NeuExcell Therapeutics and Spark Therapeutics Announce Research Collaboration Agreement to Develop a Novel Gene Therapy for Huntington's Disease

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.


This important white paper provides an in-depth analysis of the clinical trial landscape in 10 Asian countries related to effective therapies of treatment of the five most prevalent and life-threatening Asian cancers.


This important white paper provides an in-depth analysis of the clinical trial landscape in 10 Asian countries related to effective therapies of treatment of the five most prevalent and life-threatening Asian cancers.

Related News


Innovent Announces First Patient Dosing of Universal Modular CAR-T Cell product IBI345

Innovent Biologics | February 21, 2022

Innovent Biologics, Inc. a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, metabolic, autoimmune and other major diseases, today announced the first patient dosing for its first-in-class IgG-based universal "modular" Claudin 18.2-targeting chimeric antigen receptor T (CAR-T) cell product (development code: IBI345) for the treatment of advanced Claudin18.2-positive solid tumors in an investigator-initiated-trial (IIT). Since Innovent announced its strategic cooperation with Roche on June 9, 2020, this is the first disclosure of the development milestone for the cell therapy products based on Roche's proprietary innovative technology platform. CAR-T cell therapy has established a meaningful role in the treatment of hematological tumors with 5 products approved by the FDA, and 2 by the NMPA in China, respectively; however, antitumor efficacy for solid tumors remains a challenge calling for technological breakthroughs. As the world's first universal "modular" CAR-T cell product, IBI345 has potentially differentiated advantages over conventional CAR-T cell therapy products, including: 1) leveraging antibody function to target tumor antigens and consequently amplifying the tumor antigen signal to guide CAR-T cells to enter the tumor, thus aiding in the initiation of tumor recognition and killing; 2) controlling the functional activity of the CAR-T cells via concomitantly administered antibodies which act as a bridge between the target cell and CAR-T cell, potentially providing a better safety profile; 3) allows for the sequential or simultaneous administration of more than one antibody targeting different antigen targets without changing CAR-T cells, to treat tumors with highly heterogeneous antigen expression or relapsed tumors due to antigen-loss, thereby improving the accessibility and increasing the flexibility of CAR-T cell therapy for patients. The principal investigator of the study, Professor Weichang Chen, the Secretary of the Party Committee and Chief Physician of the Department of Gastroenterology of the First Hospital Affiliated to Soochow University, pointed out: "As gastric and pancreatic cancer are highly aggressive tumors with high incidence rates in the world, patients are in urgent need of novel treatment options to improve clinical outcomes. IBI345, with its unique design to control CAR-T functional activity through the antibody bridging, will likely provide improved safety and efficacy. We look forward to the positive results of the safety, tolerability and efficacy data of IBI345 in patients with solid tumors, which will potentially provide a new option for the treatment of patients with advanced gastric or pancreatic cancer." "IBI345 is a highly differentiated CAR-T cell product with a new mechanism of action developed by Innovent based on Roche's proprietary technology. As a universal "modular" CAR-T cell therapy product, equipped with a precisely targeted antibody switch, it regulates the expansion of CAR-T cells to control side effects, potentially providing an opportunity to switch to another antibody that targets different antigens to prevent recurrence and treat different tumors. Our preclinical studies have validated this "modular" CAR-T technology, which provides us with the sufficient scientific data to initiate clinical studies. In the following clinical studies, we will explore the safety, tolerability, pharmacokinetics and preliminary efficacy of IBI345, and optimize the clinical dosage and regimen to support the subsequent IND application. We firmly believe that Innovent will be able to introduce more innovative cell therapies through platform technology innovation, product transformation and in-depth collaboration with the clinical academic community that will ultimately benefit more patients and families." Dr. Xu Wei, VP of Innovent, R&D Head of Cell Therapy About Claudin 18.2 Claudin protein is a critical