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STAT+: Finally cracking KRAS as a druggable target

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Hiya. Today, we see a proliferation of KRAS, as new data validate a once-undruggable space. Also, a double-edged push for PBM transparency, and the Breakthrough Prize was awarded to researchers for advances in gene therapy and ALS genetics.

The need-to-know this morning

  • Novo Nordisk said its experimental oral drug, called etavopivat, reduced the risk of pain crises and improved hemoglobin response in patients with sickle cell disease, achieving the goals of a Phase 3 clinical trial. 
  • Eli Lilly is reportedly nearing a deal to acquire Kelonia Therapeutics for more than $2 billion, according to the Wall Street Journal. Kelonia is a privately held developer of so-called in-vivo CAR-T therapies that make engineered cancer-killing cells inside the body. Its lead therapy is being investigated in an early stage study in multiple myeloma.
  • Helus Pharma, developer of psychedelics-based therapies for depression, said CEO Michael Cola has resigned “at the request of the board of directors.” Chairman Eric So was appointed interim CEO.

Finally cracking KRAS as a druggable target

When Revolution Medicines released results for its drug daraxonrasib in pancreatic cancer patients last week, it finally seemed like there was hope in a condition that has long proven intractable for researchers. But as STAT’s Angus Chen reports, it was the product of years of effort — and could be just the start of a wave of new RAS inhibitors. 

Continue to STAT+ to read the full story…

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Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

Hiya. Today, we see a proliferation of KRAS, as new data validate a once-undruggable space. Also, a double-edged push for PBM transparency, and the Breakthrough Prize was awarded to researchers for advances in gene therapy and ALS genetics.

The need-to-know this morning

  • Novo Nordisk said its experimental oral drug, called etavopivat, reduced the risk of pain crises and improved hemoglobin response in patients with sickle cell disease, achieving the goals of a Phase 3 clinical trial. 
  • Eli Lilly is reportedly nearing a deal to acquire Kelonia Therapeutics for more than $2 billion, according to the Wall Street Journal. Kelonia is a privately held developer of so-called in-vivo CAR-T therapies that make engineered cancer-killing cells inside the body. Its lead therapy is being investigated in an early stage study in multiple myeloma.
  • Helus Pharma, developer of psychedelics-based therapies for depression, said CEO Michael Cola has resigned “at the request of the board of directors.” Chairman Eric So was appointed interim CEO.

Finally cracking KRAS as a druggable target

When Revolution Medicines released results for its drug daraxonrasib in pancreatic cancer patients last week, it finally seemed like there was hope in a condition that has long proven intractable for researchers. But as STAT’s Angus Chen reports, it was the product of years of effort — and could be just the start of a wave of new RAS inhibitors. 

Continue to STAT+ to read the full story…

Read More

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SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines

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Biotechnology company SonoThera has raised $125 million in an oversubscribed Series B financing round. The financing was led by Vida Ventures, with participation from ARK Invest, CureDuchenne Ventures, Leaps by Bayer, Otsuka Pharmaceutical, SymBiosis, UCB Ventures SA, Vivo Capital, and existing investors ARCH Venture Partners, Alexandria Venture Investments, Duquesne Family Office, Illumina Ventures, Johnson & Johnson Innovation – JJDC, Medical Excellence Capital, RA Capital, and Vertex Ventures HC.

SonoThera will use the funds to advance its lead programs in Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) in the clinic. The funds will also support efforts to expand its pipeline of targeted redosable genetic medicines across multiple organ systems and scale its proprietary platform technologies for safe, targeted therapy delivery.

The company’s platform combines a proprietary ultrasound-mediated delivery technology dubbed RIPPLE™, with a payload engineering platform dubbed PORE™. The platforms are designed to support the development of DNA and RNA therapeutics, gene editing, and gene silencing approaches. SonoThera is using its tech to develop genetic medicines that it claims will address key limitations of conventional gene therapies including delivery challenges, payload size constraints, immune responses, safety events, and difficulties with redosing. 

As Kenneth Greenberd, PhD, SonoThera’s co-founder and CEO, stated “we founded SonoThera to take a fundamentally different approach, with a platform designed to broaden the therapeutic possibilities of the field. We believe our technology has the potential to expand the range of diseases addressable by genetic medicines while enabling more precise, durable, safer, and repeatable therapies for patients.”

SonoThera has already demonstrated the targeted delivery and expression capabilities of its platform across multiple tissues, including skeletal muscle, heart, liver, kidney, adipose, and brain. It has also shown that it can deliver large payloads such as full-length dystrophin for DMD and RNA-based payloads for gene silencing applications in preclinical studies. 

The company expects to initiate its first clinical trial in DMD in 2027.

Commenting on the financing, Rajul Jain, MD, managing director at Vida Ventures, said “we believe SonoThera, with its RIPPLE delivery and PORE payload engineering technologies, has the potential to unlock opportunities in diseases with significant unmet need that have been previously inaccessible to other genetic medicine approaches.” 

In connection with the financing, Jain and Rakhshita Dhar, MS, vice president & head of Healthcare Venture Investments at Leaps by Bayer, have joined SonoThera’s Board of Directors.

The post SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines appeared first on GEN – Genetic Engineering and Biotechnology News.

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STAT+: Up and down the ladder: The latest comings and goings

Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others. That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going.

And here is our regular feature in which we highlight a different person each week. This time around, we note that AstronauTx hired Michelle Mellion as chief medical officer. Previously, she held the same role at PepGen and EveryONE Medicines.

But all work and no play can make for a dull chief medical officer.

Continue to STAT+ to read the full story…

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Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others. That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going.

And here is our regular feature in which we highlight a different person each week. This time around, we note that AstronauTx hired Michelle Mellion as chief medical officer. Previously, she held the same role at PepGen and EveryONE Medicines.

But all work and no play can make for a dull chief medical officer.

Continue to STAT+ to read the full story…

Read More

Continue Reading

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FDA imposes import alert on Indian plant after inspectors flag GMP failings

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Officials sanctioned Dabur India months after FDA inspectors found bird droppings and data integrity deficiencies during an inspection of the plant.

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