Infantile neuroaxonal dystrophy

Charles River and INADcure Announce Gene Therapy Manufacturing Collaboration

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Thursday, June 8, 2023
Neurology, Biotechnology, Pharmaceutical, General Health, Health, Medical Devices, Genetics, Clinical Trials, GMP, Seizure, Good, Associate Director of National Intelligence and Chief Information Officer, DNA, Infantile neuroaxonal dystrophy, Plasmid, Good manufacturing practice, Therapy, Hearing loss, Neurodegenerative disease, Cognate, Display resolution, Mutation, Research, Life expectancy, Doctor of Philosophy, PLAN, HQ, CDMO, Patient, INAD, Drug discovery, Diagnosis, CRL, PLA2G6, Nerve, NYSE, Tecmar, Disease, Pharmaceutical industry, Vaccine

The collaboration will leverage Charles River’s market leading contract development and manufacturing organization (CDMO) expertise in High Quality (HQ) plasmid DNA production, to manufacture its leading candidate for Phase I/II clinical trials.

Key Points: 
  • The collaboration will leverage Charles River’s market leading contract development and manufacturing organization (CDMO) expertise in High Quality (HQ) plasmid DNA production, to manufacture its leading candidate for Phase I/II clinical trials.
  • Plasmid DNA is a critical starting material for many cell and gene therapy therapeutics and demand continues to outstrip supply.
  • In response to this, Charles River recently announced the opening of a state-of-the-art HQ plasmid manufacturing center of excellence to address these supply shortages and support the growing needs of the cell and gene therapy field.
  • Their invaluable expertise will undoubtedly contribute to the advancement of our gene therapy program.” - Leena Panwala, Founder & Executive Director at INADcure.

INADcure Foundation Launches Inaugural International Scientific Conference Specifically Focused On Innovative Discoveries And Accelerating Research Of Infantile Neuroaxon

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Thursday, November 4, 2021
Seizure, Neurodegeneration, Parkinson's disease, Industry, Government, Gaucher, Caregiver, Clinical trial, Alzheimer's disease, Glycogen storage disease, Organization, Disease, Chan Zuckerberg Initiative, Degenerative disease, Hearing loss, PLAN, Research, Foundation, Infantile neuroaxonal dystrophy, Conference, INAD, Awareness, The One Network, Risk, Gaucher's disease, Drug development, Pharmaceutical industry

The event, the International Scientific Conference on Infantile Neuroaxonal Dystrophy and other types of PLA2G6-Associated Neurodegeneration (PLAN) , will take place virtually on November 10-11, 2021.

Key Points: 
  • The event, the International Scientific Conference on Infantile Neuroaxonal Dystrophy and other types of PLA2G6-Associated Neurodegeneration (PLAN) , will take place virtually on November 10-11, 2021.
  • INAD is an ultra-rare disorder, with no known treatment or cure, affecting approximately 150 children worldwide.
  • The goal of the scientific conference is to advance research and build a collaborative community engaged in finding treatments and a cure.
  • Medical and scientific professionals in academia, government, biotech, and pharmaceutical industries can learn more about the scientific conference and see a draft agenda here .

Positive Results from Studies of Retrotope’s RT011 in Animal Models of Retinal Degeneration to be Featured in Oral Presentations at 2nd Annual Dry AMD Therapeutic Development Summit

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Tuesday, October 19, 2021
ALS, IND, Visual impairment, Tissue, Death, PUFA, Cell damage, Investigational New Drug, Western world, FA, Reading, Oxidative stress, GLOBE, Patient, University, INAD, Animal, RT001, LA, Retinal pigment epithelium, Demonstration, GA, AMD, Infantile neuroaxonal dystrophy, Collection, Amyotrophic lateral sclerosis, Time, DHA, University of Pennsylvania, Vicious Circle, RATS, Disease, Drusen, LPO, Time-invariant system, Retina, Dihydroxy-E,Z,E-PUFA, Geographic atrophy, Inflammation, Central retinal artery occlusion, Degenerative disease, Dry, Injection, Â, Severe cognitive impairment, RPE, Lipid peroxidation, LOS, Program, Research, Stress (linguistics), PSP, Therapy, Vaccine, Pharmaceutical industry, Dietary supplement

LOS ALTOS, Calif., Oct. 19, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced that positive results from preclinical studies of RT011, the second compound to emerge from the company’s novel technology platform, are being reported in multiple oral presentations at the 2nd Annual Dry AMD Therapeutic Development Summit. Findings to be presented demonstrate that RT011, which is being developed as an oral therapy for the treatment of dry age-related macular degeneration (AMD), provided dose-dependent protection in animal models of oxidative damage implicated in dry AMD. Importantly, study results showed that animals treated with RT011 experienced preservation of retinal cells, photoreceptors, and visual function relative to control animals. The conference, which is being conducted in a virtual format, is being held October 19-21, 2021.

Key Points: 
  • Findings to be presented demonstrate that RT011, which is being developed as an oral therapy for the treatment of dry age-related macular degeneration (AMD), provided dose-dependent protection in animal models of oxidative damage implicated in dry AMD.
  • Importantly, study results showed that animals treated with RT011 experienced preservation of retinal cells, photoreceptors, and visual function relative to control animals.
  • The company expects to file an Investigational New Drug (IND) application for RT011 in the first half of 2022 and initiate first-in-human clinical studies in dry AMD soon thereafter.
  • Study results demonstrated that animals treated with RT011 experienced decreasing levels of retinal damage with increasing tissue concentrations of RT011.

