Mitochondrial disease

Juntendo University Broadens the Scope of Mitochondrial Disease Testing

Retrieved on: 
Monday, February 12, 2024

TOKYO, Feb. 12, 2024 /PRNewswire/ -- Mitochondrial disease occurs when malfunctioning mitochondria cannot sustain the production of adenosine triphosphate (ATP)—the energy currency of life—in the inner mitochondrial membrane. The loss of this vital molecule has far-reaching consequences that affect various organ systems and can cause psychiatric symptoms, cardiomyopathy, seizures, kidney and liver dysfunction, and hearing loss.

Key Points: 
  • The Intractable Disease Research Center at Juntendo University will conduct broader analyses of mitochondrial genetic data to confirm genetic diagnosis.
  • Unfortunately, mitochondrial disease is the most common congenital metabolic condition, with one case detected for every 5000 live births.
  • In terms of testing, Juntendo University screens many babies diagnosed with mitochondrial disease each year.
  • In this regard, Prof. Murayama's team commenced mitochondrial disease outpatient care at Juntendo Medical Center's Pediatric and Adolescent Department in July 2023.

Unicycive Therapeutics Issues Shareholder Letter to Highlight Corporate Progress and Key Upcoming Milestones

Retrieved on: 
Tuesday, January 23, 2024

LOS ALTOS, Calif., Jan. 23, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company or “Unicycive”), today issued a letter to shareholders.

Key Points: 
  • As I sit down to write this letter, I am filled with a profound sense of gratitude and excitement, energized for the year ahead.
  • In this letter, I am pleased to recap for you another year of great accomplishments.
  • We’ve made significant advancements in our clinical programs which will propel us to new heights in 2024.
  • I would like to express my deep appreciation to the physician investigators, study participants, and especially to our dedicated Unicycive employees.

Precision BioSciences Announces Publication in Nature Metabolism Supporting ARCUS® In Vivo Gene Editing as a Potentially Curative Treatment for Mitochondrial Diseases

Retrieved on: 
Monday, December 4, 2023

For mitochondrial diseases, what makes ARCUS such an elegant and simple tool is that it is a single protein that both recognizes and eliminates the mutant mitochondrial DNA,” said Jeff Smith, Ph.D., Chief Research Officer of Precision BioSciences.

Key Points: 
  • For mitochondrial diseases, what makes ARCUS such an elegant and simple tool is that it is a single protein that both recognizes and eliminates the mutant mitochondrial DNA,” said Jeff Smith, Ph.D., Chief Research Officer of Precision BioSciences.
  • “Today’s publication further validates the ability of ARCUS to overcome the limitations of CRISPR-based gene editing technologies for treating mitochondrial disease and eliminating mutated mitochondrial DNA with high specificity to improve overall mitochondrial function.
  • By eliminating the mutant mitochondrial DNA and allowing the wild-type mitochondrial DNA to repopulate, mitoARCUS drives a shift toward healthy mitochondrial DNA, a process known as shifting heteroplasmy.
  • “To date, there are no curative treatments for mitochondrial diseases, so a gene editing approach is a novel way to offer hope to the patients suffering from mitochondrial myopathy.

Owl Therapeutics Enters a Strategic Clinical Collaboration With Abliva to Advance NeuroSTAT® (OWL-1410) for Traumatic Brain Injury

Retrieved on: 
Friday, November 17, 2023

The collaboration agreement provides Owl Therapeutics with a global license to develop, manufacture, and commercialize NeuroSTAT®.

Key Points: 
  • The collaboration agreement provides Owl Therapeutics with a global license to develop, manufacture, and commercialize NeuroSTAT®.
  • "Abliva stands out as an exceptional partner for Owl Therapeutics, and we are excited to join forces in our collective commitment to advance NeuroSTAT® (OWL-1410)," said William E. Haskins, PhD, Co-Founder of Owl Therapeutics and Chief Executive Officer of Gryphon Bio.
  • "We are thrilled to focus our team's scientific, clinical, and business experience on this potentially game-changing treatment for military and civilian TBI patients."
  • Owl Therapeutics, a wholly-owned subsidiary of Gryphon Bio, is a clinical-stage biopharmaceutical company created to develop diagnostic-powered therapeutics for traumatic brain injury (TBI) and brain health.

The PolG Foundation awards over $3.5M in academic grants to pioneer POLG research

Retrieved on: 
Wednesday, November 15, 2023

Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.

Key Points: 
  • Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.
  • View Shop - https://shopmito.polgfoundation.org/
    A central pillar of the Foundation is to raise funding and provide grants to world-class investigators of basic science, clinical trial research and development of novel therapies for PolG mitochondrial disorders in both paediatric and adult PolG populations.
  • Today, The PolG Foundation is pleased to announce four awardees granted to our first Call for Research Initiatives proposals.
  • With your help and financial backing we can further our ambitious mission by discovering new therapies and a potential cure for PolG.

The PolG Foundation awards over $3.5M in academic grants to pioneer POLG research

Retrieved on: 
Wednesday, November 15, 2023

Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.

