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HEALTHCARE | Biotechnology
biosplice.com

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Founded Year

2008

Stage

Series B | Alive

Total Raised

$561.5M

Last Raised

$120M | 6 mos ago

About Biosplice Therapeutics

Biosplice Therapeutics is a clinical-stage biotechnology company pioneering therapeutics based on alternative pre-mRNA splicing for major diseases. Its scientific platform is based on biological discoveries that govern tissue specialization and enable it to selectively eliminate harmful proteins using small molecules. From the company's foundational discoveries in Wnt pathway modulation to unlocking the broad therapeutic potential of the CLK/DYRK target class, its focus is on the realization of new, potentially curative therapies for patients by selectively reprogramming cellular behavior.

Biosplice Therapeutics Headquarter Location

9360 Towne Centre Drive Suite 160

San Diego, California, 92121,

United States

858-926-2900

Latest Biosplice Therapeutics News

Researchers unravel the genetic cause of a childhood disorder and a potential way to prevent it with drugs

Sep 30, 2021

Sep 30, 2021 8:45am Scientists led by the University of California, San Diego reported positive results in mouse models for a drug to treat a newly defined genetic condition that impacts prenatal organ development. (Pixelistanbul/Getty) An international research team led by the University of California, San Diego (UCSD) has identified a genetic cause for a syndrome that has stumped clinicians for many years. And the scientists say they might be able to prevent the disease-causing mutation during pregnancy based on promising mouse studies. The researchers identified the condition, dubbed Zaki syndrome, after doctors from around the world compared clinical notes that showed children born with it had DNA mutations in the Wnt-less, or WLS, gene. By boosting Wnt signaling with a drug in mouse models, they were able to reverse developmental disabilities caused by the disease, they reported in The New England Journal of Medicine. Zaki is a rare condition that hampers prenatal development of the eyes, brain, hands, kidneys and heart and that causes lifelong disabilities. The syndrome is named after Maha Zaki, M.D., Ph.D., at the National Research Center in Cairo, who was the first to spot it. The UCSD team, working with Rady Children’s Institute for Genomic Medicine and researchers around the world, started by scouring databases of 20,248 families who had children with neurodevelopmental disorders. They zeroed in on mutations in the WLS gene, which controls signaling levels for Wnt, a hormonelike protein that's involved in embryonic development. The scientists then created stem cells and mouse models of Zaki syndrome and used them to test a drug that boosts Wnt signaling. The drug, CHIR99021, amped up Wnt signaling and restored development in the models. The mouse embryos grew body parts that had failed to develop, and their organs resumed normal growth, the scientists said. The Wnt signaling pathway, discovered in the 1980s, has caught investor interest and is core to the missions of biotechs Surrozen  and Biosplice Therapeutics, formerly known as Samumed . Biosplice's Wnt-modulating kinase inhibitor is in multiple phase 3 trials and has garnered licensing agreements in China and Korea in recent months. Meanwhile, Surrozen is targeting Wnt in designing regenerative medicines for diseases in the eye, lung, kidney and a host of other areas. Phase 1 studies are slated  for next year, and the biotech hit the Nasdaq last month. The UCSD-led team was surprised to discover that Zaki syndrome could could be prevented in preclinical models with a drug, said author Guoliang Chai, Ph.D., a former postdoctoral fellow at UCSD's School of Medicine, in a statement . Now the researchers are thinking about how to transform Zaki into an entirely preventable condition. “We can see this drug, or drugs like it, eventually being used to prevent birth defects, if the babies can be diagnosed early enough.”  Read more on

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Research containing Biosplice Therapeutics

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CB Insights Intelligence Analysts have mentioned Biosplice Therapeutics in 1 CB Insights research brief, most recently on Sep 30, 2021.

Expert Collections containing Biosplice Therapeutics

Expert Collections are analyst-curated lists that highlight the companies you need to know in the most important technology spaces.

Biosplice Therapeutics is included in 6 Expert Collections, including Unicorns- Billion Dollar Startups.

U

Unicorns- Billion Dollar Startups

863 items

P

Pharma Startups

6,549 items

Pharmaceutical companies working across drug discovery, drug development and drug manufacturing.

C

Cancer Therapeutics

1,179 items

Pharmaceutical and biotechnology companies with cancer therapy drug candidates.

R

Rare Diseases

229 items

R

Regenerative Medicine

1,767 items

Regenerative medicine refers to the process of activating, replacing, engineering or regenerating human genetic material, cells, tissues or organs to restore normal function. It also includes bioengineered tissues used for in vitro testing (e.g. organ-on-a-chip, organoids).

B

Biopharmaceuticals

14,138 items

Companies involved in the research, development, and commercialization of chemically- or biologically-derived therapeutic & theranostic drugs. Excludes vitamins/supplements, CROs/clinical trial services.

Biosplice Therapeutics Patents

Biosplice Therapeutics has filed 114 patents.

The 3 most popular patent topics include:

  • Transcription factors
  • G protein coupled receptors
  • Clusters of differentiation
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8/3/2021

Transcription factors, Senescence, Rare diseases, Skin care, Cell biology

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Transcription factors, Senescence, Rare diseases, Skin care, Cell biology

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