glucokinase

– Kriya anticipates up to 5 programs to enter clinic by the end of 2025 – – Current pipeline of gene therapies for common diseases spans three major therapeutics areas: ophthalmology, metabolic disease and neurology – – Company enters 2024 with cash balance of $325 million and runway into late 2026 – PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Kriya Therapeutics, Inc., ("Kriya") a biopharmaceutical company developing gene therapies for common diseases affecting millions of people around the world, today provided an update on its pipeline of gene therapies for prevalent conditions including geographic atrophy, thyroid eye disease, diabetes, NASH, trigeminal neuralgia and epilepsy. Kriya is advancing the first of its gene therapy product candidates into the clinic in 2024 and expects up to five programs in the clinic by the end of 2025. "Our mission is to revolutionize the field of medicine by delivering transformative, one-time gene therapies to patients suffering from common diseases," said Shankar Ramaswamy, M.D., Co-Founder and CEO of Kriya. "In parallel with our near-term goal of advancing up to five gene therapies into the clinic by the end of 2025, we remain deeply committed to our vision of significantly reducing the cost of manufacturing gene therapies through innovations in process sciences and product engineering.” Kriya's platform includes an end-to-end gene therapy product development engine that integrates cutting-edge research and computational biology with a scalable GMP manufacturing facility deploying next-generation processes and analytical characterization techniques. Kriya’s pipeline includes gene therapies across three major therapeutic areas: ophthalmology, metabolic disease and neurology. Its programs share the following features: direct-to-tissue delivery, validated biology, de-risked clinical and regulatory paths and large commercial markets. Kriya’s publicly disclosed programs are summarized below. Dr. Ramaswamy will share further information on Kriya’s portfolio during his presentation at the J.P. Morgan 42nd Annual Healthcare Conference on Tuesday, January 9th at 9:00 AM PT in San Francisco, California. Ophthalmology ProgramsGeographic Atrophy (GA): KRIYA-825 is designed as a one-time gene therapy expressing a fusion protein inhibiting complement C3 and C5, delivered by an in-office suprachoroidal injection with the objective of slowing GA lesion growth and vision loss. GA affects approximately two million people in the United States and the European Union. Existing treatment options require monthly or bimonthly physician-administered intravitreal injections that can be burdensome for patients. Kriya has designed KRIYA-825 for GA with the following potential goals in mind: Robust complement inhibition: A novel complement receptor 2 -complement receptor 1 (CR2-CR1) fusion protein—where the CR1 domain is designed to block the function of both C3 and C5, while the CR2 domain is designed to bind to cell surfaces where complement fragments deposit and cause damage;Multi-year durability: Adeno-associated virus (AAV)-mediated continuous expression of CR2-CR1 fusion protein following a one-time injection to eliminate the need for frequent intravitreal injections as required by currently available therapies for GA; andTargeted delivery: One-time suprachoroidal injection with the goal of achieving transduction of retinal cells while minimizing inflammation and overall patient burden. Thyroid Eye Disease (TED): KRIYA-586 is designed as a one-time gene therapy expressing an antibody blocking insulin-like growth factor-1 receptors (IGF-1R), delivered by an in-office peribulbar injection with the objective of reducing proptosis and diplopia as well as other key features of TED. Approximately one million people in the United States and the European Union have TED. The only FDA-approved treatment is an intravenously administered monoclonal antibody that works by blocking IGF-1R. Kriya has designed KRIYA-586 for TED with the following potential goals in mind: Minimized treatment burden: One-time, in-office peribulbar injection to eliminate the burdensome requirement for multiple infusions or injections;Focal delivery: Localized antibody expression in extraocular muscles and fat tissue to limit the potential for side effects that can be observed with systemically administered therapies; andLong-term durability: AAV-mediated antibody expression to deliver sustained improvements in proptosis and diplopia as well as other key manifestations of TED. For more information on Kriya's Ophthalmology programs, please visit the Ophthalmology page of our website. Metabolic Disease ProgramsDiabetes: KRIYA-839 is designed as a one-time gene therapy expressing insulin and glucokinase, delivered intramuscularly with the objective of driving durable glycemic control and reducing or eliminating the need for exogenous insulin. Of the approximately 90 million people in the United States and the European Union who have type 1 and type 2 diabetes, 17 