Uncategorized

Stem Cell Editing Programs the Immune System to Make Own Therapeutic Proteins

Published

2 months ago

April 17, 2026

For pathogens like HIV, malaria, and rapidly evolving influenza strains, coaxing the immune system to produce the rare, highly potent antibodies needed for protection has long been a scientific bottleneck. Vaccines can train B cells to evolve such broadly neutralizing antibodies, but only under ideal conditions—and only in a small fraction of people. Even attempts to genetically edit mature B cells produced responses that faded as the cells died out.

A team at the Rockefeller University has now taken a more upstream approach: programming hematopoietic stem and progenitor cells (HSPCs)—the source of all B lymphocytes—to carry permanent genetic instructions for therapeutic antibodies or other proteins. Because the immune system naturally amplifies rare, useful cells after vaccination, even a tiny number of edited stem cells can seed a durable, boostable immune response.

“The immune system is inefficient in that it produces a vast quantity of cells to protect itself,” said Harald Hartweger, a research assistant professor in Michel Nussenzweig’s Laboratory of Molecular Immunology. “We wanted to take advantage of the immune system’s ability to amplify useful, rare cells.”

The study, published in Science and titled “B lymphocyte protein factories produced by hematopoietic stem cell gene editing,” demonstrates that CRISPR‑edited HSPCs can mature into B cells that express engineered antibodies upon vaccination. A standard vaccination then acts as the trigger: antigen exposure drives those edited B cells to expand, differentiate into plasma cells, and secrete high titers of the inserted antibody that last long-term.

According to the paper, as few as ~7,000 edited HSPCs were enough to generate “high titers of long‑lasting protective or therapeutic antibodies and/or cargo proteins.” In mice engineered to produce a broadly neutralizing influenza antibody, this response was strong enough to protect against an otherwise lethal viral infection.

The platform proved unexpectedly versatile. Edited B cells could also secrete non‑antibody proteins, pointing to potential applications in genetic diseases. And by mixing HSPCs engineered with different antibody instructions, the researchers created immune systems capable of producing multiple antibodies simultaneously, an approach that could limit viral escape in HIV or other rapidly mutating pathogens. Human HSPCs edited using the same strategy produced functional human B cells in an immunodeficient mouse model, offering an early sign of translational feasibility.

“Our goal is to permanently impact the genome with a single injection, so that the body can make proteins of interest,” Hartweger said. “That protein could be an antibody that’s universally protective against HIV or influenza, but it could also be any therapeutic protein.”

The team is now moving toward preclinical testing in non‑human primates to evaluate protection against HIV and exploring whether similar strategies could be applied to T cells. The broader vision is a generalizable, long‑term protein‑production platform, one that could support treatments for infectious disease, protein deficiencies, autoimmunity, metabolic disorders, and cancer, according to Hartweger.

As Nussenzweig puts it, “The present study proposes a workaround for the antibody problem—a way of getting around the possibility that we may never get to a universal HIV vaccine, while still providing a promising, long‑lasting solution.”

The post Stem Cell Editing Programs the Immune System to Make Own Therapeutic Proteins appeared first on GEN – Genetic Engineering and Biotechnology News.

Uncategorized

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

Published

51 minutes ago

June 12, 2026

Temp User

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.

Uncategorized

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…

Published

4 hours ago

June 12, 2026

Temp User