Gross

Myrtelle to Present Positive 6-month Post-Treatment Data in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay-Sachs and Allied Diseases Annual Family Conference

Retrieved on: 
Wednesday, May 31, 2023
Neurology, Biotechnology, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Other Health, CD, Gross, Canavan disease, Inc., Drug development, Natural history, IV, Clinical trial, Mutation, Human, Brain, ASPA, AAV, Doctor of Philosophy, Language, NAA, System, MRI, MRS, Magnetic resonance imaging, Metabolism, MD, Patient, Preyasi Raave, White matter, CSF, FIH, Conference, Caregiver, Vaccine, Medical imaging, Myrtelle Canavan

Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.

Key Points: 
  • Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients in Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
  • “Connecting with patients and caregivers allows us to incorporate the patient voice into our drug development activities.”

Myrtelle Announces Positive 6-month Post-Treatment Data in Patients in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease

Retrieved on: 
Wednesday, April 19, 2023
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, CD, Gross, Canavan disease, Inc., Natural history, Boonshoft School of Medicine, In vivo magnetic resonance spectroscopy, OH, Clinical trial, Mutation, Neuroscience, Brain, ASPA, Cerebrospinal fluid, Language, Assistant, NAA, Chromosome 11, MRI, MRS, Magnetic resonance imaging, Metabolism, MD, Patient, Wright, N-Acetylaspartic acid, Preyasi Raave, Annual general meeting, Neurology, White matter, CSF, FIH, Medical imaging, Medicine

In several domains, the observed improvement in treated patients are in contrast to the observed deterioration in untreated age-matched CD patients within Myrtelle’s natural history data set.

Key Points: 
  • In several domains, the observed improvement in treated patients are in contrast to the observed deterioration in untreated age-matched CD patients within Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme Aspartoacylase (ASPA).
  • The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function and hence the metabolism of NAA and brain development in patients with CD.
  • In addition, volumetric MRI (magnetic resonance imaging) measurements showed increases in multiple brain tissue compartments and reductions in CSF volume.

Myrtelle Announces Positive Interim Data in Phase 1/2 Clinical Trial of Its Proprietary Investigational Gene Therapy rAAV-Olig001-ASPA in Canavan Disease

Retrieved on: 
Monday, January 9, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, White matter, CSF, Clinical trial, MRI, Associate, Gross, N-Acetylaspartic acid, Food, Cerebrospinal fluid, Wright, European Medicines Agency, MD, Boonshoft School of Medicine, Inc., ASPA, CD, Metabolism, FIH, NAA, Patient, Myrtelle Canavan, Pediatrics, Classification, US Foods, Mutation, Canavan disease, Magnetic resonance imaging, Rita Raave, Orphan drug, Hospital, Fast Track, Neurodegeneration, Pharmaceutical industry, Medical imaging

Early data from the study, being conducted at Dayton Children’s Hospital (Dayton, Ohio), at three months following gene therapy treatment have shown encouraging results to date.

Key Points: 
  • Early data from the study, being conducted at Dayton Children’s Hospital (Dayton, Ohio), at three months following gene therapy treatment have shown encouraging results to date.
  • The observed improvements following gene therapy are in contrast to the continuous clinical decline expected for untreated CD patients.
  • The first three patients treated with the gene therapy in cohort one are now at least 18 months post therapy.
  • “The early results to date in patients treated in Myrtelle’s Phase 1/2 clinical trial are encouraging.

