Cartilage Regenerating therapy, Invossa, pulled from market despite lack of evidence of harm

The health authorities canceled the license for Kolon Life Sciences' osteoarthritis gene therapy drug Invossa, Tuesday, over the mislabeling of a key ingredient. In addition, they have decided to pursue criminal charges against the pharmaceutical firm.Invossa was the first regenerative gene therapy drug for patients with the degenerative joint disease. The Ministry of Food and Drug Safety approved it use in Korea...

Inhibiting Macrophage Death May Offer New Therapy for Rheumatoid Arthritis

A collaborative study by research groups from the University of Cologne, VIB, Ghent University, the Βiomedical Sciences Research Center ‘Alexander Fleming’ in Athens and the University of Tokyo identified a new molecular mechanism causing rheumatoid arthritis. The researchers found that death of macrophages, an immune cell type, can trigger the disease. Moreover, they discovered how the protein A20...

Semma Therapeutics Announces Publication Demonstrating Advancements in Regenerative Cell Therapy for Type 1 Diabetes

Semma Therapeutics, Inc., a biotechnology company pioneering the curative use of stem cells in regenerative medicine, today announced the journal Nature published data demonstrating improvements in the production of beta cells (cells that produce insulin) from stem cells. Led by Harvard University researchers in collaboration with scientists from Semma, the study characterized the types of cells produced by the applicable cell differentiation methods,...

GenSight Biologics reports positive data in Phase III clinical trial of GS010 gene therapy for Leber Hereditary Optic Neuropathy

Durable improvement in best-corrected visual acuity (BCVA), as GS010-treated eyes sustain +15.4 ETDRS letters equivalent improvement versus baseline and +28.1 ETDRS letters equivalent versus nadirBilateral improvement continues in key visual functions BCVA and contrast sensitivityResponder analyses suggest improved clinical outcomes for GS010-treated eyes relative to sham-treated eyesGS010 safety and tolerability established over the course of the trialBriefing packages incorporating findings under preparation for...

GenSight Biologics announces positive DSM Board review and continuation of PIONEER Phase I/II clinical trial of GS030 gene therapy for Retinitis Pigmentosa

GenSight Biologics announced that the independent Data Safety Monitoring Board (DSMB) completed its first safety review of the ongoing PIONEER Phase I/II clinical trial of GS030 combining gene therapy and optogenetics for the treatment of Retinitis Pigmentosa. The DSMB confirmed the absence of any safety issues for the first cohort of three subjects who received a single intravitreal...

Neurocrine Biosciences and Voyager Therapeutics announce positive phase 1 results of genetic therapy for Parkinsons disease.

Results Confirm the Posterior Trajectory as an Additional Surgical Delivery Route for VY-AADC in Patients with Parkinson’s DiseaseTreatment with VY-AADC Improved Good ON Time (ON Time Without Troublesome Dyskinesia) by 1.7 Hours and Reduced OFF Time by 2.2 Hours at 12 Months in Patients with Parkinson’s Disease SAN DIEGO, CA and CAMBRIDGE, MA, USA I May 05, 2019 I ...

Researchers putting the brakes on lethal childhood cancer

Malignant rhabdoid tumor (MRT) is one of the most aggressive and lethal childhood cancers. Although rare — about 20 to 25 new cases are diagnosed annually in the United States — there is no standard effective treatment for the disease, which is driven by loss of an anti-cancer protein called SNF5. The chances are very small that a child will...

Kolon Life Science’s stock plunges on Invossa cell labeling fiasco – and other developments.

The hits keep coming for Kolon Life Science. Early statements that the company was unaware that the GP-293 cells were present in the therapy appear to be falling apart. Export deals with Mundipharma and other companies have fallen apart, and a complete stop-sale is in place for the cartilage regenerating drug under suspicion that the GP-293 cells may be harmful,...