Key Points: 
  • Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.
  • View Shop - https://shopmito.polgfoundation.org/
    A central pillar of the Foundation is to raise funding and provide grants to world-class investigators of basic science, clinical trial research and development of novel therapies for PolG mitochondrial disorders in both paediatric and adult PolG populations.
  • Today, The PolG Foundation is pleased to announce four awardees granted to our first Call for Research Initiatives proposals.
  • With your help and financial backing we can further our ambitious mission by discovering new therapies and a potential cure for PolG.

Kevin Borg, PharmD, Honored as the 2023 PCCA Compounding Pharmacist of the Year

Retrieved on: 
Friday, October 27, 2023

Houston, Texas, Oct. 27, 2023 (GLOBE NEWSWIRE) -- PCCA has named Kevin Borg, PharmD, FAPC, FACA, owner of Potter’s House Apothecary in Peoria, Arizona, and Prescott Compounding Pharmacy in Prescott, Arizona, the 2023 M. George Webber, PhD, Compounding Pharmacist of the Year.

Key Points: 
  • Houston, Texas, Oct. 27, 2023 (GLOBE NEWSWIRE) -- PCCA has named Kevin Borg, PharmD, FAPC, FACA, owner of Potter’s House Apothecary in Peoria, Arizona, and Prescott Compounding Pharmacy in Prescott, Arizona, the 2023 M. George Webber, PhD, Compounding Pharmacist of the Year.
  • “Since becoming a member many years ago, he has relentlessly supported PCCA and the compounding profession — never hesitating to help a fellow compounder or participate in PCCA events.
  • The M. George Webber Pharmacist of the Year award is presented annually to an independent compounding pharmacist who demonstrates service excellence to patients, health care providers and pharmacy colleagues.
  • Borg expanded his compounding services with the opening of Prescott Compounding Pharmacy in Prescott, Arizona, in 2021.

Tisento Therapeutics Initiates Interview Study with Individuals with MELAS to Elucidate the Symptoms and Impact of Disease and Inform the Zagociguat Clinical Development Program

Retrieved on: 
Wednesday, October 25, 2023

CAMBRIDGE, Mass., Oct. 25, 2023 (GLOBE NEWSWIRE) -- Tisento Therapeutics, a privately held, clinical-stage biotechnology company developing novel medicines for diseases with significant unmet medical needs, today announced the initiation of a patient interview study to identify and describe the signs, symptoms, and health-related quality-of-life impacts of MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes) that are most burdensome to adults and adolescents living with this rare mitochondrial disease. The interviews will inform the selection of clinical outcomes assessments and endpoint strategy for the company’s planned Phase 2b study of its investigational medicine, zagociguat, in MELAS. 

Key Points: 
  • The interviews will inform the selection of clinical outcomes assessments and endpoint strategy for the company’s planned Phase 2b study of its investigational medicine, zagociguat, in MELAS.
  • This interview study is one way we will thoughtfully and intentionally listen to individuals with MELAS and take action in response,” said Peter Hecht, Ph.D., chief executive officer of Tisento.
  • “The first step in improving quality of life for this community is to better understand what individuals with MELAS face on a daily basis.
  • Tisento plans to use the findings from the interviews to identify clinical outcomes assessments that may be used as clinical study endpoints to evaluate the efficacy of zagociguat for the treatment of MELAS.

Mission Therapeutics to Present at Michael J. Fox Foundation's 15th Annual Parkinson's Disease Therapeutics Conference

Retrieved on: 
Tuesday, October 10, 2023

Fox Foundation's Parkinson's Disease Therapeutics Conference on October 19, 2023, in New York.

Key Points: 
  • Fox Foundation's Parkinson's Disease Therapeutics Conference on October 19, 2023, in New York.
  • Fox Foundation (MJFF) is dedicated to accelerating a cure for Parkinson's Disease and improved therapies for those living with the condition today.
  • A major unmet need for Parkinson's Disease patients is a treatment that slows or prevents the progression of disease.
  • Mission Therapeutics is currently developing two DUB inhibitors: MTX325 (targeting the CNS) and MTX652 (peripheral) which can potentially be used to treat any disease driven by mitochondrial dysfunction.

Mission Therapeutics to Present at Michael J. Fox Foundation's 15th Annual Parkinson's Disease Therapeutics Conference

Retrieved on: 
Tuesday, October 10, 2023

Fox Foundation's Parkinson's Disease Therapeutics Conference on October 19, 2023, in New York.

Key Points: 
  • Fox Foundation's Parkinson's Disease Therapeutics Conference on October 19, 2023, in New York.
  • Fox Foundation (MJFF) is dedicated to accelerating a cure for Parkinson's Disease and improved therapies for those living with the condition today.
  • A major unmet need for Parkinson's Disease patients is a treatment that slows or prevents the progression of disease.
  • Mission Therapeutics is currently developing two DUB inhibitors: MTX325 (targeting the CNS) and MTX652 (peripheral) which can potentially be used to treat any disease driven by mitochondrial dysfunction.