Cellular Intelligence, backed by the Chan Zuckerberg Initiative, is building an AI model to predict how cells respond to signals over time, supported by a capsule platform that enables exponential experimentation. Here’s our interview with the founder on how it works, plans to advance Novo’s…
The randomized, double-blind study will enroll about 150 patients to receive intravenous ALT001 at one of two doses or placebo, with functional change assessed at 24 weeks.
The UMC Utrecht-sponsored, randomized placebo-controlled study plans to enroll 162 infants with MRI-confirmed hypoxic-ischemic brain injury and assess motor development at 24 months.
Researchers used whole-genome sequencing and a machine-learning RNA assay to assess dopamine neuron precursors made from patients’ skin cells for identity and consistency.
A small trial tested whether repeated donor cord blood cell infusions could safely influence social symptoms in children with autism and blood signs of immune dysregulation.
The analyses track skeletal muscle and heart outcomes through 24 months in 82 of 106 randomized participants, including delayed-start and natural-history comparisons submitted with a regulatory amendment.
The approval was based on a trial comparing 17 treated children with 27 untreated peers, with follow-up extending to nearly eight years and shipments expected within 30 to 60 days.
Backed by $6.4M in grants, the team will create and validate more than 20 patient-derived stem cell lines, then share the cells, protocols and data publicly.
The designation was based on early U.S. trial data in spinal cord injury patients, where this non-replicating HSV-1 gene therapy is being tested as a single-course treatment aimed at reducing bladder overactivity without impairing normal function.
The multicenter study will begin with 6 to 24 participants to select a dose, followed by about 95 participants divided into two disease-pattern cohorts.
Researchers administered the vesicles intranasally over time to female mice genetically engineered to model Alzheimer’s and separately tested them in neurons grown from patients’ induced pluripotent stem cells.
Nonclinical findings showed the investigational therapy reduced inflammation, tissue damage and pain sensitivity while promoting cellular repair, supporting its ongoing Phase 1 trial.
The designation gives Aspen added FDA support as it advances sasineprocel, a personalized autologous cell therapy for Parkinson’s disease, through clinical development.
The iPSC-derived neural cell therapy takes its next step in potentially restoring dopaminergic neurons lost to the condition.
The company is hoping to be the 2nd Taiwanese cell therapy approved abroad.
The Chinese company also received an IND in China a few weeks ago. Supported by good preclinical data, they'll advance clinical trials in both countries soon.
The published Phase I/IIa study found that MSC therapy reduced markers of brain inflammation and improved depression, fatigue, pain, and imaging biomarkers in adults with chronic traumatic brain injury, supporting ongoing larger controlled trials.
REGENXBIO said the FDA will consider existing longer-term data for NAVSUNLI under the accelerated approval pathway without requiring new studies, allowing the company to target a BLA resubmission in Q3 2026.
Developed in hand with the DoD, the company is hoping to lay the groundwork for cell therapy in concussive injuries for soldiers, athletes, and the broader population.
Their Chinese Phase 1 reportedly showed motor improvement in 90% of patients. With that data in hand, the FDA is allowing them to move forward out West.
Published in the Journal of Nanobiotechnology, the iPSC-derived therapy delivers BDNF mRNA to injured spinal tissue, improving motor recovery in preclinical studies.
The company is working on iPSC-derived, AAV, and small molecule therapies for neurodegenerative, orthopedic, and blood-based disorders, with the help of OpenAI's models which seem to be accelerating life science research.
Cellular Intelligence will use its AI platform to speed the clinical development and scalable manufacturing of a Phase 1/2 stem cell-derived Parkinson’s therapy acquired from Novo Nordisk, which also took an equity stake in the company.
The funding will support Latus Bio’s lead gene therapy programs in Huntington’s and CLN2 disease while advancing its low-dose AAV delivery platform designed to make gene therapies safer, more scalable, and accessible to broader patient populations.
The recommendation is backed by Phase III data showing that Novartis’ one-time gene therapy improved motor function in people aged two and older with SMA and could lessen reliance on ongoing chronic treatment.