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| Acute Myocardial Infarction Patients Treated Early With Cell Therapy Delivered With BioCardia’s HElix Endocardial Delivery System Improved in Ejection Fraction and NYHA Class May 23, 2019 – (BioCardia) – At 12 months, patients with Helix-delivered cell therapy experienced a 7.33 percent imprvement in ejection fraction at 12 months compared to the control group. Helix patients also showed an improvement in NYHA class of more than one class over control at six months. |
| Exuma Biotechnology’s Affiliate Shanghai Perhum Therapeutics Announces Preliminary Results of Two First-In-Human Solid Tumor CAR-T Products May 22, 2019 – (Exuma) – The is early radiologic evidence of antitumor activity. Of the 7 patients with recurrent or refractory stage IV metastatic renal cell carcinoma, 6 are alive with a median follow-up of 140 days. |
| Amphera Recruits First Patients to Phase II Study of MesoPher to Treat Pancreatic Cancer May 21, 2019 – (Amphera) – The trial is an open-label phase II study in 10 adult patients with surgically resected pancreatic cancer who have received adjuvant standard of care treatment. |
| Abeona Therapeutics Announces FDA Clearance of Investigational New Drug Application for ABO-202 Gene Therapy in CLN1 Disease May 21, 2019 – (Abeona) – ABO-202 is designed to deliver a functional copy of the PPT1 gene to cells of the central nervous system and peripheral organs using a combined intravenous and intrathecal delivery via the AAV9 vector. The company will provide guidance on the timing of the clinical trial later this year. |
| 25 Up-and-Coming Gene Therapies of 2019 May 20, 2019 – (GEN) – The number of gene therapy clinical trials inched up by 10 from the 362 recorded as of the end of 2018. More significantly, the number of clinical studies jumped 17% year-over-year from the 319 trials in progress as of the first three months of 2018. |
| Axovant Announces Dosing of First Patient in Clinical Program for AXO-AAV-GM1, a Novel Gene Therapy for GM1 Gangliosidosis May 16, 2019 – (Axovant) – Axovant expects initial data from the AXO-AAV-GM1 clinical program in the second half of 2019, as well as continued enrollment of patients in this program throughout 2019. The endpoints of the clinical study include safety, biomarker, neurodevelopment, and Magnetic Resonance Imaging and Magnetic Resonance Spectroscopy measures. |
Advances in the field
| Improving Viral Vectors for Hemophilia Gene Therapies by Tricking the Immune System May 22, 2019 – (Fierce Biotech) – Researchers integrated the protein CD47 into the surface of lentiviral vectors so they could escape detection and destruction by the immune system. In tests with monkeys, the LVs were able to transfer therapeutic genes to liver cells, the target in patients with hemophilia B, without causing toxicity. |
| Scientists Modify Viruses With CRISPR To Create New Weapon Against Superbugs May 22, 2019 – (NPR) – Scientists are developing a new kind of antibiotic, made out of viruses that have been genetically modified using the gene-editing tool CRISPR, to fight bacterial infections that are resistant to traditional antibiotics. |
| Mount Sinai Researchers Discover Placental Stem Cells That Can Regenerate the Heart After Heart Attack May 20, 2019 – (Mount Sinai) – To test the Cdx2 cells’ regenerative properties, the researchers induced heart attacks in three groups of male mice. They found that every mouse in the group with Cdx2 stem cell treatments had significant improvement and regeneration of healthy tissue in the heart. |
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