component of tight junction complex molecules which play an important role in the life activities of the human body. On the one hand, tight junctions can function as a "barrier" to select the size and charge of substances, thereby regulating the transport of substances in the para-cellular pathway. For example, brain vascular endothelial cells can pass through this barrier, preventing blood from mixing with the extracellular fluid in the brain. On the other hand, tight junctions can also function as "fences", maintaining cell polarity by regulating the free diffusion of lipids and proteins between the apical and the basolateral membrane. About Gastric and Pancreatic adenocarcinoma Gastric and pancreatic cancer are both malignant tumors of the digestive system that seriously endanger human life and health. The latest global cancer statistics show that there will be about 1.089 million new cases of gastric cancer worldwide in 2020, accounting for 5.6% of the global cancer incidence, and about 768,000 deaths, accounting for 7.7% of the global cancer deaths. There are about 495,000 new cases of pancreatic cancer, accounting for 2.6% of the global cancer incidence, and about 466,000 deaths, accounting for 4.7% of the global cancer deaths. According to the national cancer statistics released by the National Cancer Center of China in 2019, the incidence of gastric cancer in China in 2015 was about 403,000, accounting for 10.26% of the total cancer incidence, second only to lung cancer at 20.03%, with an incidence rate of 29.31/100,000 people, and about 291,000 deaths, accounting for 12.45% of the total cancer deaths, behind lung and liver cancer, with a mortality rate of 21.16/100,000 people. In 2015, the number of cases and deaths of pancreatic cancer nationwide were 95,000 and 85,000 respectively, ranking the 10th and 6th in the incidence and mortality of malignant tumors. The mortality ratio of pancreatic cancer was 0.89, making it a true "King of Cancer". Despite the treatment progress in recent years, drug resistance, recurrence and metastasis are still inevitable. The 5-year survival rate of patients with advanced gastric cancer is about 5-20%, and the median survival rate is about 10 months. Pancreatic cancer has a worse prognosis, with a 5-year survival rate of only 6 to 8%. About IBI345 IBI345 is the first universal "modular" CAR-T cell product developed by Innovent based on Roche's proprietary technology. It is an internationally pioneered highly differentiated CAR-T cell product with a new mechanism of action. Different from conventional CAR-T cells that directly recognize and kill tumor cells, IBI345 comprises of two components, anti-Claudin18.2 antibodies and "modular" CAR-T cells where anti-Claudin18.2 antibody recognizes tumor antigen, thus calibrating and amplifying the antigen signal, and guides "modular" CAR-T cells to enter the tumor to initiate the cytotoxic and antitumor activity of CAR-T cells. Compared with conventional CAR-T cells, IBI345 has a variety of potential advantages, including dual effects of antibodies and CAR-T cells to increase antitumor efficacy, and flexible control of side effects by regulating CAR-T cell activity through antibodies; in addition, as having a universal CAR molecule, "modular" CAR-T cells can be easily switched to different antibodies without changing CAR-T cells, by sequentially or simultaneously administering more than one antibody targeting different antigen targets, to treat tumors with highly heterogeneous antigen expression or antigen-loss relapsed tumors, which holds a great potential to reduce the cost of CAR-T cell therapy and improve the accessibility of CAR-T cell therapy for patients. Innovent announced that first patient dosing has been completed in February 2022 in the investigator-initiated clinical trial of IBI345 for the treatment of advanced Claudin18.2-positive solid tumors, and enrollment is currently ongoing. The safety, tolerability, pharmacokinetics and preliminary efficacy of IBI345 will be explored, and the clinical dosage and regimen will be explored to provide clinical data support for the subsequent IND application. About Innovent Inspired by the spirit of "Start with Integrity, Succeed through Action," Innovent's mission is to develop, manufacture and commercialize high-quality biopharmaceutical products that are affordable to ordinary people. Established in 2011, Innovent is committed to developing, manufacturing and commercializing high-quality innovative medicines for the treatment of cancer, autoimmune, metabolic and other major diseases. On October 31, 2018, Innovent was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 01801.HK.

