Cathy Miller

Six major advances in gene editing and gene therapy

Blog Post created by Cathy Miller on Oct 31, 2018

1. FDA and NIH simplify the regulation of gene therapy


On August 15th, Dr. Francis S. Collins, Director of the National Institutes of Health (NIH), and Dr. Scott Gottlieb, Director of the US FDA, published a joint article in The New England Journal of Medicine to explain NIH and FDA’s regulatory policy trends in gene therapy for the treatment of human diseases. The purpose of this move is to accommodate new market-based management needs for medical products based on emerging biotechnologies such as gene therapy and gene editing. The article points out that NIH has begun to integrate gene therapy into existing regulatory systems, change NIH guidelines for recombinant or synthetic nucleic acid molecule studies, and modify the role of RAC (Recombinant DNA Advisory Committee). In particular, RAC will no longer be required to review human gene therapy protocols.


2. Boehringer Ingelheim launches new collaboration to develop the first gene therapy for cystic fibrosis


On August 9th, Boehringer Ingelheim announced that it will launch a global partnership with the UK Cystic Fibrosis Gene Therapy Consortium (GTC, composed of Imperial College, Oxford University and Edinburgh University), Imperial Innovation and Oxford Biomedical Company (OXB) to develop a “first-in-class” therapy for long-term treatment for patients with cystic fibrosis. The collaboration will focus on introducing a healthy copy of the CFTR gene into lung cells through a novel approach using replication-deficient lentiviral vectors in inhaled formulations. This method has demonstrated a high efficiency of gene transfer, providing a possibility to maintain therapeutic effects by repeated administration. Gene therapy is by far the only treatment that addresses all CFTR mutations, thus providing the possibility of a universal treatment option.


3. Orchard completes $150 million Series C financing to advance multiple gene therapies for rare diseases


Four months after the success of GSK's rare disease gene therapy portfolio, the UK-based Orchard Therapeutics received an oversubscribed C-round financing of $150 million, which will be used to advance the three post-projects it received from GSK. These three items are OTL-101 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID), OTL-200 for the treatment of metachromatic leukodystrophy (MLD), and OTLS-103 for the treatment of Wiskott - Aldrich syndrome (WAS). In addition to these three post-projects, Orchard said the financing will also be used to advance clinical and preclinical development of its rare disease gene therapy pipeline. Orchard's other products from GSK include a clinical project for beta thalassemia, and three projects respectively for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD), and a preclinical project for malnutrition of globular white matter.


4. Astellas acquired Quethera for $100 million to advance ophthalmic gene therapy


Astellas announced the acquisition of Quethera, a UK-based gene therapy company that develops new treatments for eye diseases such as glaucoma. Under the terms of the agreement, Astellas may pay up to 85 million pounds ($109 million) in prepayments and contingent payments to Quethera. Upon completion of the acquisition, Quethera will become a wholly-owned subsidiary of Astellas. Through this acquisition, Astellas will receive Quethera's ophthalmic gene therapy program. The project uses a recombinant adeno-associated virus vector system (rAAV) to introduce therapeutic genes into target retinal cells for the treatment of glaucoma. The project's main preclinical drug candidates have been demonstrated in preclinical models to significantly improve retinal ganglion cell (RGC) survival.


5. Recombinetics completes $34 million Series A financing to accelerate the commercialization of gene editing technology


On August 21st, a genetic editing company in the United States, Recombinetics, announced the completion of a $34 million Series A financing, which will be used to accelerate the promotion of the company's gene editing technology to help humans find ways to treat diseases, develop new regenerative medicine products as well as cultivate healthier animals.


Founded in 2008 and located in Minnesota, USA, Recombinetics is recognized as one of the leading companies in the field of gene editing for large animal models. The company uses two breakthrough gene editing technologies - TALEN (transcriptional activator-like effect nuclease) and CRISPR/Cas9 for precise genetic alterations. The company's global intellectual property portfolio currently includes 22 patents (including, but not limited to, CRISPR and TALEN), as well as more than 300 patent applications for animal genetic editing methods, characterization and novel breeding methods.


6. PNAS: Scientists discover innovative gene therapy that is expected to treat hereditary blindness


Researchers at the National Eye Institute (NEI) and the University of Pennsylvania have used gene therapy to successfully prevent vision loss in the dog's hereditary retinitis pigmentosa (RP) model. This gene therapy may be used to delay or prevent vision loss in human patients with the same disease. In this study, the researchers used an innovative gene therapy strategy. First, shRNA technology was used to reduce RHO gene expression in rod cells, which attenuated the effects of toxic RHO proteins on rod cells. They then introduced a replacement RHO gene into the cell that expressed normal RHO protein but was not inhibited by shRNA. The advantage of this strategy is that it is no longer limited to correcting single gene mutations, and thus can treat all hereditary RP due to mutations in the RHO gene. To date, scientists have discovered that more than 150 RHO mutations can lead to hereditary RP. Researchers hope to launch preclinical safety trials within a year or two, and their ultimate goal is to conduct clinical trials in human patients.


About the author

During the last decade, scientists in Creative Peptides have become increasingly involved with immunotherapy, cell therapy or gene therapy in an effort to combat cancer and other major diseases. To this end, many other techniques have also been employed, including Silver Nanoparticles Conjugation, bioconjugation, peptide nucleic acid synthesis, surface plasmon resonance imaging, neoantigen peptides vaccine synthesis. For more information, please contact