Posts by: Patricia Inacio, PhD

News

GS010, a gene therapy designed to improve vision clarity in people with Leber hereditary optic neuropathy (LHON), continues to show benefits in the second scheduled readout — at week 72 — ... Read more

March 29, 2019March 29, 2019

New Phase 1 Trial of Potential Treatment for Mitochondrial Diseases, KL1333, Enrolling in UK

News

A second early clinical trial evaluating KL1333, an investigational treatment for genetic mitochondrial diseases, has enrolled its first participant, NeuroVive announced. The Phase 1a/b trial (NCT03888716) of this small molecule treatment ... Read more

October 19, 2018October 19, 2018

NV354, Potential Therapy for Mitochondrial Diseases, Shows Promise in Preclinical Studies

News

NeuroVive Pharmaceutical’s therapeutic candidate NV354 for mitochondrial diseases, such as Leigh syndrome, was able to restore the body’s own energy substrate — succinate — in an in vivo model of ... Read more

August 24, 2018August 24, 2018

Short-Term Treatment With KH176 Lessens Depression, Improves Mood in Mitochondria Patients, Study Shows

News

Short-term treatment with oral KH176 was safe and well-tolerated by mitochondria disease patients, and lessened their symptoms of depression while also having a positive effect in alertness and mood, ... Read more

July 20, 2018July 20, 2018

HotSpot Therapeutics Secures $45M to Advance Drug Discovery Platform

News

HotSpot Therapeutics has secured $45 million to advance its drug discovery strategy that identifies so-called “hot spots” in proteins, allowing for their precise targeted regulation. Using this technology, HotSpot Therapeutics ... Read more

June 15, 2018June 15, 2018

Mutations in Mitochondrial Gene ANT1 Linked to Risk of Bipolar Disease in Mouse Study

News

Mutations in the mitochondrial gene ANT1 may increase the risk of bipolar disease by altering levels of serotonin transmission in the brain, a mouse study suggests. The study, “Ant1 mutant mice ... Read more

March 26, 2018March 26, 2018

Gene Mutation Leads to Early Childhood Neurodegenerative Disorder, Researchers Find

News

Researchers found that inherited mutations in the PMPCB gene lead to a wide range of impairments in mitochondria and cause a form of early childhood neurodegenerative disorder in members of ... Read more

March 19, 2018March 19, 2018

New Cellular Pathway Helps Protect Mitochondria During Stress, Study Shows

News

A specific branch of cells’ stress-response pathway called unfolded protein response (UPR) may help protect mitochondria, new research shows. This finding suggests that controlling this particular branch may help ... Read more

March 2, 2018March 5, 2018

New Method Improves Delivery of Healthy Mitochondria To Cells Where It Is Dysfunctional, Study Shows

News

South Korean researchers have developed a simple way to deliver healthy mitochondria to cells where it is dysfunctional, rescuing the cells’ energy levels and metabolic function. The study, “Delivery ... Read more

February 19, 2018

Manganese Deficiency Underlies Mitochondria Defects in Leigh Syndrome, Other Diseases, Study Finds

News

Mutations in the SLC39A8 gene contribute to mitochondria impairments seen in Leigh syndrome by reducing the levels of manganese, a key element that protects against oxidative stress, researchers found. ... Read more