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022
Human voice, Deafness, MT-TL1, Learning, Mitochondrial disease, Cell, Cognition, Enzyme, Clinician, Natural history study, Mitochondrial encephalomyopathy, RAND, Leigh syndrome, Conference, Neuro, Pain, Degenerative disease, Fatigue, Exercise, CFQ, Growth, BDI, DNA, Life, Phase, Lactic acidosis, Global health, Jungian cognitive functions, Diabetes, Research, Severe cognitive impairment, Safety, Gross, Pharmacokinetics, Mutation, Heart failure, Beck, Mitochondrion, Muscle weakness, Government, Point mutation, Collection, Quality of life, Beck Depression Inventory, IIB, Pharmaceutical industry, Medical imaging, Medical device, Patient, MELAS, MIDD

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022
Human voice, Deafness, MT-TL1, Learning, Mitochondrial disease, Cell, Cognition, Enzyme, Clinician, Natural history study, Mitochondrial encephalomyopathy, RAND, Leigh syndrome, Conference, Neuro, Pain, Degenerative disease, Fatigue, Exercise, CFQ, Growth, BDI, DNA, Life, Phase, Lactic acidosis, Global health, Jungian cognitive functions, Diabetes, Research, Severe cognitive impairment, Safety, Gross, Pharmacokinetics, Mutation, Heart failure, Beck, Mitochondrion, Muscle weakness, Government, Point mutation, Collection, Quality of life, Beck Depression Inventory, IIB, Pharmaceutical industry, Medical imaging, Medical device, Patient, MELAS, MIDD

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

Stantec awarded US$20 million contract for Gross Reservoir Expansion Project

Retrieved on: 
Thursday, October 6, 2022
Quality of life, News, STN, Stantec, Engineering, Risk, TSX, NYSE, Drought, Denver Water, Engineering News-Record, Â, Moffat Tunnel, US, Water, Nature, Social media, GRE, GLOBE, Gross, Forward-looking statement, Creativity, Engineer, Federal Energy Regulatory Commission, Oil, Dam, Risk management

The GRE project aims to provide water security to more than 1.5 million residents in Denver Waters service area.

Key Points: 
  • The GRE project aims to provide water security to more than 1.5 million residents in Denver Waters service area.
  • Stantec was awarded the design services contract for the first phase of the project in 2017, and once expanded, the dam will be the largest dam raise using a roller-compact concrete system in the world.
  • Stantecs US$20 million construction phase contract will include owner support, contractor quality reviews, and regulatory compliance.
  • The GRE project received approval from the Colorado State Engineers Office and Federal Energy Regulatory Commission in early 2022.

Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA

Retrieved on: 
Tuesday, October 4, 2022
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, Classification, Time, Canavan disease, Association, Walking, ATMP, Clinical trial, Magnetic resonance, Spasticity, Gross, Inc., FDA, Brain, Patient, MRI, MPH, Pfizer, N-Acetylaspartic acid, Mutation, FIH, Irritability, CA, CD, Safety, Kaay Raav Tumhi, Regenerative medicine, Mesa, ASPA, Magnetic resonance imaging, Gene, European Medicines Agency, Fast Track, Forward-looking statement, NAA, Neurodegeneration, Metabolism, Seizure, EMA, Intellectual property, Company, Pharmaceutical industry, Medical imaging, MD, Aspartoacylase, Myrtelle Canavan

Myrtelle Inc. (Myrtelle or the Company), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced the completion of dosing of 8 patients with the Companys recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD).

Key Points: 
  • Myrtelle Inc. (Myrtelle or the Company), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced the completion of dosing of 8 patients with the Companys recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD).
  • The oligodendrocyte targeting rAAV vector-based gene therapy is intended to restore ASPA function and hence the metabolism of NAA and myelination in patients with CD.
  • Additionally, observations of these patients at 6 months post-treatment using Magnetic Resonance Imaging (MRI) demonstrated increases in brain white matter and myelin content.
  • Observed improvements in these treated patients are in contrast to the continuous clinical deterioration expected with the natural progression of CD.

