evolved since launching? Moorad noted:

“Our R&D team transitioned from viral delivery to non-viral lipid nanoparticle (LNP) delivery methodology. We also identified the optimal targeting sites within genes to most effectively overcome cancer drug resistance.”

Significant Milestones

What have been some of the company’s most significant milestones? Moorad cited:

  • CorriXR Therapeutics is the first spin-out from ChristianaCare.
  • Completed a successful INTERACT meeting at the FDA and have used their guidance to identify additional tumor targets.
  • CorriXR was a finalist and invited to present at the 2023 BIO Start Up Stadium.
  • Hired Deborah Moorad as full-time CEO in 2023.
  • Completed our Seed Round of funding.
  • Many strategic relationships that will help transition to human trail exponentially quickly.

Total Addressable Market

What total addressable market (TAM) size is the company pursuing? Moorad assessed:

“Our initial TAM is solid tumors, including squamous cell carcinoma of the lung, esophagus, and head & neck, as well as glioma tumors of the brain.”

Genome Editing Market Overview: The global genome editing market size for technologies is estimated to be worth USD 3.5 billion in 2023 and is expected to grow at CAGR of 12.6% during the forecast period (2023-2035). Ref. Global Genome Editing Market Size & Share | Report 2023 -2035 (rootsanalysis.com)

The global solid tumor cancer treatment market size was estimated at $185.97 billion in 2022 and is projected to hit around USD 532.42 billion by 2032, growing at a CAGR of 11.09% during the forecast period 2023 to 2032. Ref. Solid Tumor Cancer Treatment Market Size, Report 2032 (precedenceresearch.com)

The global head and neck cancer therapeutics market size was valued at USD 1.22 billion in 2021 and is expected to expand at a compounded annual growth rate (CAGR) of 12.5% from 2022 to 2030. The increasing prevalence of head and neck cancer is anticipated to fuel market growth. According to the American Society of Clinical Oncology (ASCO) in the U.S., approximately 4% of all cancer cases are related to the head and neck. It is estimated that around 66,470 people including 48,520 men and 17,950 women were diagnosed with this disease in 2022. Head and neck cancer patients are potentially at high risk of exposure to SARS-CoV-2 infections as they require frequent outpatient visits to assess disease progression and the use of systemic drug therapies to control serious disease conditions. Ref. Head And Neck Cancer Therapeutics Market Size Report, 2030 (grandviewresearch.com)

Glioma Treatment Market Size to Hit Around $7.55 Billion by 2030 Ref. Glioma Treatment Market Size to Hit Around USD 7.55 BN by 2030 | BioSpace

Differentiation From The Competition

What differentiates the company from its competition? Moorad affirmed:

“CorriXR is one of the first to enter the solid tumor space using genetic medicine, and one of the first to target overcoming drug resistance to improve the success of current cancer drugs.”

Future Company Goals

What are some of the company’s future company goals? Moorad concluded:

  • Clinical trials in 1Q2025
  • FIH clinical data
  • Acquisition