Mitochondria

Human cells
#Junevity #Mitochondria #University of California San Francisco

Cellular ageing could be reversed, research reveals 

Cellular ageing could be reprogrammed back towards health, new research finds.  A study published in Proceedings of the National Academy of Sciences (PNAS) shows precise modulation of individual transcription factors can reverse core hallmarks of cellular ageing and restore tissue function in vivo.  The analysis was led by biotechnology company Junevity,… Read More

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#Biopharma #Business #Duchenne muscular dystrophy #Immunology / Virology & Vaccines #Mitochondria #Obesity

DDW Highlights: 11 November 2025

The latest episode of the DDW Highlights Podcast is now available to listen to below. DDW’s Bruno Quinney narrates five key stories of the previous week to keep DDW subscribers up-to-date on the latest industry news. Last week’s leading story came from a positive development in biopharma, with top companies… Read More

#Podcasts

#Biopharma #Business #Duchenne muscular dystrophy #Immunology / Virology & Vaccines #Mitochondria #Obesity

This week in Drug Discovery (3 – 7 November) 

News round-up for 3 – 7 November by Bruno Quinney, Content Team at DDW.    This week’s leading story is a positive development in biopharma, with top companies reporting market growth in Q3 of 2025. Elsewhere, new research could help combat yellow fever and weight loss, while £500,000 was awarded to a… Read More

#News

Mitochondria
#FDA #Mitochondria

FDA approval of first TK2d drug offers ‘hope for the future’ 

The US Food and Drug Administration (FDA) has approved the first treatment for thymidine kinase 2 deficiency (TK2d), paving the way for landmark treatment options for the mitochondrial disease.  Doxecitine and doxribtimine, approved by the FDA, are oral solutions developed by biopharmaceutical company UCB. The powder, also known as Kygevvi, has been approved… Read More

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#DNA #Drug therapy #Mitochondria

Drug therapies could prevent hereditary diseases, study finds

New drug therapies could be developed to prevent hereditary diseases, research reveals.  According to a study published in the latest issue of Science, researchers have pinpointed a cellular mechanism involved in the inheritance of genetic mutations. It is believed the findings could help develop treatments… Read More

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FDA
#FDA #Mitochondria #PTC Therapeutics #Rare disease

FDA rejects PTC Therapeutics’ drug for Friedreich’s ataxia

The US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) stating its decision not to recommend PTC Therapeutics’ vatiquinone for Friedreich’s ataxia (FA).  The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional… Read More

#News #Regulatory

#Gene mutation #Genetic diseases #MHRA #Mitochondria #Rare disease #UCB

TK2d drug gets UK Promising Innovative Medicine designation

Dox – comprising of doxecitine and doxribtimine – to treat thymidine kinase 2 deficiency (TK2d) has been granted Promising Innovative Medicine (PIM) designation in the UK by the Medicines and Healthcare Products Regulatory Agency (MHRA).  TK2d is caused by a genetic mutation in the TK2 gene. This… Read More

#News #Regulatory

Mitochondria
#Mitochondria #Neuroscience #NRG Therapeutics #Parkinson's #The Michael J Fox Foundation for Parkinson’s Research (MJFF)

Novel Parkinson’s therapy protects mitochondrial function

NRG Therapeutics has been awarded a $5 million grant from The Michael J Fox Foundation for Parkinson’s Research (MJFF) to support the development of NRG5051 as a novel disease-modifying treatment for Parkinson’s disease. The company says NRG5051 also has potential in other neurological diseases such as… Read More

#Business & Investment #Editor's Choice #News

Woman standing on scales
#Jagiellonian University Medical College #Mitochondria #MitoRX Therapeutics #Obesity #University of Exeter #Weight loss

New drug for obesity reduced weight gain by 32%

Therapies that deliver hydrogen sulphide to cells could one day become the basis of new treatments for obesity and related diseases, new research has concluded. Mounting evidence suggests that hydrogen sulphide plays an important role in the liver. Previous research shows that tiny amounts of hydrogen… Read More

#News

Disabled patient having rehabilitation
#Fujita Health University #Japan #Mitochondria #Muscle atrophy #Muscles

Discovery suggests new treatment for muscle atrophy

Researchers in Japan have revealed the key underlying mechanisms in muscle atrophy, paving the way towards potential new treatments. While the disuse of muscles is the most frequent catalyst for muscle atrophy, there are several other possible causes, including chronic diseases, injury and exposure to low-gravity… Read More

#News

Human heart
#Brazil #Foresee Pharmaceuticals #Heart failure #Mitochondria #University of São Paulo

Research proves synthetic molecule can mitigate heart failure

Researchers at the University of São Paulo (USP) in Brazil and Foresee Pharmaceuticals have tested a synthetic molecule which could increase heart function in patients with heart failure. Heart failure is a condition in which the heart cannot pump enough blood to meet the body’s needs… Read More

#News

#Drug development #Drug discovery #Drug safety #Drug screening #Mitochondria #Toxicology

App Note: Measure mitochondrial toxicity using primary hepatocytes

Mitochondrial dysfunction is known to play a central role in drug-induced hepatotoxicity, and thus early detection of mitochondrial toxicity is a critical component of initial drug development efforts. While immortalised cell lines are widely used for in vitro assessment of hepatotoxicity, cryopreserved primary human hepatocytes are a preferred model for drug… Read More

#News

#Drug development #Drug discovery #Drug safety #Drug screening #Mitochondria #Toxicology

Measurement of mitochondrial toxicity using primary hepatocytes

Mitochondrial dysfunction is known to play a central role in drug-induced hepatotoxicity, and thus early detection of mitochondrial toxicity is a critical component of initial drug development efforts. While immortalised cell lines are widely used for in vitro assessment of hepatotoxicity, cryopreserved primary human hepatocytes are a preferred model for drug… Read More

#Resources

#Agilent #Biosafety testing #Drug discovery #In vivo #Infographic #Mitochondria

Learn new ways to identify mitochondrial liability

Download this new mitochondrial toxicity infographic by Agilent and discover the benefits of novel in vitro strategies for consumer safety. Drug-induced mitochondrial dysfunction has been linked to compound attrition, organ toxicity, black-box warnings, and market withdrawals. The infographic will help you: Gain an historical perspective on mitochondrial… Read More

#News

#Agilent #Biosafety testing #Drug discovery #In vivo #Infographic #Mitochondria

Learn new ways to identify mitochondrial liability

Download our mitochondrial toxicity infographic and discover the benefits of novel in vitro strategies for consumer safety. Drug-induced mitochondrial dysfunction has been linked to compound attrition, organ toxicity, black-box warnings, and market withdrawals. Our infographic will help you: Gain an historical perspective on mitochondrial toxicity. Understand the importance of testing… Read More

#Intelligence #Resources

#Agilent #Mitochondria #Neurodegenerative disorders #Neurology #Rare disease #Webinar

Watch now: Neuronal metabolism and early drug discovery

A new webinar is now available to watch anytime. CLN7 neuronal ceroid lipofuscinosis is an inherited lysosomal storage neurodegenerative disease highly prevalent in children. In her webinar, Dr García Macía discusses how failure in autophagy causes the accumulation of structurally and bioenergetically impaired neuronal mitochondria in the… Read More

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