6 December 2022 - - US-based preclinical biotech company Eikonoklastes Therapeutics and US-based gene therapy-focused contract development and manufacturing organization Forge Biologics have forged a manufacturing partnership that will advance Eikonoklastes' AAV-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis, the companies said.

Forge will provide adeno-associated viral process development and manufacturing services for ET-101.

Eikonoklastes will utilize Forge's platform process including its proprietary HEK 293 suspension Ignition Cells and pEMBR adenovirus helper plasmid.

All development and AAV manufacturing activities will occur at the Hearth, Forge's 200,000 square foot gene therapy facility in Columbus, Ohio.

ALS is a rare neurological disease with significant socioeconomic impact, whose patients are desperate for novel, safe and effective therapies.

ALS is the lead indication for Eikonoklastes' ET-101, which has just received Orphan Drug Designation from the FDA--an important regulatory milestone reflecting the compelling data underlying the ET-101 program and its potential as a first-in-class treatment option for both familial and sporadic ALS.

Amyotrophic Lateral Sclerosis, also known as Lou Gehrig's Disease, is a rare, progressive, debilitating, and universally fatal neurodegenerative disease affecting the motor neurons connecting the brain and spinal cord. Degeneration of these neurons leads to paralysis as the brain loses its ability to control muscle movement.

Most people with ALS succumb to respiratory failure, usually within three to five years from when symptoms first appear.

An estimated 10% of ALS is familial and caused by genetic mutations that are inherited. The 90%+ of sporadic ALS may be due to a combination of environment and genetic risk factors. There is currently no cure for either familial or sporadic ALS.

Eikonoklastes Therapeutics is a preclinical biotech company rapidly developing a pipeline of disruptive new treatments for TODAY's most challenging diseases. The company's 1st-in-class gene therapy (ET-101) has been engineered to overexpress Caveolin-1, which organizes and regulates synaptic receptors essential for neuromuscular signaling and function.

Eikonoklastes is also advancing a new generation of tissue factor therapies (ET-201) for the treatment of Triple Negative Breast Cancer and other oncology indications.

Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company.

Forge's mission is to enable access to life changing gene therapies and help bring them from idea to reality.

Forge's 200,000 square foot facility utilizes 20 cGMP suites in Columbus, Ohio, the Hearth, to serve as its headquarters.

The Hearth is a custom-designed cGMP facility dedicated to AAV manufacturing and hosts scalable, end-to-end manufacturing services.

Offerings include process and analytical development, plasmid DNA manufacturing, viral vector manufacturing, final fill, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.

By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.