As part of the agreement, the foundation will provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD).
The project brings together Novartis drug discovery and gene therapy expertise with the
'Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease,' said
SCD is a hereditary blood disease-one of the oldest known and most common genetic disorders. The disease affects millions around the world, with over 300,000 born with the condition annually1,2. It disproportionately affects those of African descent, and sub-Saharan Africa bears roughly 80 percent of the disease burden3. It also is common among people with ancestry from
SCD is a complex genetic disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently and, early on, progresses to a chronic vascular disease1,4,5,6. The disease can lead to acute episodes of pain known as sickle cell pain crises, or vaso-occlusive crises, as well as life-threatening complications7,8,9. The resulting, frequent hospitalizations associated with sickle cell disease combined with an overall lack of specialized care places a significant burden on patients and their families, healthcare systems, and the overall economy. Even with today's best available care, SCD continues to drive premature deaths and disability.
While the genetic cause of SCD has been known for decades, it's only recently that the medical world has gained the tools to potentially fix the errant gene that causes the disease. Current clinical-stage gene-based therapies require extracting cells from a patient, altering those cells in a laboratory, and then reintroducing them to the patient through a complex procedure akin to a bone marrow transplant. The lab and manufacturing facilities and hospital infrastructure for such procedures often do not exist in the areas where SCD is most prevalent, excluding the vast majority of those with the disease from these life-changing gene therapies.
'Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings,' said
Novartis envisions developing an accessible in vivo gene therapy for SCD that could potentially be administered once, directly to the patient, without the need to modify the cells in a lab. This would have the advantage of mitigating the need for long or repeated hospital stays or specialized lab infrastructure. To facilitate the research, the
In addition to research funding, the
'Novartis is proud to lead this effort to find an accessible genetic therapy for sickle cell disease, with support from the
The agreement builds on the Novartis commitment to improve the lives of patients with sickle cell disease through the development of new treatments, including crizanlizumab, strategic partnerships with government and non-government organizations, and support for such interventions as newborn screenings and the distribution of existing drugs.
Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as 'potential,' 'potentially,' 'can,' 'will,' 'could,' 'would,' 'believe,' 'commitment,' 'pipeline,' 'to discover,' 'aims,' 'to address,' 'promise,' 'envisions,' 'to facilitate,' 'to provide,' 'lead,' or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, including crizanlizumab, or regarding the collaboration to discover and develop an accessible in vivo gene therapy for sickle cell disease. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release, including crizanlizumab, will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that the activities and efforts described in this release will be achieved or succeed, in the expected time frame, or at all. In particular, our expectations regarding such products and efforts could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in
About Novartis
Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world.
Contact:
Tel: +41 61 324 1374
Email: antonio.ligi@novartis.com
(C) 2021 Electronic News Publishing, source