million use insulin to manage their disease. Current treatments for type 1 diabetes require burdensome chronic insulin injections and constant treatment modification based on dietary intake and blood glucose monitoring. Kriya has designed KRIYA-839 for diabetes with the following potential goals in mind: Robust glycemic control: Improving time-in-range and lowering HbA1c to reduce the risk of long-term diabetic complications;Insulin independence: Enabling patients to achieve insulin independence while maintaining robust glycemic control; andMinimized treatment burden: Substantially reducing or eliminating the need for multiple daily injections of insulin with a one-time treatment. Nonalcoholic Steatohepatitis (NASH): KRIYA-497 is designed as a one-time gene therapy expressing the native FGF21 protein, delivered intramuscularly with the objective of reducing fibrosis, reversing steatosis, and improving the overall metabolic profile of patients with later stages of NASH with liver predominant pathology (F3 and compensated F4). NASH affects approximately 40 million people in the United States and the European Union. There are currently no FDA-approved therapies for the treatment of NASH. Kriya has designed KRIYA-497 for NASH with the following potential goals in mind: Robust anti-fibrotic activity: Expression of native FGF21 protein to drive natural ligand/receptor interactions and enhance biodistribution across multiple organs;Minimized treatment burden: One-time intramuscularly administered therapy to reduce complexity for patients who are often taking multiple other concomitant therapies; andContinuous expression: Consistent concentration of native FGF21 protein to limit Cmax-mediated gastrointestinal issues. For more information on Kriya's Metabolic Disease programs, please visit the Metabolic Disease page of our website. Neurology ProgramsTrigeminal Neuralgia (TN): KRIYA-748 is designed as a one-time gene therapy expressing a chemogenetically-gated ion channel, delivered through an injection into the trigeminal nerve with the objective of reducing the frequency and severity of debilitating paroxysmal pain attacks. Once expressed in the nerve, the channel is designed to selectively open in the presence of the orally-administered CNS-penetrant small molecule, varenicline (the FDA-approved generic medication for smoking cessation originally marketed under the brand name "Chantix®"), leading to the passage of chloride ions and reduced excitation of target neurons. Trigeminal Neuralgia affects approximately 400,000 people in the United States and the European Union. Current treatments include anticonvulsants, which can have significant tolerability issues and waning efficacy over time. Kriya has designed KRIYA-748 for trigeminal neuralgia with the following potential goals in mind: Pain reduction: Goal of reducing the frequency and severity of pain attacks by focally inhibiting neuronal hyperexcitability in the trigeminal nerve;Long-term durability: One-time administration of gene therapy to eliminate the need for cycling through anti-epileptic medications and surgical procedures; andRegulatable activity: Targeted delivery to the trigeminal nerve, additionally engineered with the ability to “turn off” the gene therapy through withdrawal of the orally-administered small molecule, varenicline. Focal Epilepsy: KRIYA-382 is designed as a one-time gene therapy expressing a chemogenetically-gated ion channel, delivered through a direct injection into epileptic foci within the brain with the objective of reducing the frequency and severity of seizures. Kriya’s epilepsy gene therapy uses a similar chemogenetic technology as is utilized in its trigeminal neuralgia program, described above. Focal epilepsy affects close to 5 million people in the United States and the European Union. Current treatments include first- and second-line antiseizure medications. A significant proportion of patients suffer from refractory epilepsy, many of whom are candidates for invasive neurosurgery. Kriya has designed KRIYA-382 for focal epilepsy with the following potential goals in mind: Seizure reduction: Goal of minimizing seizures by inhibiting neuronal hyperexcitability at epileptic foci;Long-term durability: One-time administration of gene therapy designed to eliminate the need for cycling through anti-epileptic medications; andRegulatable activity: Targeted delivery to epileptic foci, additionally engineered with the ability to “turn off” the gene therapy through withdrawal of the orally-administered small molecule, varenicline. For more information on Kriya's Neurology programs, please visit the Neurology page of our website. About Kriya TherapeuticsOur mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease. Kriya is a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $600 million, which will be used to advance a broad pipeline of gene therapies for ophthalmology, metabolic disease and neurology. For more information, please visit www.kriyatx.com and follow us on LinkedIn and X.

