Pipeline

REALIZING THE POTENTIAL OF CELLULAR AND GENE THERAPY

We believe our cell+gene platform has the potential to reshape cellular medicine. While our authentic cells enable us to replace cells, restore function, and reverse disease, our engineered cells enable us to deliver payloads in ways never before thought possible, enabling therapies to treat previously intractable common and rare diseases.

Lead Program in Parkinson’s Disease

BlueRock’s founders have pioneered the derivation of dopaminergic neurons that have demonstrated robust preclinical data to restore motor function and increased dopamine release as they mature. Our lead program is an authentic cellular therapy candidate to treat Parkinson’s disease, which we expect to advance into clinical development, having already established preclinical proof of concept.

Why Parkinson’s Disease?

Parkinson’s Disease is the second most common neurodegenerative disorder, affecting an estimated 1.3 million people in the U.S. and greater than 7.5 million globally. It is a chronic and progressive disease that current treatments do not adequately address. The primary and most debilitating symptom is a progressive loss of motor control.

Using our authentic dopaminergic neurons as therapy, we aim to re-innervate the human brain and reverse degenerative disease, potentially restoring motor function to the millions of patients suffering from Parkinson’s.

Clinical Trials

The first clinical trial in the United States and Canada to study pluripotent stem cell-derived dopaminergic neurons in people living with Parkinson’s disease is currently underway. This Phase 1 study of DA01 is enrolling patients with advanced Parkinson’s Disease who meet the following criteria:

  • Aged: 60-76 in the U.S.; 50-76 in Canada
  • Diagnosed with Parkinson’s disease between 5 and 15 years ago
  • Their regular Parkinson’s medications are no longer fully effective or have caused side-effects
  • Have not had a neurosurgical treatment for their Parkinson’s disease (i.e., Deep Brain Stimulation)

The study objectives are to assess the safety and efficacy. Participants in the trial will receive dopamine-producing neural cells through surgical implantation into the putamen, an area of the brain that regulates movement.

DA01 is an experimental product candidate and has not been approved by any regulatory agencies. To learn more about the clinical trial, visit www.clinicaltrials.gov (Identifier: NCT0480273).

Expanded Access Policy

An expanded access program (also known as EAP) and sometimes called “compassionate use,” is a potential pathway for a patient with an immediately life-threatening condition or serious disease to gain access to an investigational drug for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. These investigational drugs have not yet been approved or cleared by regulatory bodies like the FDA, and these products have not yet been found to be safe and effective for their specific use.

We understand that some patients may wish to access our investigational drugs that have not yet been approved. At this stage of drug development and trials, we do not have enough clinical product or evidence of clinical safety and efficacy of our investigational drugs to support expanded access. Therefore, BlueRock Therapeutics does not currently offer an expanded access program at this time. The most appropriate way for patients to access our investigational drug is by participating in our clinical trials, and we encourage you to speak with your doctor about whether you would qualify for this trial. Information regarding ongoing clinical trials can be accessed at www.clinicaltrials.gov. (Identifier: NCT0480273)

In accordance with the 21st Century Cures Act, this policy may be revised at any time. If you have additional questions about BlueRock’s expanded access policy, please speak to your physician, or contact regulatory@bluerocktx.com.  We will make our best effort to respond within 5 days.