Your Missing Links Are Here (19 March 2021)

Top pick

• To advance the treatment of Duchenne Muscular Dystrophy (DMD), efficient restoration of dystrophin expression in induced myotubes has been achieved using SpCas9 and dual gRNAs. A Danish-Chinese cooperation was behind the achievement, and the results were published in Molecular Therapy - Nucleic Acids.

Research

• Long term follow-up of five patients treated with gene therapy (not gene editing) of stem cells for X-linked severe combined immunodeficiency (SCID-X1) shows sustained beneficial effects after 18 years. This happened despite that the body had cleared the corrected stem cells.
• β-thalassemia is due to an imbalance of too little β-globin (HBB) and too much α-globin (HBA1/2). A novel CRISPR gene editing strategy targets both issues by replacing the entire HBA1 gene with a full-length HBB transgene in hematopoietic stem and progenitor cells (HSPCs). The American researchers established proof of concept for the therapeutic strategy for curing β-thalassemia in mice. We recently described a similar approach developed by Mario Amendola at Genethon.

Industry

• SNIPR Biome, a Danish CRISPR microbiome company, specialising in the precision killing of bacteria, presented its business model at the BioCapital Europe event last week. You can watch the presentation by CEO Christian Grøndahl here.
• A long read in Fast Company Magazine entitled "Meet the CRISPR pioneers who are making gene editing easy" tells an inspiring story of Inscripta's innovative microfluidics device for precise gene editing, Onyx.
• Precision BioSciences reports fourth quarter and fiscal year 2020 financial results and provides business update for its proprietary ARCUS genome editing platform. ARCUS is based on a homing endonuclease found in algae called I-Crel.
• European Medicines Agency (EMA) has granted Orphan Designation of BIVV003 - an investigational ex vivo gene-edited cell therapy by Sangamo Therapeutics. BIVV003 uses zinc finger nuclease technology to treat sickle cell disease.
• Beam Therapeutics announces fourth quarter and full-year 2020 financial results and recent business highlights and pipeline updates. The biotechnology company develops precision genetic medicines through base editing.
• Investors wonder if CRISPR Therapeutics can rebound after its stocks lost 12.6% since last month's earnings report. CRISPR Therapeutics reported fourth-quarter 2020 loss per share of $1.50, wider than the Zacks Consensus Estimate of a loss of $1.23.
• The Japanese biopharma company AnGes has invested $61 million in Emendo Biotherapeutics to further develop OMNI. It is a pioneering next-generation allele-specific gene-editing platform that uses synthetic biology.

Vision and opinion

• Actor Morgan Freeman and CRISPR pioneer George Church are partnering to develop and produce a drama series centred on the dangerous and life-affirming ways the technology can impact the world. The series explores the personal and global implications of cutting-edge gene editing and engineering technologies being used to combat climate change.
• Yet another review recommends Walter Isaacson's book "The Code Breaker" about Nobel laureate Jennifer Doudna. George J. Annas wrote the review in Science, and it is accompanied by an editorial that takes a look behind the scenes of the CRISPR story.

Reviews

• Strategies to improve sterile insect control by precise introduction of orthologous point mutations in pest insect species with CRISPR/Cas9 genome editing technology is presented in a review in Insects.
• Directed evolution represents a promising strategy to improve the existing genome editing systems and enable new editing functions. A review in Current Opinion in Chemical Biology looks at recent strategies to harness the power of directed evolution to improve genome editing systems.
• A review of current management and clinical trials for Leber congenital amaurosis/early-onset severe retinal dystrophy has been published in the British Journal of Ophthalmology. Nine ongoing clinical trials targeting specific genes are listed.
• An overview of gene therapies used for the treatment of diseases of the eye is presented in Ophthalmology Times. Larry Hanover wrote the piece that also covers other diseases.

Conferences and events

• An online webinar by the International Association of Lawyers will host a webinar on "The Future of Humanity: Genetic Engineering, CRISPR, and Beyond". The webinar will focus on laws and regulations, including those governing medical care, experimentation, intellectual property rights, and the technology commercialisation. You can register for the webinar here.