Oryzon Genomics SA

ORYZON reports financial results and corporate update for quarter ended September 30, 2024 Read below the full press release. #oryzon #epigenetics #iadademstat #vafidemstat #LSD1

Entrevista a nuestro CEO, Carlos Buesa, en Negocios Televisión esta semana

Today, on International Kabuki Syndrome Day, we raise awareness for Kabuki Syndrome (KS), supporting those affected and their families, and advocating for greater understanding, research and support. 🌍💚   🧬 KS is a rare genetic disorder that affects approximately 1 in 32,000 children worldwide, and is caused by mutations in two known genes with complementary functions: KMT2D and KDM6A. KMT2D mutations, responsible for over 70% of KS cases, lead to KS Type I. KS can result in failures of several organ systems, including the neurological, immune, auditory and cardiac systems.   🎯 Our research focuses on developing LSD1 inhibitors, which have shown promise as potential treatments for KS. By targeting the epigenetic enzyme LSD1, LSD1 inhibitors can restore the unbalanced methylation status in the brain, which is crucial in conditions like KS. Using a KS mouse model with a KMT2D mutation, researchers at Johns Hopkins University have demonstrated that inhibition of LSD1 can restore adult neurogenesis, improve memory deficits, and normalize immune defects.   At Oryzon, we remain committed to advancing precision medicine and developing innovative therapies for rare diseases like KS. In collaboration with our partners, we strive to make progress in understanding this condition and continue to support awareness and research efforts.   #KabukiSyndrome #Oryzon #KabukiSyndromeAwareness #LSD1 #vafidemstat

🌍 Oryzon is proud to join the global awareness campaign for Phelan-McDermid Syndrome (PMS) on this International Phelan-McDermid Syndrome Awareness Day. PMS is a rare genetic disorder caused by a deletion or variation of the long arm of chromosome 22 in the 22q13 region, most often involving the SHANK3 gene. PMS presents with a range of symptoms, including intellectual disability, delay or absence of speech, low muscle tone, and autistic features. Around 75-80% of individuals with PMS are also diagnosed with an autism spectrum disorder.   🔬 Recent research has highlighted the involvement of LSD1 in PMS. In animal models defective for Shank3 which recapitulate many symptoms of the human syndrome, LSD1 inhibitors were able to restore neuronal electrophysiology and rescue learning deficits. This finding opens the possibility to explore vafidemstat, our clinical-stage LSD1 inhibitor for CNS disorders, as a potential treatment for PMS.   🤝 In collaboration with the Institute of Medical and Molecular Genetics (INGEMM) at the La Paz Hospital in Madrid, we have conducted an observational trial aimed at psychometrically characterizing individuals carrying deletions or pathogenic variants in SHANK3, to lay the groundwork for a potential precision medicine clinical trial with vafidemstat in PMS. A scientific article discussing the results of this observational trial has recently been submitted for publication.   Together, let's continue to raise awareness and support research efforts towards potential treatments that could improve the lives of individuals with PMS and their families.   #PhelanMcDermidSyndrome #PMSAD #Oryzon #vafidemstat #LSD1 #epigenetics #INGEMM

ORYZON announces formal grant of key European patent for vafidemstat. Read below the full press release. #oryzon #epigenetics #iadademstat #vafidemstat #LSD1

🌍🧠 Oryzon Genomics is proud to stand in solidarity with those affected by psychiatric disorders on this World Mental Health Day. As a company committed to advancing mental health research and treatments, we are pleased to announce that we have received feedback from the FDA supporting the initiation of our Phase III PORTICO-2 trial. This trial will investigate vafidemstat, our LSD1 inhibitor, as a potential treatment for Borderline Personality Disorder (BPD).   🎯 BPD is a complex disorder with significant unmet medical needs, and currently, there are no FDA-approved treatments specifically for this condition. Our recent Phase IIb PORTICO trial showed encouraging results, particularly in reducing agitation and aggression, which are core symptoms of BPD. With the FDA's feedback, we are now preparing to advance vafidemstat into Phase III trials, aiming to bring a much-needed treatment option to those affected by BPD.   🔬 In addition to BPD, vafidemstat also shows potential in treating other conditions, such as schizophrenia. We remain committed to leveraging our expertise in epigenetics to develop effective therapies for various psychiatric disorders.   💪💙 On this World Mental Health Day, we reaffirm our dedication to supporting global research efforts and improving the lives of those living with psychiatric conditions. Together, we can work towards a future where mental health is prioritized and adequately addressed.   #WorldMentalHealthDay #MentalHealthAwareness #Oryzon #Vafidemstat #BPD #Schizophrenia #ClinicalResearch #Epigenetics

ORYZON receives minutes from End-of-Phase II meeting with the FDA on the PORTICO-2 Phase III vafidemstat trial in Borderline Personality Disorder. Read below the full press release. #oryzon #epigenetics #iadademstat #vafidemstat #LSD1

ORYZON presents the final data from PORTICO, a global Phase IIb vafidemstat trial in Borderline Personality Disorder (BPD), at the European College of Neuropsycopharmacology (ECNP) congress. Read below the full press release. #oryzon #epigenetics #iadademstat #vafidemstat #LSD1

🧠 September is Charcot-Marie-Tooth (CMT) Awareness Month, so let’s use this opportunity to educate the global community about CMT and the need to increase research efforts to find treatments for this disease. It’s time to say #LetsBeatCMT.   🌍 🔬 CMT is a group of inherited disorders that cause peripheral nerve damage, leading to muscle weakness, atrophy, and sensory loss, profoundly impacting the lives of those who live with it. There is no known cure for CMT. At Oryzon, we are committed to advancing research and developing innovative therapies for this often-overlooked neurological disorder.   🎯 ORY-4001, our highly selective histone deacetylase 6 (HDAC-6) inhibitor, has been nominated as a clinical development candidate for the treatment of CMT and other neurological diseases like amyotrophic lateral sclerosis. ORY-4001 is progressing through IND-enabling studies that will prepare the compound for clinical trials.   This month, we stand in solidarity with the CMT community and reaffirm our dedication to pushing the boundaries of science to find better treatments.   Let’s work together to raise awareness and support CMT research.   #CMTAwarenessMonth #Oryzon #CharcotMarieTooth #CMT #ORY-4001 #epigenetics