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Oak Hill Bio Squares Off Against Big Rivals to Find Angelman Treatment
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By Brian Gormley, WSJ Pro
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Good day. Startup Oak Hill Bio trails larger rivals in the race to find a treatment for the rare genetic disease Angelman syndrome—but could still emerge a winner.
Armed with a freshly raised $32.5 million Series A, Oak Hill is competing with Ultragenyx Pharmaceutical and Ionis Pharmaceuticals in the search for a treatment for Angelman, which causes severe developmental delays and speech impairment, among other symptoms.
It’s hardly the norm for a startup to go head-to-head against bigger drugmakers. But Oak Hill believes the potency of its drug, licensed from life sciences company Roche, could overshadow other contenders, said Chief Executive Josh Distler.
“Best-in-class potency we hope will translate into best-in-class efficacy,” he said.
Current treatments manage symptoms, but these companies are advancing what could be the first drugs to arrest the underlying disease, which affects 500,000 people worldwide, according to the Angelman Syndrome Foundation.
Angelman is caused by abnormalities in the UBE3A gene. In a healthy brain, the UBE3A gene inherited from the mother is active and the paternal copy is suppressed. In Angelman syndrome, however, the maternal UBE3A gene is mutated or missing, leaving patients bereft of a protein essential to brain development and functioning.
Ultragenyx, Ionis and Oak Hill are developing different molecules, but the way they work is the same. Each is designed to un-suppress the paternal UBE3A gene, enabling it to express the missing protein.
Ultragenyx’s and Ionis’s drugs are in late-stage, Phase 3 clinical trials. Oak Hill will use its recent financing round—from Balyasny Asset Management, venBio, Janus Henderson Investors and KCap Biotechnology Fund—to launch Phase 3 trials this year.
The market is big enough for multiple companies as the unmet need is urgent.
“The appetite for therapies that move the needle for these patients is extremely high,” said Luca Issi, a senior biotech analyst with investment bank RBC Capital Markets.
Oak Hill’s treatment, which could lead to improvements in areas such as communication and focus, would need to be taken on an ongoing basis, Distler said. “We hope over time the benefits accumulate,” he added.
And now on to the news...
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Eli Lilly and Ascidian Therapeutics to research and develop kidney-disease treatments. MIKE SEGAR/REUTERS
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Lilly makes another biotech deal. Eli Lilly signed a collaboration and licensing agreement worth up to $1.9 billion with Ascidian Therapeutics to research and develop kidney-disease treatments.
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Ascidian, a Boston-based biotechnology company, said Wednesday it granted Eli Lilly exclusive, target-specific rights to its RNA-exon-editing technology for undisclosed kidney-disease targets.
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The RNA-exon editors are capable of altering parts of genetic code to repair genetic instructions that cause disease.
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Ascidian said it will lead discovery and certain preclinical activities, while Eli Lilly will be responsible for other preclinical work, clinical development, manufacturing and commercialization.
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$1.9 Billion
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The potential value of an Eli Lilly kidney-disease collaboration and licensing agreement with Ascidian Therapeutics.
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Radiation Device Placed in Brain Cuts Tumor Recurrence
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Brain tumors are one of the most devastating consequences of cancer’s spread—hard to treat and highly deadly. Scientists have found that using a radioactive implant precisely where a tumor was removed in the brain can help patients get their cancer treated more quickly and in many cases, live longer. A new study showed that GammaTile, a radioactive wafer the size of a postage stamp, nearly doubled survival rates and nearly eliminated tumor regrowth in people who had it placed in the spot where brain tumors were surgically removed. GT Medical Technologies, an Arizona-based company, makes the tiles.
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Top AI CEOs Call for Law Protecting Against Biological Weapons
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Top artificial-intelligence executives are joining security experts in calling for Congress to protect against biological threats posed by AI, adding to growing pressure on lawmakers to address the technology’s risks. Three major chief executive officers—OpenAI’s Sam Altman, Anthropic’s Dario Amodei and Demis Hassabis of Google’s DeepMind AI lab—are among the signatories of a letter urging Congress to require safeguards when companies order synthetic DNA and RNA, a key step in developing certain vaccines and biotech breakthroughs.
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Benchmark Breaks From Past, Embraces Mature Startups
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Benchmark, one of Silicon Valley’s older venture-capital firms known best for early-stage bets on Uber and Twitter, has raised two new funds and plans to expand into investments in more mature startups, according to people familiar with the matter. The move would break with a strategy that Benchmark has long defended. While much of Silicon Valley has raised bigger and bigger funds, Benchmark has kept its flagship vehicles at roughly $425 million since 2004.
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Benchmark’s new funds include a $750 million fund dedicated to early-stage investments and a $1.25 billion fund for late-stage bets, the firm’s first-ever growth fund. A representative for Benchmark didn’t comment.
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People
Corrixr Therapeutics, a genetic medicine startup targeting lung, head and neck, and esophageal squamous cell carcinomas, appointed Hilary M. Malone as chief executive officer. She succeeds Eric B. Kmiec, who will continue to serve as chief scientific officer. Malone most recently served as CEO of Stylus Medicine.
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Contraline, a Charlottesville, Va.-based startup developing novel male contraceptives, closed a $92.5 million Series B round. BVF Partners and RA Capital Management co-led the investment, which saw participation from GV, Lumira Ventures and Invus.
Adaptive Innovations, an AI-native home healthcare agency based in New York and Dallas, raised $50 million in Series A funding. Felicis led the round, which included additional support from Bain Capital Ventures, Optum Ventures, Sunflower Capital and BoxGroup.
Apoha, a London-based startup building a foundational data layer revealing how molecules and materials behave under real-world conditions, emerged from stealth with $36 million in funding. Singular led the round, which included participation from Draper Associates, Redalpine and Seedcamp.
Subtle Medical, a Menlo Park, Calif.-based medical imaging software provider, scored $33 million in growth capital led by funds managed by Morgan Stanley Expansion Capital. The company also appointed Ohad Arazi as chief executive officer.
Advanced NanoTherapies, a startup focused on improving outcomes for patients with coronary and peripheral artery disease, raised more than $31 million in Series B funding from investors including S3 Ventures and T45 Fund.
Novellia, a New York-based provider of data built on information patients volunteer to share with pharmaceutical companies, snagged $18 million in Series A funding. Spark Capital led the round, which included participation from Khosla Ventures, Acrew Capital, Bling Capital and TMV.
Ingenix, a Warsaw-based drug development startup, added €13 million (about $15 million) in seed extension funding led by Sofinnova Partners.
Ilant Health, a New York-based obesity treatment startup, secured $15 million in Series A funding led by Cornucopian Capital.
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The FDA had twice rejected Replimune’s experimental skin-cancer treatment. ANDREW KELLY/REUTERS
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