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Brainstorm Therapeutics’ Stem Cell Therapy for ALS Fails to Meet Expected Results. 

Brainstorm Therapeutics recently revealed that its experimental treatment for ALS did not meet the main goal of a late-stage clinical trial, dealing the latest blow to a research field tainted by drug failures.

The study recruited patients with rapidly progressing ALS, or amyotrophic lateral sclerosis, and gave them either a placebo or a therapy developed by Brainstorm Therapeutics, to possibly treat neurodegenerative illnesses through the use of adult stem cells.

Although the therapy showed positive effects, however, it didn’t significantly outperform the placebo because of better-than-expected results in that group.

Moving forward, Brainstorm Therapeutics will use this to improve the therapy.

(Source: Biopharma Dive, 2020)

Fujifilm Cellular Dynamics and Lonza Enters Into A Partnership.

Fujifilm Cellular Dynamics and Lonza recently entered into a strategic partnership to leverage both companies’ expertise and technologies for the development of iPSCs through licensing agreements.

Under the agreement, Fujifilm Cellular Dynamics grants Lonza a non-exclusive right to use their patents related to iPSC generation, including episomal vectors and reprogramming factors, for the clinical manufacture and differentiation of iPSC lines for cell therapies. 

While Lonza grants FUJIFILM Cellular Dynamics expanded use of its non-exclusive license to its innovative NucleofectorTM technology, to enable efficient transfection of cells, stem cells and cell lines.

(Source: Contract Pharma, 2020)

 MeiraGTx’s AAV5-RPGR gene therapy for X-linked retinitis pigmentosa shows positive results.

Safety data suggest that AAV5-RPGR is generally safe and well-tolerated. No adverse effects were reported either. 

At 12 months, data from the phase 1/2 clinical study of AAV5-RPGR gene therapy for RPGR-associated X-linked retinitis pigmentosa showed significant improvement in retinal sensitivity and functional mobility with the low and intermediate doses.

In the low- and intermediate-dose cohorts, significant improvement in retinal sensitivity was observed at 3 months and 9 months and was maintained at 1 year, both in terms of mean retinal sensitivity and volumetric analysis of the 30° hill of vision on the Octopus 900 perimeter (Haag-Streit).

(Source: Healio, 2020)