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5 Companies Leveraging CRISPR to Herald the End of Genetic Diseases

11 Sep 5 Companies Leveraging CRISPR to Herald the End of Genetic Diseases

CRISPR, which is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, is a revolutionary genome editing approach that helps change an individual’s DNA, by altering it at a specific location. The Crisper-Cas9, short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9, offers an efficient and faster alternative to existing genome editing techniques.

CRIPSR has the potential to offer therapeutic solutions to areas with large unmet medical needs, though still at a nascent stage, where its efficacy and safety is still being studied in animal models, the technology has the potential to be a game-changer in the treatment space of various diseases such as Cystic Fibrosis, Hemophilia, Sickle-Cell diseases, which are primarily single-cell disorders. Further research is underway in areas such as oncology, immunodeficiency disorders and cardiovascular ailments.

According to a report by Markets and Markets, the global CRISPR technology market that was worth USD 562 million in 2018, will grow at a CAGR of 25 percent, to reach USD 1.72 billion by 2023.

However, CRISPR remains a very controversial technology, as there are ethical and medical concerns about Germline cell and embryo genome editing. Furthermore, though edits to adult cells are only at the somatic level and hence any unintended outcome or damage is only limited to the individual, changes done in germine cells can be passed down to future generations. Further research and development is needed to fully understand the implications of this technology, which is currently being evaluated by various companies in clinical trials for multiple indications. We take a look at some of them below.

Intellia Therapeutics, (NASDAQ: NTLA)

Market Cap: $726.73M; Current Share Price: 14.38 USDChart
Data by YCharts

Intellia aims to be a full-spectrum genome editing company, by uncovering the potential of CRISPR/Cas9 in treating the underlying genetic causes of most chronic ailments across multiple indications, using its proprietary modular lipid nanoparticle delivery system. The company is not only aiming to personalize the treatments for individual patients but also simply the manufacturing process to facilitate a much larger application of the technology.

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As a pioneer in the genome editing field, the company is leveraging its expertise, experience and an extensive intellectual property rights portfolio, to create a strong pipeline using its delivery expertise. It is actively seeking product-centric partnerships and applying a multi-pronged approach to building a diverse pipeline to reduce risk and ensure efficient and optimal use of its resources.

Based on the ground-breaking work of Jennifer Doudna, the company’s co-founder, CRISPR/Cas9 technology can help in precision targeting and modification, at any location of a human genome, facilitating editing, deleting or repairing of dysfunctional genetic material.

The company’s pioneering work is backed by a robust intellectual property portfolio covering the use of CRISPR/Cas9 systems for genome editing, improvements and modifications of systems and components.

In addition it also protects the product guides and methods of treatment; mRNA optimization; lipid nanoparticle technologies for delivering protein/nucleic acid complexes and RNA into cells; and cell expansion technology relevant to stem cell-based therapies according to the company.

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Intellia is focused on creating engineered cells using CRISPR/Cas9 genome editing, which have the potential to treat numerous indications in immuno-oncology and autoimmune diseases, all in a single visit to the clinic for administration of off-the-shelf allogeneic cellular medicine. Its pipeline consists of both in vivo and ex vivo programs, including the use of Lipid Nanoparticle (LNPs), for delivery of the CRISPR/Cas9 complex to the liver. It is developing a proprietary program for the treatment of transthyretin amyloidosis (ATTR), in association with Regeneron Pharmaceuticals, (NASDAQ: REGN), besides candidates for alpha-1 antitrypsin deficiency (AATD), and inborn errors of metabolism (IEMs).

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The company has partnered with Novartis (NYSE: NVS), to develop a chimeric antigen receptor T cells (“CAR T cells”) and hematopoietic stem cells (“HSCs”), for autoimmune and inflammatory indications. Intellia is planning to submit an IND for NTLA-2001, its first in vivo candidate, for the treatment of transthyretin amyloidosis (ATTR) in mid-2020.

Sangamo Therapeutics, (NASDAQ: SGMO)

Market Cap: $1.27B; Current Share Price: 10.73 USDChart
Data by YCharts

The company is leveraging its expertise in gene therapy, genome editing and gene regulation technologies, to deliver adeno associated virus (AAV) and mRNA platforms based therapeutics for the treatment of inherited metabolic diseases, CNS, Oncology, hematology and immunology applications. Its zinc finger nuclease technology is the gold standard for genomic therapies when it comes to three critical criterions of specificity, precision, and efficiency. It is currently developing ZFN 2.0 platform, touted to be the next-generation ZFN construct, which is 20 times more active than current options, and has the potential to develop genomic therapies for a broad range of monogenic diseases.

Its lead product candidate for the treatment of Beta-Thalassemia is ST-400, a gene-edited cell therapy based on its ZFN genome editing technology, which can be used to alter antilogous hematopoietic stem cells (HSCs) using fetal hemoglobin to produce functional red blood cells. The company has tied-up with Sanofi (NASDAQ: SNY) for the development of this product candidate. It is currently engaged in a Phase 1/2 clinical trial to study the safety and efficacy of ST-400 in managing beta thalassemia. As part of the study, patients will be administered a one-time infusion of ST-400, created using a patient’s own blood stem cells, which are edited in a laboratory to produce blood with a higher oxygen-carrying capacity.

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In addition, its pipeline consists of product candidates for the treatment of Inherited Metabolic diseases, Hematology and CNS diseases such as MPS, Fabry Disease, Hemophilia, Sickle Cell Disease, Tauopathies, Huntington’s Disease to name a few. It has entered into strategic collaborations with numerous companies such as Kite, a Gilead company for engineered cell therapies for oncology, with Pfizer Inc. (NYSE: PEE), for gene therapy programs for Hemophilia A, with Bioverativ for hemoglobinopathies, including beta thalassemia and sickle cell disease and Shire International GmbH to develop therapeutics for Huntington’s disease as per the company. It has also signed an option agreement with Brammer Bio, a gene therapy contract development and manufacturing organization, to gain access to large-scale, commercial-grade AAV manufacturing.

