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STAT+: Cell therapy primed liver transplant patients to avoid organ rejection, small study shows
Immune tolerance has long been the holy grail in transplant medicine, a hoped-for end to the downsides of anti-rejection regimens for patients after they receive lifesaving organ transplants. A small, early-stage study now shows promise in taking cells from living donors — people giving a portion of their livers — to teach recipients’ immune systems to accept the foreign organs as their own and achieve the ultimate healthy outcome.
Living donations take advantage of the liver’s ability to regenerate, meaning donors can part with a piece of their liver and later see it grow back. Recipients can regain enough liver function from the partial organs that also grow, replacing livers damaged by alcohol-associated liver disease, metabolic-associated liver disease, liver cancer, or other causes. Immunosuppression keeps their bodies from rejecting the new organs, but it also raises their vulnerability to infectious diseases and certain cancers. Serious side effects from the drugs include developing diabetes and kidney damage.
Cell therapy has been tried before to disarm the immune system’s attack by recruiting regulatory T immune cells taken from the donor. In the new study, whose results were published Friday in Nature Communications, different immune cells known as regulatory dendritic cells were obtained from donors’ white blood cells and generated in a lab. The idea behind both cell therapies is the same: to teach immune cells in the recipient’s body to treat the donated liver fragment as familiar tissue, not an invader be attacked.
Immune tolerance has long been the holy grail in transplant medicine, a hoped-for end to the downsides of anti-rejection regimens for patients after they receive lifesaving organ transplants. A small, early-stage study now shows promise in taking cells from living donors — people giving a portion of their livers — to teach recipients’ immune systems to accept the foreign organs as their own and achieve the ultimate healthy outcome.
Living donations take advantage of the liver’s ability to regenerate, meaning donors can part with a piece of their liver and later see it grow back. Recipients can regain enough liver function from the partial organs that also grow, replacing livers damaged by alcohol-associated liver disease, metabolic-associated liver disease, liver cancer, or other causes. Immunosuppression keeps their bodies from rejecting the new organs, but it also raises their vulnerability to infectious diseases and certain cancers. Serious side effects from the drugs include developing diabetes and kidney damage.
Cell therapy has been tried before to disarm the immune system’s attack by recruiting regulatory T immune cells taken from the donor. In the new study, whose results were published Friday in Nature Communications, different immune cells known as regulatory dendritic cells were obtained from donors’ white blood cells and generated in a lab. The idea behind both cell therapies is the same: to teach immune cells in the recipient’s body to treat the donated liver fragment as familiar tissue, not an invader be attacked.
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.
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.
Officials sanctioned Dabur India months after FDA inspectors found bird droppings and data integrity deficiencies during an inspection of the plant.
Illinois’ financial crisis could bring the state to a halt
The final 6 ‘Game of Thrones’ episodes might feel like a full season
New Season 8 Walking Dead trailer flashes forward in time
Mod turns ‘Counter-Strike’ into a ‘Tekken’ clone with fighting chickens
Meet Superman’s grandfather in new trailer for Krypton
Disney’s live-action Aladdin finally finds its stars
SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines
STAT+: Up and down the ladder: The latest comings and goings
FDA imposes import alert on Indian plant after inspectors flag GMP failings
STAT+: Pharmalittle: We’re reading about a discontinued cancer drug, a Novo security breach, and more
Amgen shores up Tavneos’ FDA defense with Duke data analysis
Chile offers new data on food warning label efficacy
Illinois’ financial crisis could bring the state to a halt
The final 6 ‘Game of Thrones’ episodes might feel like a full season
New Season 8 Walking Dead trailer flashes forward in time
Mod turns ‘Counter-Strike’ into a ‘Tekken’ clone with fighting chickens
Meet Superman’s grandfather in new trailer for Krypton
Disney’s live-action Aladdin finally finds its stars
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