About QurAlis
At QurAlis, we are pioneering the path to conquering ALS and other neurodegenerative diseases with genetically validated targets with next-generation precision medicines. Our proprietary platforms and unique biomarkers enable the design and development of precision medicines that act directly on disease-causing genetic alterations.
We are advancing a deep pipeline of antisense oligonucleotides (ASOs) and small molecule programs addressing sub-forms of ALS that account for the majority of ALS patients. And, we are leveraging our insights, platforms, and successes in ALS to collaborate and expand our pipeline to other neurodegenerative diseases, such as frontotemporal-degeneration (FTD).
Our goal is to halt disease progression and significantly improve outcomes.
Our mission is to make a meaningful difference in patients’ lives.
That is QurAlis.
Our Company Values
Pioneering
We are pioneers in neurodegenerative disease biology, stem cell and ASO technology, biomarkers, and small molecule design. We invoke creativity to achieve success. We are at the forefront of developing cutting-edge precision medicine for neurodegenerative diseases. We dare to follow science and to break through existing barriers.
Urgency and Passion
We feel a great sense of urgency to bring our therapies to patients. To succeed in bringing new treatments to patients worldwide, we must remain honest and empathetic to our patient community, our science, our colleagues, and ourselves. Our passion and commitment motivate us by generating excitement and infusing energy in our work. We remain agile to ensure a rapid embrace of new scientific insights and technologies, and to persevere until we achieve our goal of bringing new hope to patients with ALS and other neurodegenerative diseases.
Diversity and Collaboration
The world’s biggest problems can only be solved by working together, in an environment that respects all ideas and contributors. Our strength comes from a foundation of world-renowned pioneers, team members and patient community members, all from various backgrounds and viewpoints. Our diversity guides our strategy and decision-making toward our goal of treating patients who come from all regions and social backgrounds across the world.
Precision
Precision and excellence are embedded in all layers of our organization as the hallmark of our methodology from discovery, research, and development, to patients. We believe that success in treating neurodegenerative diseases will be achieved by being precise - targeting the right patients, identifying the right disease mechanism, and carefully developing disease-modifying, clinically meaningful therapies to improve the lives of patients.
QurAlis Early Access Policy
At QurAlis, our goal is to halt disease progression and significantly improve outcomes. We are trailblazing the path to conquering amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets with next-generation precision medicines. QurAlis’ proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations.
Our mission is to make a meaningful difference in patients’ lives.
Patients with serious or life-threatening diseases or conditions sometimes seek medical products that are not yet approved or available in their country. This is known as early access, managed access, or expanded access. Early access programs are a means by which investigational therapies are made available, in certain circumstances, to treat patients with serious diseases who are unable to participate in an ongoing clinical trial or whose treatment options are otherwise limited.
Before a new product can be placed on the market, it must undergo well-controlled clinical trials to demonstrate that it is safe and effective, and its potential benefit to patients outweighs the possible risks. A clinical trial is a research program conducted with volunteer participants to evaluate an investigational new drug or medicine in which comprehensive safety and efficacy data are compiled. Clinical trial results and related product information are then submitted to the relevant health authorities for review. Clinical trials result in the generation of evidence and information that may lead to the approval of an investigational new drug or medicine, which can make it more widely available to patients.
Currently, QurAlis does not have investigational medicines available through an expanded access program. QurAlis believes that participating in clinical trials is the best way for patients to access investigational medicines. QurAlis will strive to design trials that are inclusive of as many patients as possible, but we recognize that not every patient will be eligible for these trials.
For those patients who have completed a QurAlis clinical trial, it is our plan, when supported by the scientific data, to provide access to the investigational medicine until it is approved and available locally.
QurAlis will continue to assess early access as our clinical programs progress and may reconsider making one or more of its investigational medicines available through an Expanded Access Program in the future.