Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, announced today that it has entered into an agreement with the Bill & Melinda Gates Foundation to jointly fund the company’s in vivo program for sickle cell disease (SCD). The funding includes an initial investment, with potential total investment of up to $50 million, from the Gates Foundation intended to support the development of Tessera’s SCD program into the clinic. The investment will support Tessera’s efforts towards advancing a SCD treatment and help further its objectives to develop transformative genetic medicines that are accessible globally.
Developing Gene Writers™ for SCD designed to enable a true correction of the sickle mutation to wild-type with one-time intravenous administration in vivo, without the need for complex stem cell mobilization or toxic chemotherapy conditioning. Critical in enabling in vivo therapies, Tessera is leveraging its proprietary non-viral delivery platform to develop lipid nanoparticles targeting delivery of Gene Writers to long-term hematopoietic stem cells.
“Sickle cell disease patients globally remain deeply underserved by existing treatment options. We are excited to develop what we believe will be a disruptive one-time curative treatment for SCD that is safer, easier, and more scalable than ex vivo genetic approaches,” said Michael Severino, M.D., CEO of Tessera Therapeutics. “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for sickle cell disease worldwide.”
About Tessera Therapeutics
Tessera Therapeutics is pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim to unlock broad new therapeutic frontiers. Our Gene Writing platform is designed to write therapeutic messages into the genome by efficiently changing single or multiple DNA base pairs, precisely correcting insertions or deletions, or adding exon-length sequences and whole genes. Our proprietary lipid nanoparticle delivery platform is designed to enable the in vivo delivery of RNA to targeted cell types. We believe our Gene Writing and delivery platforms will enable transformative genetic medicines to not only cure diseases that arise from errors in a single gene, but also modify inherited risk factors for common diseases and create engineered cells to treat cancer and potentially autoimmune and other diseases. Tessera Therapeutics was founded in 2018 by Flagship Pioneering, a life sciences innovation enterprise that conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability.
For more information about Tessera, please visit www.tesseratherapeutics.com.
Contact
Kristin Politi, PhD
LifeSci Communications, LLC
kpoliti@lifescicomms.com
