Kriya Therapeutics, Inc.

– Results demonstrate statistically significant fibrosis reversal and protection against hepatocellular carcinoma in animal models of MASH – – Kriya plans to evaluate KRIYA-497, a one-time intramuscular gene therapy designed to enable continuous expression of native FGF21, in people with MASH – Nov. 13, 2024 -- Kriya Therapeutics, Inc., ("Kriya"), a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world, today announced the publication of preclinical data for its investigational AAV-FGF21 gene therapy, generated by researchers at the Universitat Autonoma de Barcelona (UAB), Spain, in Molecular Therapy, the founding journal of the American Society of Gene & Cell Therapy (ASGCT). The results underscore the potential of KRIYA-497, an investigational, intramuscularly-administered AAV gene therapy designed to express native fibroblast growth factor 21 (FGF21) protein. Other FGF21-based therapies have clinically demonstrated beneficial metabolic effects across several target organs including the liver. “We are thrilled with the latest published data on our FGF21 gene therapy program, which has demonstrated fibrosis reversal and protection against hepatocellular carcinoma in translatable animal models of MASH," said Shankar Ramaswamy, M.D., Co-Founder and CEO of Kriya. "MASH represents one of the most significant chronic diseases of our time, and patients are in desperate need of additional safe, effective and durable treatment options. We are excited to advance a potential one-time gene therapy to address this debilitating disease.” The UAB-based authors of the article, titled "Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy", noted the following: One-time intramuscular administration of AAV1-FGF21 in obese male and female mice resulted in sustained increased circulating levels of FGF21; The long-term treatment resulted in complete reversal of hepatic fibrosis while halting the development of liver tumors in animals followed for over 9 months following gene therapy treatment; AAV-FGF21 treatment also counteracted obesity, adiposity and insulin resistance in animals, which are significant drivers of MASH; and The same treatment in dogs resulted in durable protein expression and biological activity in key metabolic tissues. The Online First edition of the article can be found here. "These results provide strong support for the durability and efficacy in animals of a one-time intramuscular AAV-FGF21-based gene therapy," said Fátima Bosch, Ph.D., Professor in Biochemistry and Molecular Biology at UAB, Kriya Scientific Advisory Board Member, and director of the study. "Based on this research, we are very excited about the potential to impact the lives of people with MASH, and we are hopeful that this approach may someday be a major breakthrough in the treatment of MASH and its related comorbidities." MASH is a condition in which excess fat deposits in the liver cause inflammation, cell damage and fibrosis. Approximately 20 percent of people with MASH will progress to advanced liver disease and associated complications, including cirrhosis and cancer. MASH affects approximately 40 million people in the United States and European Union. There is currently only one FDA approved treatment for MASH, with significant unmet need remaining to be addressed. Our 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. The content above comes from the network. if any infringement, please contact us to modify.

– Preclinical data demonstrate pharmacodynamic activity of KRIYA-586, an investigational gene therapy which encodes for an anti-IGF1R antibody – – Kriya anticipates advancing KRIYA-586 into the clinic in 2025 to evaluate its safety and efficacy in people suffering from TED – Sept. 12, 2024 -- Kriya Therapeutics, Inc., (Kriya), a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world, today announced preclinical data from its gene therapy program for thyroid eye disease (TED). The data are being presented at the 42nd Annual Meeting of the European Society of Ophthalmic Plastic & Reconstructive Surgery (ESOPRS) being held September 12-14 in Rotterdam, the Netherlands. KRIYA-586 is an investigational adeno-associated virus (AAV) gene therapy product engineered to express an antibody that inhibits Insulin-Like Growth Factor 1 Receptor (IGF1R), a key target implicated in the pathogenesis of TED. Currently, the only medicine approved in the U.S. for the treatment of TED is an intravenously infused human monoclonal antibody that blocks IGF1R. KRIYA-586 is designed for one-time delivery through a focal peribulbar injection performed in the office—with the goal of providing local and durable expression of anti-IGF1R antibody from transduced periorbital fat and muscle cells to help address the ocular manifestations of TED while minimizing systemic toxicities. “We are excited to present these preclinical results that demonstrate the robust activity of KRIYA-586 in blocking IGF1R activity,” said Shankar Ramaswamy, M.D., Co-Founder and CEO of Kriya. “These data support the potential of KRIYA-586 to address the unmet medical needs in thyroid eye disease, and we look forward to advancing KRIYA-586 into the clinic in 2025.” The Kriya ESOPRS presentation, “Activity of an adeno-associated virus (AAV)-derived anti-Insulin-like Growth Factor 1 Receptor (IGF1R) in vitro and pharmacodynamic activity in an IGF1R+ Human Colon Carcinoma Xenograft Mouse Model”, is outlined below. demonstrate in vitro secretion of a human antibody with anti-IGF1R activity, and compare in vivo pharmacodynamic activity of the anti-IGF1R antibody (produced by KRIYA-586) to weekly teprotumumab administration in a humanized xenograft mouse model. A vectorized antibody capable of blocking IGF1R to a similar degree as teprotumumab; and In a preclinical in vivo model, anti-IGF1R antibody produced by KRIYA-586 demonstrated a similar reduction in levels of IGF1R and downstream activity as teprotumumab. To date KRIYA-586 has been characterized in multiple in vitro and in vivo pharmacodynamic models, including the study described above. Kriya has designed KRIYA-586 for TED with the following potential goals: One-time administration: One-time, in-office peribulbar injection to eliminate the burdensome requirement for multiple intravenous infusions. Focal delivery: Localized antibody expression in extraocular muscles and fat tissue, with low systemic exposure to limit the potential for side effects. Long-term durability: AAV-mediated anti-IGF1R expression to deliver sustained improvements in proptosis and diplopia as well as other key manifestations of Thyroid Eye Disease. KRIYA-586 has not been approved for use by the U.S. Food and Drug Administration. The clinical safety and efficacy of KRIYA-586 for the treatment of Thyroid Eye Disease has not yet been established. TED is an autoimmune disease that causes inflammation and enlargement of the muscle and fat tissue behind the eye. This results in proptosis and diplopia which can significantly impact quality of life of patients with TED. The only FDA approved therapy for the treatment of TED is an intravenously administered monoclonal antibody that works by blocking IGF1R. There is a need for safe and durable treatments that can address the approximately one million patients in the United States and the European Union who have TED. Our 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. The content above comes from the network. if any infringement, please contact us to modify.

