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This week in Drug Discovery (13 – 17 April) 

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News round-up for 13 – 17 April by Bruno Quinney, Content Team at DDW. 

This week, breakthroughs have emerged in cancer research. Elsewhere, regulatory reforms have accelerated clinical trial set up in the UK. 

The top stories: 

 

Novel therapy could slow tumour growth

A novel cancer therapy targeting tumour acidity could boost immune response and slow cancer growth, studies show.  

Read more… 

B7-H4-targeted ADC shows 62% response rate in ovarian cancer

The global Phase I BEHOLD-1 clinical trial for mocertatug rezetecan (Mo-Rez) has shown confirmed objective response rates (cORR) of 62% in platinum-resistant ovarian cancer (PROC) and 67% in recurrent or advanced endometrial cancer (EC).  

Read more… 

 

Immunopeptide could delay inflammatory arthritis, study says 

Scientists investigating the naturally occurring immunopeptide PEPITEM (Peptide Inhibitor of Trans-Endothelial Migration) have shown reductions in arthritic joint swelling to a degree comparable to the current standard of care, reducing inflammatory changes that cause joint damage.  

Read more… 

 

 

Speech overlooked as clinical marker, biotech says

SynaptixBio, which became the only company to commercialise a treatment for the rare disease H-ABC, is set to launch a study into speech as a non-invasive, digital clinical marker in neurodegenerative disease research.  

Read more… 

 

 

Regulatory reforms accelerate UK clinical trial set up
 

NHS patients are accessing groundbreaking treatments faster as the UK government fulfils its promise for a 150-day clinical trial target. 

Read more… 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

The post This week in Drug Discovery (13 – 17 April)  appeared first on Drug Discovery World (DDW).

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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

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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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