Retrotope Reports Data from Phase 2/3 Clinical Trial of RT001 and Concurrent Natural History Study in Patients with Infantile Neuroaxonal Dystrophy (INAD)

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Wednesday, October 6, 2021
Infantile neuroaxonal dystrophy, Food, PLA2G6, Infant, Degenerative disease, Failure, FA, University of California, Patient, NDA, Lipid peroxidation, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Therapy, RT001, Hypotonia, Amyotrophic lateral sclerosis, FDA, Disorder, Death, Spasticity, AMD, PSP, Dihydroxy-E,Z,E-PUFA, INAD, University, Food and Drug Administration, LPO, Neurology, Comparative effectiveness research, LA, Disease, Autonomic nervous system, New Drug Application, Pneumonia, HIV disease progression rates, Mitochondrial membrane transport protein, Technology, DHA, ALS, Research, Oxidative stress, Tissue, Brain, Genetics, Radical (chemistry), LOS, Survival, SAP, Membrane, Neurodegeneration, Totality, Seizure, GLOBE, Metabolism, Pharmaceutical industry

LOS ALTOS, Calif., Oct. 06, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today reported data from its Phase 2/3 clinical trial of RT001 in patients with infantile neuroaxonal dystrophy (INAD) and its concurrent natural history study of disease onset and progression in INAD patients. Results demonstrated statistically significant improvements in overall survival and progression free survival for patients treated with RT001 as compared to control. These survival endpoints included a combination of efficacy and safety measures. Additionally, clinical improvements were observed in patients receiving RT001 as measured by the Modified Ashworth Spasticity Scale, the study’s primary efficacy endpoint, and other measurements of efficacy as compared to patients in the natural history study. These single efficacy outcomes did not reach statistical significance likely due to the small study size.

Key Points: 
  • Results demonstrated statistically significant improvements in overall survival and progression free survival for patients treated with RT001 as compared to control.
  • The treatment study of RT001 included 19 INAD patients and the concurrent natural history study included 36 INAD patients as the control arm.
  • Patients in the treatment study received RT001 for a minimum period of one year with a 30-day treatment free follow up period.
  • Infantile Neuroaxonal Dystrophy is an ultra-rare, infantile genetic neurological disorder and part of a spectrum of diseases called PLA2G6-associated neurodegeneration.

Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Progressive Supranuclear Palsy (PSP)

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Wednesday, August 11, 2021
ALS, Hospital, Radical (chemistry), Amyotrophic lateral sclerosis, Dihydroxy-E,Z,E-PUFA, Speech, Health, Medulla oblongata, Walking, Safety, FA, Caregiver, Patient, Retrotope, Physician, Neurodegeneration, PSP, Lipid peroxidation, Technology, Hannover Medical School, Membrane, Massachusetts General Hospital, Death, Cognition, INAD, Tissue, HIV disease progression rates, LOS, Clinical trial, Brain, Behavior, Government budget balance, RT001, Massachusetts, LA, LPO, Research, Oxidative stress, Cell, Degenerative disease, AMD, Department of Neurology, Xuanwu hospital, History, FDA, Disease, GLOBE, Infantile neuroaxonal dystrophy, Mitochondrial membrane transport protein, Neurology, DHA, Pharmaceutical industry

PSP is a progressive, neurodegenerative disorder that dramatically impacts the health and lives of not only patients, but also their families and other caregivers.

Key Points: 
  • PSP is a progressive, neurodegenerative disorder that dramatically impacts the health and lives of not only patients, but also their families and other caregivers.
  • Study participants have been randomized to receive either RT001 or placebo daily for 48 weeks.
  • The primary endpoint of the trial is change from baseline in the PSP Rating Scale (PSPRS) at 48 weeks.
  • The study also includes several secondary and exploratory endpoints intended to further elucidate the efficacy and safety profile of RT001 as compared to placebo.

Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications

Retrieved on: 
Thursday, February 25, 2021
Rare diseases, Branches of biology, Clinical medicine, Health, Retrotope, Priority review, Di-deuterated linoleic acid ethyl ester, Food and Drug Administration, Infantile neuroaxonal dystrophy, Friedreich's ataxia, Fast track, Ifood

The first rare pediatric disease designation is for the treatment of infantile neuroaxonal dystrophy (INAD), with the second covering the treatment of Friedreichs ataxia (FA).

Key Points: 
  • The first rare pediatric disease designation is for the treatment of infantile neuroaxonal dystrophy (INAD), with the second covering the treatment of Friedreichs ataxia (FA).
  • To qualify for rare pediatric disease designation, these diseases must affect fewer than 200,000 people in the United States.
  • Retrotope is eligible to receive a priority review voucher should the company secure approval for RT001 in either one of these indications.
  • Recipients of Fast Track designation are eligible for greater access to the FDA during the clinical development process and may also qualify for priority review.

Dr. Dennis Gross Skincare Announces New Partnership With Bisous For Léo

Retrieved on: 
Wednesday, March 20, 2019
Rare diseases, Infantile neuroaxonal dystrophy

NEW YORK, March 20, 2019 /PRNewswire/ -- Dr. Dennis Gross Skincare announces its important partnership with Bisous For Lo , continuing its goal to positively impact families and communities through philanthropic efforts.

Key Points: 
  • NEW YORK, March 20, 2019 /PRNewswire/ -- Dr. Dennis Gross Skincare announces its important partnership with Bisous For Lo , continuing its goal to positively impact families and communities through philanthropic efforts.
  • Through this partnership, Dr. Dennis Gross Skincare seeks to expand awareness of Infantile Neuroaxonal Dystrophy (INAD), a rare genetic disease affecting children.
  • "Lo's story touched me on so many levels," Dr. Dennis Gross Skincare CEO Carrie Gross says.
  • "To say I'm grateful for Dr. Dennis Gross and his team's support of Bisous For Lo is an understatement," said Lo's mom Deborah Vauclare.