Nicole Bean for BioSpace With promising early results, cell and gene therapies are making headway against both rare and common ocular and auditory diseases. Eyes and ears. Two of our most critical sensory organs yet usually taken for granted—until something goes wrong. But while largely ignored by busy individuals and most insurance plans, biopharma R&D—specifically cell and gene therapy—is paying attention. Let’s start with hearing. The human ear is made up of three different parts: the outer ear, middle ear and inner ear—a complex structure offering many opportunities for something to go wrong. Hearing Loss Breakthroughs This spring, Regeneron made headlines when it announced that the investigational gene therapy DB-OTO improved hearing in two young children with “profound genetic deafness” since birth, with one patient returning to normal hearing levels within six months of treatment. In this case, the children’s hearing loss was due to a deficiency in otoferlin, a protein expressed in the hair cells of the inner ear. Regeneron’s gene therapy delivers a functioning copy of the gene to the inner ear, stimulating otoferlin production. Eli Lilly subsidiary Akouos and Fudan University in Shanghai and Mass Eye and Ear have also enjoyed early success with otoferlin gene therapies . Of course, these therapies will only help the 1% to 8% of patients afflicted by this particular genetic mutation. However, according to the American Hearing Research Foundation , about 75% of congenital deafness has a genetic cause, indicating the potential for more gene therapies. Although I can hear perfectly well, hearing loss is a condition with which I am quite familiar. My grandfather was born deaf. He never knew the cause but, today, he could have had his DNA tested and had the hope of a treatment on the horizon. Ocular CGT Advances On the ocular side, cell and gene therapies are making progress against more common diseases like wet age-related macular degeneration (AMD), a leading cause of blindness, as well as dry eye syndrome (DED)—and here my interest is even more personal. Four years ago, I began to experience a scratchy, gritty sensation in my eyes. This quickly graduated to a burning, sandpaper-dry condition that makes everyday tasks—like driving a car or staring at a computer screen—challenging. I’m far from the only one dealing with these issues. I’ve commiserated with at least two colleagues, which isn’t surprising as DED affects around one in 11 people worldwide, according to the NIH. After exhausting the first tier of treatments—such as prescription eye drops, omega-3 fatty acids, intense pulsed light therapy and scleral contact lenses—I learned about a cellular therapy called amniotic membrane treatment . Extracted from the innermost layer of the human placenta, amniotic membrane is full of neurotrophic factors such as nerve growth factor, collagen and basal epithelial cells. It also contains unique anti-inflammatory, anti-fibrotic and pro-healing properties that can help ease conditions like DED, corneal neuropathic pain, graft versus host disease and post-corneal transplants, according to an article published in Ocular Surface . A 2018 study found that 74 of 84 patients enjoyed an improved ocular surface and a “notable” reduction in symptom severity after just over a week of treatment with amniotic membrane. As with many therapies, it doesn’t always work the first time; 10% of eyes in this study required repeated treatment, and I personally am on my second round. Interestingly, this study breaks the data down by individual eyes, as patients did not always have the same response in both eyes. I have so far experienced slightly better results in my right eye from this second round of treatment. Companies are also making progress on the gene therapy side, specifically for wet AMD . REGENXBIO and AbbVie, Adverum and 4D Molecular Therapeutics have all seen positive results from their investigative gene therapies for this condition, which affects around 1.5 million people in the U.S. alone—a far bigger patient population than current rare disease gene therapies target. The mechanism for these wet AMD treatments is also different from traditional gene therapies, which work by repairing or replacing a faulty disease-causing gene. For wet AMD, investigational gene therapies deliver the coding sequence for the therapeutic molecule to the eye, which serves as a biofactory to produce the medicine. This could ease the treatment burden of the multiple injections currently required to treat the disease. Several other companies, including AbbVie and Capsida Biotherapeutics , Kriya Therapeutics and SpliceBio , are developing gene therapies to treat thyroid eye disease, inherited retinal disease and more. Having been treated with one of these therapies, I can also attest to the fact that they are not cheap. But progress is being made. While gene therapies for rare diseases that correct or replace a faulty gene can carry a multi-million-dollar price tag, experts say the cost of the wet AMD treatments currently in development will be much lower. There is definitely a ray of light and a joyful noise on the horizon.