CELL AND GENE THERAPY
NeuExcell Therapeutics | September 13, 2021
NeuExcell Therapeutics and Spark Therapeutics, a member of the Roche Group announced a gene therapy collaboration aimed at developing a safe and effective treatment for patients suffering from Huntington's Disease (HD).
Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell's proprietary neuro-regenerative gene therapy platform and capabilities. NeuExcell's research team will collaborate closely with Spark Therapeutics to advance the program. Under the Option License NeuExcell is eligible to receive upfront, license fees, R&D and Sales milestone payments up to approximately $190 million plus product royalties. Under this Agreement, Spark Therapeutics has the option to license the exclusive worldwide rights of the NeuExcell's HD program.
The prevailing assumption has been that mammalian adult neurons cannot be replaced, and so therapeutic approaches for brain diseases tend to focus on slowing disease progress. NeuExcell Therapeutics may have unlocked the method for regenerating neural tissue. The company's neuroregenerative gene therapy platform is built around transcription factor-based trans-differentiation technology. The platform seeks to reprogram endogenous glial cells like astrocytes, which surround neurons and are often reactive after neurons are injured or die, into functional new neurons. While neurons cannot divide to regenerate themselves, glial cells are a renewable source for generating new neurons at the site of injury, and at the scale needed to have a meaningful therapeutic impact. NeuExcell is developing adeno-associated viruses (AAVs)-based neuroregenerative gene therapy to regenerate functional new neurons at the site of the neurodegeneration.
"At Spark, we understand that in order to break down barriers for people and families affected by genetic diseases, we need to work with like-minded partners that can integrate innovative technologies with our advanced proprietary AAV vector platform," said Joseph La Barge, Chief Business Officer of Spark Therapeutics. "Using our existing expertise in gene therapy development and NeuExcell's neuro-regenerative gene therapy research and capabilities, together we can progress the potential of gene therapy for patients living with Huntington's Disease."
Spark Therapeutic's advanced proprietary AAV vector platform targeted to the central nervous system offers the HD research and development (R&D) program a major advantage. At the forefront of gene therapy research for more than two decades, Spark Therapeutics has extensive knowledge and capabilities in this field that it will use to bring the HD program forward.
About Huntington's Disease
HD is an incurable, hereditary brain disorder caused by a single defective gene on chromosome 4. As the disease affects different parts of the brain, it impacts movement, behavior, and cognition. It becomes harder to walk, think, reason, swallow, and talk. Eventually, the person will need full-time care. The complications associated with HD are usually fatal.
About NeuExcell Therapeutics.
NeuExcell is a privately held early-stage gene therapy company headquartered in Pennsylvania, USA. Its mission is to improve the lives of patients suffering from neuro-degenerative diseases and CNS injuries. Based upon the scientific work of Prof. Gong Chen (Co-Founder and Chief Scientific Advisor), the Company has developed a potentially disruptive neural repair technology through in vivo astrocyte-to-neuron conversion by introducing neural transcription factors through adeno-associated virus (AAV)-based gene therapy. NeuExcell's pipeline covers major neurodegenerative diseases such as Stroke, Huntington's Disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer's Disease, Parkinson's Disease, Traumatic Brain Injury, Spinal Cord Injury, and Glioma.
About Spark Therapeutics
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease.
BioLineRx | October 30, 2020
BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, today announced positive results from a planned interim analysis of the ongoing GENESIS Phase 3 trial of motixafortide for stem cell mobilization (SCM) in multiple myeloma patients. At a meeting of the study's independent Data Monitoring Committee (DMC), a planned interim analysis of the study's primary endpoint was conducted independently by the DMC. Based on the statistically significant evidence favoring treatment with motixafortide, the DMC issued a recommendation to the Company that patient enrollment may be ceased immediately, without the need to recruit all 177 patients originally planned for the study. In accordance with the DMC's recommendation, study enrollment is now complete at 122 patients. Full results for the study, including secondary and exploratory efficacy endpoints, as well as extended safety data, will be announced after the last patient enrolled reaches 100 days of follow-up post-transplantation, which is expected to occur in the first half of 2021.
Harris-Stowe State University, United Health Foundation | November 20, 2021
The United Health Foundation, the philanthropic foundation of UnitedHealth Group (NYSE: UNH), has awarded a $2 million, three-year grant to Harris-Stowe State University to create a bioinformatics program for undergraduate students at the historically Black university located in St. Louis. Bioinformatics is an emerging field that combines science, physics, math and biology to aid in the diagnosis, treatment and discovery of new therapeutic advancements. An example of bioinformatics is the use of computer analysis on the Human Genome Project, which has recorded the 3 billion basic pairs of the human DNA system.
HSSU will develop a new undergraduate program to train students for careers as bioinformatics professionals. HSSU will use the support to
Develop new curricula combining coursework and experiential learning opportunities.
Expose high school students in surrounding school districts to the field of bioinformatics through a summer bioinformatics “boot camp” program.
Offer academic scholarships for up to 25 students each year.
“In the past decade, Harris-Stowe State University has emerged as a leader in training students for high-tech careers. This new program will help us to build on that important work, as well as continue to fulfill our mission of serving historically underrepresented students. Bioinformatics is a rapidly growing field of study, and it is vital for all people to play a role in its advancement.”
Dr. LaTonia Collins Smith, interim president of HSSU
Studies have shown that there is a substantial gap in the number of diverse college students trained in biomedical sciences. Black, Hispanic and Native American people account for only 7.1% of the employed biological/biomedical and life sciences workforce, according to the National Science Foundation. A diverse health workforce helps provide personalized, culturally competent care to an increasingly diverse population.
“The United Health Foundation is honored to collaborate with Harris-Stowe State University to increase the diversity of the life sciences workforce. We are excited about HSSU training students who will make discoveries, develop therapies and advance health care for all,” said Patrick Quinn, CEO of UnitedHealthcare in Missouri, a UnitedHealth Group company. “This partnership illustrates UnitedHealth Group’s commitment to health equity and to building a diverse health workforce reflective of our society.”
The commitment in Missouri is one of many ways UnitedHealth Group is working to advance health equity by diversifying the health workforce of the future. The United Health Foundation’s Diverse Scholars Initiative, for example, partners with nine nonprofit and civic organizations and has provided over 3,000 scholarships to diverse students studying medicine and public health across the U.S. since 2007. Optum Technology, part of Optum which is a UnitedHealth Group company, offers a mentor-led STEM program that has provided science, technology, engineering and mathematics training to over 7,000 diverse and underrepresented students at 103 middle and high schools since 2019. To learn more about the company’s commitment to health equity as well as its efforts to build healthier communities, improve outcomes and create a modern, high-performing health care system.
About Harris-Stowe State University
For over 160 years, Harris-Stowe State University (HSSU) has served the historically underrepresented. As a Historically Black College and University, HSSU is strongly committed to providing a high-quality higher education experience that is both affordable and accessible to the diverse populations within and beyond the metropolitan St. Louis region. More than 90% of student population are racially and ethnically diverse and receive some form of financial aid.
About the United Health Foundation
Through collaboration with community partners, grants and outreach efforts, the United Health Foundation works to improve our health system, build a diverse and dynamic health workforce and enhance the well-being of local communities. The United Health Foundation was established by UnitedHealth Group (NYSE: UNH) in 1999 as a not-for-profit, private foundation dedicated to improving health and health care. To date, the United Health Foundation has committed more than $500 million to programs and communities around the world.