All human beings are genetically very similar, sharing approx. 99.9% of the DNA code. The remaining 0.1% explains the natural differences between people, including our predisposition to hereditary diseases. Although sequencing of our genetic material is becoming a routine diagnostic analysis, it is unfortunately far from simple to determine whether specific small differences in our DNA affect our risk of developing disease. The usefulness of DNA sequencing is therefore often limited to the few cases where it is already known if a gene variant increases the risk of disease. Researchers at the Department of Biology, University of Copenhagen, have now contributed to solving this problem for a specific gene called GCK. The study has just been published in Genome Biology. Figure: GCK gene Rasmus Hartmann-Petersen, Professor at the Department of Biology, explains: - “The GCK gene, which codes for the enzyme glucokinase, regulates the secretion of insulin in the pancreas. GCK gene variants can therefore cause a form of hereditary diabetes. Although the connection between GCKand diabetes has been known for several years, we have, until now, only known the effect of a few percent of the possible variants of this gene”. Together with colleagues at the PRISM centre, UCPH, who are currently studying the effects of genetic variations, the researchers measured the effect of all of the possible variants of GCK. PhD student Sarah Gersing, who is the first author of the article, explains: - “We used yeast cells to measure the activity of over 9000 different GCK variants. In this way, we were able to generate a list of the effects — both of already known variants, but also of variants that patients might carry, but that have not yet been discovered. This provides us with a reference for future GCK diagnostics”.  Prof. Kresten Lindorff-Larsen, who heads the PRISM centre, continues: - “Our results are quite unique; not only have we measured the effect of several thousand variants, but for many of the variants, we can now explain what they do to the glucokinase protein. In our centre, we have gathered researchers working across a range of research fields, bridging from data analysis and biophysics to cell biology and medicine, and it is now clear how this broad approach pays off in explaining how diseases arise”. Gene variants of GCK can, among other things, cause a form of hereditary diabetes called "GCK maturity onset diabetes of the young" (GCK-MODY). Professor of genetics, dr. med. Torben Hansen, who is also a member of the PRISM centre, says: - "Although GCK-MODY patients exhibit elevated blood glucose levels, this is often not associated with complications. Hence, unlike other forms of diabetes, most GCK-MODY patients might therefore not need to be treated with medication. However, due to missing or inaccurate genetic data, more than half of the GCK-MODY patients are classified with having either type 1 or type 2 diabetes – and are therefore unnecessarily medicated. We estimate that approx. 1% of those who have recently been diagnosed with type 2 diabetes in Denmark have a variant in the GCK gene, meaning that they don’t need treatment, or need to be treated differently. Our new map of GCK variants can hopefully help give these patients a more correct diagnosis.” The next step for PRISM is to transfer these methods to other genes and diseases. - "We are already well underway with genes involved in e.g., neurodegenerative diseases, and we are trying to develop precise methods that can provide us with insights on disease mechanisms", says Rasmus Hartmann-Petersen.  Kresten Lindorff-Larsen continues: - "Our data gives us the opportunity to test and develop computational models for variant effects, which will then be transferable to other genes and diseases." - “Now, we have measured the effects of almost all variants of GCK, giving us knowledge on which variants that function, and which that do not. The next step is to understand why, and how the same underlying molecular mechanisms can give rise to a wide range of different diseases", concludes Sarah Gersing.

SHANGHAI, Feb. 16, 2023 /PRNewswire/ -- On February 15, 2023, 111, Inc. ("111" or the "Company") (NASDAQ: YI), a leading tech-enabled healthcare platform company in China, announced that during the HuaTangNing® Market Launch for Innovative Commerce Cooperation, Dr. Yu Gang, Co-founder and Executive Chairman of 111, and Dr. Chen Li, Founder and CEO of Hua Medicine (HKEx stock code: 2552.HK), jointly unveiled the plaque of Hua Medicine's first official online flagship store on 111. 111 is committed to digitally connecting patients with medicine and healthcare services, aspiring to build itself into an integrated online and offline healthcare platform. The official flagship store launched by Hua Medicine on 111 will serve as an official window to showcase the outstanding brand image of Hua Medicine and an important platform for Hua Medicine to better serve both patients and doctors. For its part, 111 will further help Hua Medicine expand its commercial layout, broaden its drug supply and distribution channels, and explore innovative and diversified supply service modes. The official flagship store will provide patients with Hua Medicine's innovative treatment concepts, carry out patient education on drug safety and other topics, and release the latest news from Hua Medicine. Dr. Yu Gang, Co-founder and Executive Chairman of 111, said: "Hua Medicine is a leader in innovative drug research and commercialization in China. Over the years, it has devoted itself to the research and development of original drugs. HuaTangNing®, a first-in-class drug of its kind, is from Hua Medicine. The official opening of its flagship store on 111 is a timely and innovative measure taken by a Chinese innovative pharmaceutical company to embrace the Internet. At the same time, it also marks a key milestone for 111 and Hua Medicine in the exploration of online cooperation. 