Harvard University

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder

8 days ago

• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder. • The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein. • This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.

Federal Circuit Remands CRISPR Patent Dispute to PTAB in Ongoing Editas Medicine Case

10 days ago

• The U.S. Court of Appeals for the Federal Circuit has partially vacated the Patent Trial and Appeal Board's previous decision in the CRISPR/Cas9 patent interference case, remanding it for further review. • Editas Medicine maintains confidence in its intellectual property portfolio, emphasizing that the decision does not affect existing licenses or its ability to license foundational CRISPR patents. • The company holds extensive patents covering CRISPR/Cas9 and CRISPR/Cas12a gene editing in human cells, with only a fraction involved in the ongoing interference proceedings.

Axoft's Ultrasoft Brain-Computer Interface Shows Promising Results in First Human Trial

a month ago

• Axoft has successfully completed its first four cases in a first-in-human clinical study of its novel Fleuron™ brain-computer interface, demonstrating safe implantation and reliable neural signal recording. • The Fleuron™ material is 10,000 times softer than traditional polyimide implants, allowing it to move with the brain's natural pulsations and significantly reducing tissue scarring and lead migration. • Neurosurgeons at The Panama Clinic implanted Axoft's devices in brain tumor patients, successfully recording high-density single neuron information and detecting a biomarker of consciousness during sensory stimulus tasks.

GLP-1 Medications Reshape Obesity Treatment Landscape as Bariatric Surgeries Decline 25%

a month ago

• The rise of GLP-1 medications like Wegovy and Zepbound has coincided with a 25.6% decrease in bariatric surgeries between 2022 and 2023, according to Harvard researchers. • U.S. obesity rates declined for the first time in a decade in 2023, dropping from 46.2% in 2021 to 45.6%, with the steepest reductions observed in the South where GLP-1 medication use was highest. • Despite their effectiveness, GLP-1 medications face challenges including high costs, gastrointestinal side effects, and supply shortages, which may influence future treatment patterns.

FDA to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Drugs

a month ago

• The FDA announced it will phase out requirements for animal testing of monoclonal antibodies and other drugs, citing the availability of "more effective, human-relevant methods" for safety evaluation. • The initiative aims to improve drug safety, accelerate evaluation processes, reduce R&D costs, and ultimately lower drug prices by implementing AI-based computational models, humanoid models, and real-world human data. • This regulatory shift, enabled by the bipartisan FDA Modernization Act 2.0 of 2022, represents what FDA Commissioner Marty Makary calls "a paradigm shift in drug evaluation" that could expedite development of new treatments.

Gene Editing Breakthrough: David Liu's Base and Prime Editing Technologies Transform Treatment of Genetic Diseases

2 months ago

• David Liu, Harvard professor and Broad Institute researcher, has been awarded the Breakthrough Prize for developing base and prime editing technologies that correct DNA mutations without cutting the double helix. • Liu's gene editing technologies have been distributed to over 20,000 non-profit labs worldwide and are currently being tested in 15 clinical trials across five countries, showing promising results for rare genetic diseases. • The technology has already demonstrated life-saving outcomes in patients with T-cell leukemia, sickle-cell disease, beta-thalassemia, and high cholesterol, making treatments for rare genetic diseases economically feasible.

Vizgen Settles Litigation with 10x Genomics and Harvard, Secures Future for MERFISH Technology

2 months ago

• Vizgen has reached a settlement agreement with 10x Genomics and Harvard University, ending litigation in both U.S. and European courts after three days of trial in Delaware. • The resolution secures Vizgen's freedom to operate its MERFISH technology, allowing the company to continue building its intellectual property portfolio in spatial biology without legal impediments. • Since introducing MERSCOPE in 2021, Vizgen's technology has been featured in over 300 scientific publications spanning neurodegenerative disease, oncology, and other research fields.

Artis BioSolutions Launches with Landmark Bio Acquisition to Transform Advanced Therapy Manufacturing

2 months ago

• Artis BioSolutions has emerged from stealth mode with the strategic acquisition of Landmark Bio, positioning itself as a premier CDMO for advanced genetic medicine development. • Landmark Bio, founded in 2021 by leaders from academia and Boston's research hospitals, will continue to operate independently while expanding its capabilities in cell and gene therapy manufacturing. • The acquisition aims to address critical bottlenecks in the rapidly growing genetic medicines sector by providing integrated end-to-end solutions from preclinical development through commercialization.

Roche Launches Innovation Center at Harvard's Enterprise Research Campus to Advance CVRM Research and AI Applications

3 months ago

• Roche and Genentech are establishing a new Innovation Center at Harvard's Enterprise Research Campus in Allston, focusing on Cardiovascular, Renal, and Metabolism research and AI applications in drug development. • Starting with a 30,000-square-foot facility, the center plans to expand to accommodate up to 500 employees under the leadership of Manu Chakravarthy, SVP and Global Head of CVRM Product Development. • The initiative builds upon Roche's decade-long partnership with Harvard University and represents a significant investment in the Boston healthcare ecosystem, reinforcing the company's commitment to the U.S. market.

Editas Medicine Achieves In Vivo Gene Editing Milestones, Plans Clinical Push

4 months ago

• Editas Medicine achieved in vivo preclinical proof of concept by editing hematopoietic stem cells in non-human primates, a crucial step for sickle cell disease and beta thalassemia treatment. • The company demonstrated in vivo editing of liver cells in non-human primates and delivered to two additional cell types in humanized mice, expanding therapeutic possibilities. • Editas anticipates declaring two in vivo development candidates by mid-2025, one for HSCs and one for the liver, alongside presenting further in vivo data. • Strategic priorities through 2027 include submitting at least one IND/CTA and achieving human in vivo proof of concept in HSC editing for sickle cell disease and beta thalassemia.