DISCLAIMERThe information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.
compose your message
email sent successfully
Trusted Resources: News & Events
Latest announcements and gatherings
MaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative research & development agreement for Sickle Cell Disease
MaxCyte, Inc. today announces it has entered into a Cooperative Research and Development Agreement (“CRADA”) with the U.S. National Institutes of Health (“NIH”). Under this new agreement, MaxCyte and the National Heart, Lung, and Blood Institute (“NHLBI”), part of the NIH, will aim to develop treatments for individuals with sickle cell disease (“SCD”) using next-generation CRISPR/Cas9-based single-nucleotide correction enabled by MaxCyte’s cell engineering platform.
In the search for alternative therapies for SCD, NHLBI will conduct pre-clinical research evaluating the effectiveness and safety of CRISPR-Cas9 gene editing on models of SCD by “correcting” the faulty hemoglobin gene that causes the disease, and addressing DNA mutations in non-corrected cells that contribute to the disease. As part of the agreement, MaxCyte will supply mRNA molecules and focus on leveraging its Flow Electroporation® Technology to develop reliable and effective processes to produce clinically meaningful correction of mutated gene sequences.
news & eventsPartnership to Ensure Supply of LentiGlobin, Potential Gene TherapyBluebird Bio extended a partnership with...
news & eventsCRISPR deployed to combat sickle-cell anaemiaA mutation in a single DNA letter causes...
news & eventsbluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Depende...First patient with severe sickle cell di...
news & eventsCalimmune Expands Lentiviral Gene Therapy Pipeline Through License of Sickle Cell Disease Therapeutic CandidateLicense of Gamma-Globin from Cincinnati ...
news & eventsSickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
news & eventsSickle-Cell Patients See Hope in CRISPRSickle-cell disease is one of the most c...
news & eventsBioverativ and Sangamo announce FDA acceptance of IND application for gene-edited cell therapy BIVV003 to treat sick...Bioverativ Inc., a Sanofi company dedi...
send a message
Don’t forget to join the oneSCDvoice community!
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.