Read More


Synthace Unveils First Life Sciences R&D Cloud Addressing Complexity, Speed & Reproducibility for Scientists

Synthace | August 03, 2021

Synthace, a leading life sciences software company, today announced the first life sciences R&D cloud that includes a no-code software platform addressing the largest barriers to innovation that R&D life scientists currently face. With the release of this new platform, Synthace is also the first vendor to enable scientists to automate experimentation and insight sharing in a unified, global R&D environment. Scientists can now focus on asking the most impactful questions and unlock the true potential of biology. To solve humanity’s hardest problems, such as delivering breakthrough therapies or alternative food sources, the life sciences industry is under tremendous pressure to simultaneously overcome biology’s complexity, accelerate speed to scientific insight, and ensure the reproducibility of experiments. Synthace alleviates these challenges by empowering scientists to improve and accelerate decision-making with more statistically powerful, automated experiments that can minimize human error. Furthermore, Synthace’s cloud platform leverages intelligent, dynamic automation to produce the highest quality data sets that are primed for machine learning (ML) and other advanced analyses to lead to better insights. With Synthace, the life sciences can now benefit from a quantum leap in experimentation capabilities, accelerating development timelines that would have previously been impossible. Customers Accelerate R&D with Synthace Ipsen has been using Synthace to automate the design and construction of therapeutic candidates. With Synthace, Ipsen produced approximately 90 constructs five times faster than previous methods, substantially increasing the number of molecules entering the screening cascade. The platform also achieved a 10-fold reduction in costs associated with DNA synthesis. Karen Bunting, Director of Protein Sciences at Ipsen commented, “Synthace sits very well at the beginning of our drug discovery process. It allows us to explore larger drug design space by simplifying planning and production of multiple molecule variants with combinatorial construct assembly. These throughput improvements help us deliver well-tolerated and effective therapeutic solutions more rapidly to our patients.” Microsoft Research also uses Synthace to automatically generate biological data at a volume that allows its ML algorithms to rapidly improve. As part of reporting on advancements in programming biological systems, a member of its Biocomputation Group noted: “Synthace really comes into its own when we’re performing experiments with complex layouts like combinatorial construct assembly and design of experiments. When we’re building 12 constructs at a time, Synthace automates all the planning that would go into setting up such an experiment and allows it to become routine.” Synthace Life Sciences R&D Cloud The platform provides end-to-end management of the experimental lifecycle, from design through execution to data visualization and knowledge transfer. Synthace adheres to FAIR principles to support interoperability with other major lab informatics platforms to ensure streamlined data management for all of its customers. Only the Synthace Life Sciences R&D Cloud delivers: Complete experimental design, planning and automation, requiring no coding expertise. Scientists can define more informative and impactful experiments that would otherwise be impossible to run and easily implement Quality by Design (QbD) and Design of Experiments (DOE). Seamless, cloud-based data capture, processing, and visualization. R&D teams can deliver deeper and faster insights from fully contextualized, machine learning-ready data sets that are automatically generated from the laboratory. Minimal deployment and onboarding. Customers experience rapid time-to-insight through Synthace’s out-of-the-box platform features and pre-validated protocols for common applications such as ELISA and high-throughput purification, helping them shorten R&D cycles and study more candidates per program. About Synthace Synthace is a life sciences software company enabling life science the way it should be done. Delivering a life sciences R&D cloud to scientists who want to innovate faster, the Synthace platform seamlessly automates experimentation and insight sharing so that scientists can focus on asking the most impactful questions to unlock the true potential of biology. Top global pharmaceuticals, high-growth biotech companies, leading CDMOs, and innovators in artificial intelligence all turn to Synthace to discover solutions to humanity’s hardest problems.

Read More


Pfizer Invests $120 Million in Four Clinical-stage Biotech Companies Through the Pfizer Breakthrough Growth Initiative

Pfizer | January 15, 2021

Pfizer Inc. declared that, during the second 50% of 2020, it put a sum of $120 million of every four clinical-stage biotech organizations as a feature of the Pfizer Breakthrough Growth Initiative (PBGI). Through PBGI, Pfizer is putting up to $500 million in biotechnology organizations, giving subsidizing and admittance to Pfizer's logical skill to help guarantee the coherence of promising clinical improvement projects of likely future vital interest to Pfizer. Pfizer's initial PBGI investments include: $10 million in Vancouver, BC-based ESSA Pharma, a clinical-stage pharmaceutical company focused on developing novel and proprietary therapies for the treatment of patients with prostate cancer. $25 million in Cambridge, Mass.-based Trillium Therapeutics, a clinical-stage immuno-oncology company focused on developing innovative therapies for the treatment of cancer. In addition, Jeff Settleman, Senior Vice President and Chief Scientific Officer of Pfizer’s Oncology Research & Development Group, was named to Trillium’s Scientific Advisory Board.

Read More