All articles tagged with #biotechnology
Advocates for rare diseases express concern after the FDA rejected Regenxbio’s MPS type 2 gene therapy and signals a more cautious, data-heavy approval path that could delay access to promising treatments highlighted by newborn screening.
Researchers at the University of Waterloo are engineering Clostridium sporogenes bacteria to invade oxygen-poor tumor cores and consume nutrients from inside, potentially destroying tumors. They added an oxygen-tolerance gene and use quorum sensing to activate it only after enough bacteria accumulate, limiting safety risks. Next steps combine both features in a single strain and test in preclinical trials, showcasing interdisciplinary synthetic-biology cancer research.
Researchers studying a 5,000-year-old Psychrobacter strain from Romania’s Scărișoara Ice Cave found it resistant to multiple modern antibiotics yet capable of inhibiting several antibiotic‑resistant pathogens, suggesting ancient microbes could inspire new antibiotics but also carry a risk of spreading resistance genes if melted; calls for more research into cold-environment microbes and their biotechnological potential.
A Technion-led team, collaborating with MIT, Harvard, and others, reports a living, cell-based implant that functions as a pancreas by sensing glucose and producing insulin within the device, protected from immune attack by a crystalline shield; tested in animals, it aims to provide a self-regulating diabetes treatment and may be adaptable to other chronic diseases, but human trials have not yet begun.
Scientists have engineered a 'universal' kidney that can be accepted across blood types by using enzymes to strip away donor antigens and convert the kidney to a type O-like profile. The enzyme-converted kidney survived for days in a brain-dead recipient, signaling progress toward reducing transplant wait times, though long-term viability and immune responses remain challenges that need further work.
Researchers combine multiple donor liver cell types to form in vitro liver assembloids that recapitulate periportal tissue, moving toward miniature human livers for faster drug testing and personalized therapies.
ImmunityBio announced a productive Type B End‑of‑Phase meeting with the FDA regarding its supplemental Biologics License Application for ANKTIVA plus BCG in BCG‑unresponsive papillary NMIBC. The FDA asked for additional information (not new trials) to support a potential resubmission, which ImmunityBio will provide within 30 days. Long‑term QUILT‑3.032 data in 80 patients show about 96% bladder cancer‑specific survival at 36 months and high cystectomy‑free survival (roughly 82–93% at 12–36 months), underscoring a bladder‐sparing, chemo‑free approach. ANKTIVA is already approved for CIS in the US, UK, and Saudi Arabia, with EU conditional status for CIS and ongoing EMA discussions to extend labeling to papillary disease if US approval is achieved.
Live Science reports on SPARDA, a kamikaze-like bacterial defense system that sacrifices infected cells to prevent the spread of invaders. Scientists mapped SPARDA’s beta-relay switches in argonaute proteins with AlphaFold, revealing a self-destruct mechanism that activates in response to threats and can form DNA‑degrading chains. While SPARDA naturally guards bacteria, researchers see potential to repurpose it for diagnostics and other biotech tools, offering a CRISPR‑alternative route that could broaden target flexibility in detecting pathogens.
The piece highlights Biogen, Rivian, Bank of America, and Airbnb as among the biggest stocks moving at midday, with other names in focus across sectors.
Gene therapies are moving from trials toward patient care, with breakthroughs like personalized CRISPR treatments and new gene-editing tools powering hope for many diseases; however, turning lab successes into approved, accessible medicines is hampered by regulatory, manufacturing, safety, and cost challenges, leaving access uneven despite progress.
Scientists have reconstructed ancient enzymes from early cannabis relatives, revealing how the plant evolved to produce cannabinoids like THC and CBD. These findings provide insights into cannabis evolution and offer new biotechnological tools for producing medicinal cannabinoids more efficiently, including a specific enzyme that could lead to cannabis varieties with higher CBC content for medical use.
Scientists are making progress in transplanting genetically modified pig kidneys into humans to address organ shortages, despite ethical debates about creating human-like animals and the risks involved. The article discusses the scientific advancements, ethical concerns about animal welfare and moral status, and the regulatory history surrounding the use of animal organs and chimeras.
Despite political challenges, 2025 saw significant medical advances including a groundbreaking gene edit for a baby, approval of 44 new drugs, progress in cancer treatments, improved vaccines, and innovative biotech research, highlighting biology's pivotal role in this century of medicine.
Shares of Ultragenyx and Mereo BioPharma plummeted after their Phase 3 trial for the brittle-bone disease treatment setrusumab failed to meet primary endpoints, despite secondary improvements in bone density, leading to significant market value losses and cautious outlooks from analysts.
The article discusses the ongoing development of gene editing technologies, highlighting the excitement and competition among scientists to discover the most effective and final form of gene editing tools.