Arkuda Therapeutics, a company that leverages new insights into progranulin and lysosomal biology to develop medicines to change the trajectory of neurodegenerative diseases, today announced the completion of the $44 million Series A funding Led by a team of drug developers and scientists with deep experience in central nervous system disorders and neurodegeneration, Arkuda’s lead therapeutic approach focuses on improving lysosomal function and neuronal health by increasing the levels of progranulin and its functional subunits, granulins, in the central nervous system. Atlas Venture co-founded, seeded and incubated the company and co-led Series A financing with Pfizer Ventures, with the participation of funds managed by Tekla Capital Management LLC and BioInnovation Capital
Arkuda’s initial therapeutic focus is on a devastating genetically defined disease called GRN-related frontotemporal dementia (FTD-GRN), which affects people between the ages of 45 and 65. Individuals with FTD-GRN have an autosomal dominant mutation in one of the alleles of the GRN gene that codes for progranulin, a protein known to play an important role in lysosomal function and innate immunity in the brain. FTD-GRN is characterized by a progressive and rapid loss of nerve cells in the brain’s frontal and temporal lobes, affecting personality, behavior, language, memory and movement, with a typical life expectancy of 6 to 7 years after symptoms appear.
“We believe that the challenges in the treatment of neurodegeneration stem from the targeting of disease pathologies rather than root causes such as lysosomal dysfunction, as well as the treatment of patients at too late a time when significant damage has already occurred,” said Gerhard Koenig, PhD, co-founder, President and CEO of Arkuda Therapeutics. “FTD-GRN offers a unique and exciting opportunity to address the key driver of neurodegeneration before it progresses too far. “That’s right.
It is now understood that the deficiency of progranulin and subsequently granulins in GRN mutation carriers causes lysosomal dysfunction, which leads to neurodegeneration. Arkuda is taking advantage of new and evolving insights into the role of progranulins and granulins in lysosomal health to drive the discovery of small molecules to restore and maintain neuronal health. Using existing biomarkers to reliably identify GRN mutation carriers and those at risk of symptomatic conversion, the company’s goal is to respond to the disease as soon as possible.
“For years, companies have focused their efforts on targeting neurodegenerative diseases such as amyloid beta and tau with little success,” said Bruce Booth, DPhil, Arkuda Chairman and Partner at Atlas Venture. “There is growing scientific evidence showing the critical importance of lysosomes in the maintenance of neuronal health in FTD and other diseases such as Alzheimer’s and Parkinson’s disease. Arkuda’s unique approach to targeting progranulin, a critical driver of lysosomal function, has the potential to have a truly meaningful impact on patients and their families. “That’s right.
Arkuda Therapeutics was founded by Atlas Venture, Gerhard Koenig and Duane Burnett, PhD, Senior Vice President, Discovery, in 2018.
Arkuda Therapeutics
Arkuda Therapeutics is a biotechnology company that leverages new insights into progranulin and lysosomal biology to develop medicines to change the pathway of neurodegenerative disease. The aim of Arkuda’s lead compounds is to correct progranulin deficiency and lysosomal dysfunction in GRN-related frontotemporal dementia (FTD-GRN), a genetically defined subtype of frontotemporal dementia caused by autosomal dominant mutation in the GRN gene. Arkuda launched in November 2019 with $44 million from leading life science investors including Atlas Venture, Pfizer Ventures, funds managed by Tekla Capital Management LLC, and BioInnovation Capital Visit www to learn more The akudatx Come on,
On the Atlas Venture
Atlas Venture is a leading biotech venture capital company With the aim of doing well by doing good, the company has been building breakthrough biotech startups since 1993. Atlas works alongside outstanding scientists and entrepreneurs to translate high impact science into medicines for patients. Our Seed-Leading Venture Creation Strategy rigorously selects and focuses investment on the most compelling opportunities to build scalable businesses and realize value. Please visit www for more information. Atlasventure’s Come on,
About Pfizer Venture
Pfizer Ventures (PV) is the venture capital arm of Pfizer Inc. Founded in 2004, it invests in return in areas of current or future strategic interest to Pfizer. PV seeks to remain at the forefront of life science advancements, to identify and invest in emerging companies developing transformative medicines and technologies that have the potential to enhance the Pfizer pipeline and shape the future of our industry. Visit www for more information. Pfizerventures: Come on,
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