In April 2019, the company raised over $136.2 million in net proceeds through a public offering of common stock.

Precision BioSciences, (NASDAQ: DTIL)

Market Cap: $422.95M; Current Share Price: 8.03 USD

Data by YCharts

The company is leveraging the power of Genome editing in solving unmet needs in areas such as Oncology, genetic illnesses, agriculture, by using its proprietary ARCUS platform. ARCUS, based on a natural genome editing enzyme namely a homing endonuclease, is a next-generation genome editing platform which replicates the nature’s genome editing capabilities. Homing endonuclease are non-destructive enzymes, which can recognise long DNA sequences that rarely occur once in a complex genome and trigger gene conversion by modifying the genome precisely through insertion of a new DNA Sequence.

ARC nuclease, a fully synthetic enzyme that can replicate the actions of homing endonuclease, is a first-in-class therapeutic-grade genome editing platform. The nuclease is small and can be modified to recognise a DNA sequence in any target gene. They are manufactured using in silico lab-based techniques to prevent any off-target activity and retain their gene editing efficiency. In addition, their potency and specificity can be controlled and regulated, based on learning from relevant models.

 The company’s proprietary platform is not limited to deletion alone, it can be used to insert, delete or modify DNA in an accurate and precise manner, leaving no chance for random DNA-breaks. It is also compatible with any delivery strategy, as it is a single protein polypeptide, expressed in a single gene.

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The company is using this cutting-edge technology to find solutions for indications in cancer immunotherapy, by applying its ARCUS genome-editing to cell-based immunotherapies taken from healthy donors to offer consistent and scalable manufacturing. It is also extending the application of ARCUS to genetic diseases such as cystic fibrosis, haemophilia, muscular dystrophy, and sickle-cell disease. In July 2019, the company opened a Manufacturing Centre for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP) compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor (CAR) T cell therapy products as per a company statement.

Furthermore, the company’s subsidiary Elo Life Systems, is developing solutions to eliminate harmful genes, introduced through cross-breeding or GMO and improve crop production to offer improved quality, nutrition and higher yields to meet the growing demands on agriculture.


Market Cap: $2.47B; Current Share Price: 45.79 USDChart
Data by YCharts

The company uses a CRISPR-associated (CAS) endonuclease, or enzyme, that acts as “molecular scissors” to cut DNA at a location specified by a guide RNA to deliver cutting – edge therapies for treatment of numerous genetic diseases. The technology was co-invented by Dr. Emmanuelle Charpentier, one of its scientific founders. It is focused on creating solutions for unmet medical needs by targeting gene editing and cellular engineering using its proprietary methodology.

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Its Beta-Thalassemia treatment approach is centered on editing-genes to increase the fetal hemoglobin count, by substituting HbF for the diseased hemoglobin in β-thalassemia and SCD patients. Its lead product candidate CTX001, uses a patient’s own blood cells and edits them using CRISPR/Cas9 to promote HbF expression.

This program is being developed in association with Vertex Pharmaceuticals (NASDAQ: VRTX). The company partnered with Vertex in 2015 to advance its beta-thalassemia and Sickle Cell programs, which was further extended to cover its DMD and Mytonic Dystrophy Type 1(DM1) programs. Vertex holds the rights to CRIPSR’s IP and delivery platforms for the aforementioned programs.

It is currently being evaluated in a phase 1 / 2 study, with a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs), in Subjects with Transfusion-Dependent β-Thalassemia. It is expecting topline results from the study in May 2022. In April 2019, CTX-001 received a fast track designation from the FDA.

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In addition its pipeline consists of CTX-110, an allogenic CRISPR/Case-9 gene-edited CAR-T cell therapy for Immuno-oncology applications characterized by CD19+malignanices, Regenerative medicine aimed at Diabetes Mellitus Type I and genetic diseases such as Duchenne Muscular Dystrophy (DMD), Cystic Fibrosis and Hurler Syndrome.

Editas Medicine, (NASDAQ: EDIT)

Market Cap: $1.28B; Current Share Price: 25.53 USDChart
Data by YCharts

The company’s proprietary genome editing platform has four interrelated components which are capable of editing, accessing, precision cutting and repair, a wide range of mutations by using specific technologies and capabilities. The platform also helps design products with the optimal capabilities to achieve the desired result including type of Cas9 or Cpf1, the structure of the guide RNA’s and the delivery vector to name a few.

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The company is targeting multiple modalities such as Nanoparticle, Engineered Cell, Viral Vector and Nucleic acid along with delivery mechanisms including IV, infusions, direct injection and Inhalation. In addition, it is targeting tissues in areas such as Bone marrow, Liver, Eye, Muscles and Lungs.

The company is focusing on therapeutics areas which are severely underserved, such as genetic and infectious eye diseases, engineered cells for the treatment of cancer, Non-Malignant Hematologic Diseases (Beta Thalassemia and Sickle Cell), DMD, Cystic Fibrosis and Lung Disease to name a few.

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The company, in partnership with Allergan (NYSE: AGN), is planning to initiating a phase 1 / 2  clinical trial named Brilliance in Q2, 2019, for EDIT-101 as a treatment for Leber congenital amaurosis 10 (LCA10), which will be the first ever clinical trial of an in vivo CRISPR medicine as per the company. Brilliance will be a multi-center, open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101 in 18 patients, aged 3 years and above. It is currently engaged in IND-enabling studies for YE19 and EDIT-301 for Sickle Cell Disease and Beta-thalassemia.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any position within the next 72 hours.

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