111 will work with Hua Medicine to make cutting-edge innovative drugs more accessible for diabetes patients, empower diabetes treatment as well as patient management, and contribute value to society." Dr. Li Chen, Founder and CEO of Hua Medicine, said: "111 is a benchmark player in China's Internet medicine and healthcare industry, equipped with leading digital technology and smart supply chain. We are very much looking forward to Hua Medicine's cooperation with 111, as we provide innovative solutions of health management and medical services through the online flagship store. In the future, Hua Medicine will continue to explore partnership opportunities with Internet channels to benefit more diabetic patients." About Hua Medicine Hua Medicine is an innovative drug development and commercialization company based in Shanghai, China, focused on developing novel therapies for patients worldwide with unmet medical needs. Based on global resources, Hua Medicine teams up with global high-caliber people to develop breakthrough technologies and products, which contribute a global innovation in diabetes care. As Hua Medicine's cornerstone product (dorzagliatin tablets), targeting the glucose sensor, glucokinase, restores glucose sensitivity in T2D patients and stabilizes the imbalance of blood glucose levels in patients, it has been approved by the National Medical Products Administration (NMPA) of China in September 2022. It can be used alone or in combination with metformin hydrochloride-tolerated T2D patients. For those patients with chronic kidney disease (CKD) and Type 2 diabetes (i.e., diabetes kidney disease), no dose adjustment is required. About 111, Inc. 111, Inc. (NASDAQ: YI) ("111" or the "Company") is a leading tech-enabled healthcare platform company committed to digitally connecting patients with medicine and healthcare services in China. The Company provides consumers with better access to pharmaceutical products and healthcare services directly through its online retail pharmacy, 1 Pharmacy, and indirectly through its offline virtual pharmacy network. The Company also offers online healthcare services through its internet hospital, 1 Clinic, which provides consumers with cost-effective and convenient online consultation, electronic prescription service, and patient management service. In addition, the Company's online platform, 1 Medicine, serves as a one-stop shop for pharmacies to source a vast selection of pharmaceutical products. With the largest virtual pharmacy network in China, 111 enables offline pharmacies to better serve their customers with cloud-based services. 111 also provides an omni-channel drug commercialization platform to its strategic partners, which includes services such as digital marketing, patient education, data analytics, and pricing monitoring. For more information on 111, please visit: . Forward-Looking Statements This press release contains forward-looking statements. These statements constitute "forward-looking" statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and as defined in the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements can be identified by terminology such as "will," "expects," "anticipates," "future," "intends," "plans," "believes," "estimates," "target," "confident" and similar statements. Among other things, the Business Outlook and quotations from management in this announcement, as well as 111's strategic and operational plans, contain forward-looking statements. 111 may also make written or oral forward-looking statements in its periodic reports to the U.S. Securities and Exchange Commission, in its annual report to shareholders, in press releases and other written materials and in oral statements made by its officers, directors or employees to third parties. Such statements are based upon management's current expectations and current market and operating conditions and relate to events that involve known or unknown risks, uncertainties and other factors, all of which are difficult to predict and many of which are beyond the Company's control. Forward-looking statements involve inherent risks, uncertainties and other factors that could cause actual results to differ materially from those contained in any such statements. Potential risks and uncertainties include, but are not limited to, uncertainties as to the Company's ability comply with extensive and evolving regulatory requirements, its ability to compete effectively in the evolving PRC general health and wellness market, its ability to manage the growth of its business and expansion plans, its ability to achieve or maintain profitability in the future, its ability to control the risks associated with its pharmaceutical retail and wholesale businesses, and the Company's ability to meet the standards necessary to maintain listing of its ADSs on the Nasdaq Global Market, including its ability to cure any non-compliance with Nasdaq's continued listing criteria. Further information regarding these and other risks, uncertainties or factors is included in the Company's filings with the U.S. Securities and Exchange Commission. All information provided in this press release is as of the date of this press release, and 111 does not undertake any obligation to update any forward-looking statement as a result of new information, future events or otherwise, except as required under applicable law. SOURCE 111, Inc.