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Eversight and Emmecell Biotech partner on advanced regenerative ocular cell therapy

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Eversight and biotech company Emmecell—both operating with a singular goal of curing global blindness—are collaborating to advance Emmecell’s technology of ocular cell therapies.

Let’s start with a refresher.

As a global nonprofit eye bank, Eversight is one of the largest networks of eye banks in the United States that works to recover, evaluate, and provide human eye tissues for transplantation; support research into curing blinding eye conditions; promote donation awareness; and provide humanitarian aid across the globe.

In 2023 alone, the nonprofit:

  • Provided 8,954 corneas for transplantation
  • Distributed 3,525 of those corneas in 29 countries outside the U.S.

Emmecell is a privately held, clinical-stage biotechnology company that is developing cell-based therapies for eye disease treatment—including corneal edema and macular degeneration—via the proprietary Magnetic Cell Delivery (MCD) nanoparticle platform.

Talk about this platform.

The MCD platform incorporates a unique approach to regenerative medicine by “leveraging magnetic nanoparticles to effectively localize and integrate cell therapies to the appropriate target tissue,” according to the company.

This differs from other cell therapies, which are typically limited in their capacity to replace or enhance damaged tissue due to an “inability to localize treatment to the site of injury or disease.”

The goal: solve the challenges associated with the delivery, retention, and integration of cell therapies.

Now Emmecell’s cell therapy.

E02002 is a minimally-invasive cell therapy that injects healthy corneal endothelial cells taken from donor corneas into the eye to “repopulate a patient’s diseased cornea with functional endothelial cells, potentially eliminating the need for transplantation.”

Gotcha. So how would this partnership work?

Corneal endothelial cells obtained from a single donor tissue through Eversight’s network could restore vision via Emmecell’s technologies, including E02002’s injectable cell therapy.

And how is this being clinically tested?

Through a U.S.-based, prospective, multicenter, open-label, dose-escalation, phase 1b study (NCT04894110) that will evaluate the safety and tolerability of E02002 in participants (aged 21+) with the following criteria:

  • Phakic or pseudophakic with a posterior chamber intraocular lens (IOL)
  • Symptomatic corneal edema associated with endothelial dysfunction that may be secondary to Fuchs’ corneal dystrophy (FCD) or pseudophakic bullous keratopathy.

How is the study designed?

  • Group 1
    • Evaluate the safety of four escalating doses of E02002 for 26 weeks following treatment (E02002 standard dose, low, mid, and high doses)
    • Conducted in patients with and without either endothelial brushing (EB) or Descemet Stripping (DS)
    • Assessed in eyes with corneal edema secondary to corneal endothelial dysfunction that also qualify for surgery involving either:
      • Full-thickness corneal transplantation
      • Endothelial keratoplasty (EK)
  • Group 2
    • Three highest doses of E02002 administered in up to 21 participants in a randomized, double-masked design.
      • Low dose, mid dose, and high dose

What will be measured?

Per Clinical Trials, the primary outcome measure includes the safety/tolerability of E02002, as determined by the development of treatment-emergent adverse events following injection.

Secondary outcomes include:

  • Effect of E02002 on corneal thickness
  • Effect of E02002 on best-corrected visual acuity (BCVA)

All outcomes will be measured at 26 weeks after injection.

Any input from the collaborators?

According to Jeffrey L. Goldberg, MD, PhD, co-founder of Emmecell and chair of Byers Eye Institute at Stanford University in Palo Alto, California, the company is confident in EverSight’s capabilities.

The nonprofit’s “eye banking expertise, global impact, commitment to research and surgeon education, and history of innovation and leadership in their field will help further harness the power of the Emmecell platform to cure blindness for more people worldwide,” Dr. Goldberg stated.

What if I want to enroll my cornea patients in this trial?

Corneal specialists interested in enrolling patients are advised to either call 650-769-4232 or email clinicaltrials@emmecell.com.

And lastly, what’s the bigger picture on this?

With more than 50% of the world’s population with no access to donor corneal tissue, an estimated 10 million people are unable to receive a sight-restoring cornea transplant.

Per Eversight, this therapy could be administered with little-to-no clinical follow up needed in communities that simply cannot access tissue for transplant.

If proven safe and effective, Eversight stated, “Emmecell’s EO2002 therapy could eliminate the need for corneal transplantation in thousands of people in the U.S. annually.”

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