Myrtelle Announces Positive Data for Its investigational Proprietary rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay Sachs & Allied Diseases Association Conference

Retrieved on: 
Friday, July 8, 2022
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, Cell, FIH, Inc., Mutation, Science, Intracerebroventricular injection, Boonshoft School of Medicine, Patient, ICV, Magnetic resonance imaging, Brain, Trial of the century, Walking, Metabolism, Disease, Intellectual property, White matter, Irritability, Forward-looking statement, Spasticity, Magnetic resonance, ASPA, Gross, Canavan disease, Kaay Raav Tumhi, MPH, Pfizer, N-Acetylaspartic acid, Clinical trial, Wright, OH, Doctor of Philosophy, Seizure, Pediatrics, CD, Time, Associate, Safety, MRI, VG, Program, NAA, Neurodegeneration, Conference, Pharmaceutical industry, Medical imaging, Canavan, Myrtelle Canavan, Aspartoacylase, MD

In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA).

Key Points: 
  • In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA).
  • These improvements suggest the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease.
  • Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases.
  • The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases.

Bill and Amy Gross Announce Final City Approval of Laguna Beach Artwork

Retrieved on: 
Tuesday, June 7, 2022
Janus, Bill H. Gross, Fixed income, I'm Still Standing, California Coastal Commission, Distinguished Service Award, PIMCO, Gross, Bond Market Association, Institutional Investor, City, Money Management, Compliance, Amazon (company), Education, Online gambling, Towfiq Khan

LAGUNA BEACH, Calif., June 7, 2022 /PRNewswire/ -- Bill and Amy Gross are pleased to announce the final approval by the City of Laguna Beach of their Dale Chihuly glass sculpture following almost two years of protracted and unnecessary litigation and conflict.

Key Points: 
  • LAGUNA BEACH, Calif., June 7, 2022 /PRNewswire/ -- Bill and Amy Gross are pleased to announce the final approval by the City of Laguna Beach of their Dale Chihuly glass sculpture following almost two years of protracted and unnecessary litigation and conflict.
  • "Amy and I are relieved we can finally enjoy our art in peace and in full compliance with the City of Laguna Beach," said Bill Gross, a legendary bond and fixed income investor, and co-founder of Newport Beach-based Pimco.
  • The approval by the Laguna Beach Design Review Board and City of Laguna Beach exposes their true intent in this rather ludicrous situation."
  • The City of Laguna Beach granted final approval of the Gross's artwork following the withdrawal of an appeal filed by Towfiq and Nakahara to the California Coastal Commission.

Illinois Manufacturers' Association and Illinois Manufacturing Excellence Center Form Strategic Partnership

Retrieved on: 
Thursday, March 31, 2022
Illinois Manufacturers' Association, Organization, Partnership, NIST, Leadership, Automation, Growth, Multimedia, MEP, Association, Doctor of Philosophy, Woman, CEO, Man, Workforce, Employment, Customer engagement, Industry, IMA, IMEC, Solution, Gross, Oil, Management, Clothing, Collaboration, Technology

SPRINGFIELD, Ill., March 31, 2022 /PRNewswire/ -- On the heels of the global pandemic in which American and Illinois manufacturers led our nation forward through an economic and health crisis, the Illinois Manufacturers' Association (IMA) and Illinois Manufacturing Excellence Center (IMEC) are announcing an historic formal strategic partnership that will strengthen and build upon Illinois' rich manufacturing legacy and position the industry for future growth.

Key Points: 
  • "Today's announcement represents a historic partnership of the two leading manufacturing organizations in Illinois that will allow us to leverage our organizational strengths to grow and expand our state's manufacturing footprint."
  • "By joining forces, IMEC and the IMA will provide Illinois' manufacturing sector with the full range of products and services in a more efficient and cost-effective way," said Dave Boulay, PhD, President of the Illinois Manufacturing Excellence Center.
  • "This great partnership will allow us to continue igniting Illinois manufacturing excellence and global competitiveness by delivering comprehensive proven solutions."
  • The Illinois Manufacturers' Association is the only statewide association dedicated exclusively to advocating, promoting and strengthening the manufacturing sector in Illinois.