What are the breakthroughs in Sirna Therapeutics?
Herein, we highlight the breakthroughs in siRNA therapeutics, with special feature on the first FDA approved RNAi therapeutics Onpattro (Patisiran) and the consideration of effective siRNA delivery system focusing on current siRNA nanocarrier in clinical trials.
Which companies are winning approval for RNA-based treatments for polyneuropathy?
In July, the two companies won European Commission approval of their Tegsedi™ (inotersen), an RNA-targeted treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. The FDA followed suit three months later. 3. Alnylam Pharmaceuticals
Did any companies make it to the list of RNA-based drug companies?
Yet at least one company that didn’t make the list nevertheless left its mark in RNA-based drug development this past year. In June, Translate Bio (which changed its name last year from RaNa Therapeutics) completed its own smaller IPO, raising approximately $113.4 million in net proceeds.
What are the top RNAi and gene therapy companies?
Top RNAi and gene therapy companies Company Location Business Type EGEN (Expression Genetics) Huntsville, AL RNA/DNA Delivery Benitec Biopharma Sydney, Australia ddRNAi Susavion Biosciences Tempe, AZ Bio-active Peptides Tekmira Pharmaceuticals Burnaby, BC RNAi 33 more rows ...
How many siRNA drugs have been approved?
three therapeutic siRNAThe U.S. Food and Drug Administration (FDA) has since approved three therapeutic siRNA drugs, ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO® (lumasiran) developed and marketed by Alnylam® Pharmaceuticals. In addition, several other drugs are in the late stages of clinical trials.
Who discovered siRNA?
SiRNAs were first discovered by David Baulcombe's group in Norwich, England, as part of post-transcriptional gene silencing (PTGS) in plants, and published there findings in Science in a paper titled "A species of small antisense RNA in posttranscriptional gene silencing in plants." Shortly thereafter, in 2001, ...
Where is siRNA made?
siRNA are generally considered to have come from longer strands of exogenous growing or originating from outside an organism (RNA which is taken up by the cell and undergoes further processing). The RNA often comes from vectors, such as viruses or transposons (a gene that can change positions within a genome).
How much does siRNA cost?
Standard siRNA SynthesisStandard Custom siRNA Duplex Price ListStandard Purity*PAGE Purified*10 nmol$145.00$465.0020 nmol$180.00$570.0050 nmol$225.00$720.006 more rows
How siRNA can be used as therapeutics?
The sequence-specific gene-silencing by siRNA can be used as a new therapeutic approach for treatment of a variety of diseases that are incurable by conventional drugs. Many efforts have been made to overcome the problems related to delivery, stability, off-target gene silencing and immunostimulatory effects of siRNA.
Who developed RNAi?
Two US geneticists who discovered one of the fundamental mechanisms by which gene expression is controlled have received a Nobel prize for their achievement. Andrew Fire and Craig Mello, who revealed the process of RNA interference (RNAi) in 1998, will share the US$1.4-million award.
How is siRNA manufactured?
These siRNA duplexes are produced naturally when an enzyme, Dicer, cleaves long dsRNA (double-stranded RNA) into smaller fragments. The resulting 21-23 nucleotide dsRNA fragments, i.e. siRNA, then associate with an RNase-containing complex to form the RISC (RNA-induced silencing complex).
How siRNA is produced?
Chemical synthesis. In vitro transcription. Digestion of long dsRNA by an RNase III family enzyme (e.g. Dicer, RNase III) Expression in cells from an siRNA expression plasmid or viral vector.
Is siRNA synthetic?
In principle, any gene can be silenced by a synthetic siRNA with a complementary sequence. This makes them an important tool for drug targeting and validating gene function. There have been many important studies into the application of siRNA to different areas of medical research.
Where can I buy siRNA?
We offer traditional 21mer duplex RNAs as well as 27mer, Dicer-substrate RNAs (DsiRNAs), which engage the Dicer enzyme natural siRNA processing pathway. If you know the sequence you want to order, you can order either from the Order tab at https://www.idtdna.com/ by selecting Main Menu>Custom RNA Oligos.
How do you design siRNA?
To design a negative control siRNA, scramble the nucleotide sequence of the gene-specific siRNA and conduct a search to make sure it lacks homology to any other gene. Additional siRNA sequences targeting the same mRNA.
How much does shRNA cost?
shRNA Cloning ServicesshRNA Cloning ServicesAvailable BackbonesPrice1 shRNA constructAAV, lentivirus, and adenovirus$2754 shRNA constructs + 1 scrambled controlAAV and lentivirus$750Adenovirus$9954-in-1 shRNA construct + 1 scrambled controlAAV$9952 more rows
What is olix asymmetric siRNA?
OliX’s asymmetric siRNA (asiRNA) technology not only shows comparable gene silencing compared with a conventional siRNA, but also significantly reduces siRNA-mediated side effects, such as off-target gene silencing and immune stimulation. cp-asiRNA...
Who is Leonardo Biosystems?
Leonardo Biosystems is a drug delivery company built around technology developed by Dr. Mauro Ferrari, one of the world's best-known nano-science innovators. Leonardo has a multi-stage delivery platform that has been shown in animal models to be...
What is Neuregenix Ltd?
Neuregenix Ltd is a UK company developing products and services for regenerative neurobiology with a specialist interest in the area of acute brain and spinal cord injury and the development of novel therapies to enhance post-injury functional...
What is Phaserx Inc?
PhaseRx Inc., (PhaseRx) is an RNAi delivery technology company. The company is involved in developing a breakthrough polymer-based system, which enables the systemic delivery of RNAi-based drugs and potentially other macromolecules as therapeutic...
What is ribometrix biotechnology?
Ribometrix is a biotechnology company developing small molecule therapeutics that directly target RNA to treat human diseases. The Ribometrix team leverages its world-leading expertise in 3D structural analysis of RNA to drive its discovery...
Why was Mitotherapeutix created?
Mitotherapeutix was created to take advantage of advancements in RNA chemistry and its use in developing siRNA based drugs that can target otherwise difficult to address disease targets. Our initial gene target is a novel protein that controls...
How many base pairs are in a DsiRNA?
Dicerna's DsiRNA molecules are uniquely structured and different from first generation siRNAs. Specifically, the company's DsiRNA molecules are 25 or more base pairs in length and are processed by the Dicer enzyme. The ability to engage the Dicer...
What is the FDA approved drug for RNA interference?
Alnylam Pharmaceuticals. Alnylam won the first-ever FDA approval for an RNA interference (RNAi) drug in the U.S. on August 10, when the agency authorized Onpattro™ (patisiran) for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults.
How much did Biontech raise?
BioNTech raised a hefty $270 million Series A financing in December 2017 led by the Redmile Group, and intended to advance its clinical immunotherapy pipeline, led by its mRNA-based Individualized Vaccines Against Cancer (IVAC®) Mutanome and FixVAC® cancer vaccines. The Series A came about 10 years after the company attracted $180 million in seed funding in December 2008. BioNTech turned heads again in August by scooping up $120 million upfront, equity and near-term research payments from Pfizer under a collaboration to develop mRNA-based vaccines for prevention of influenza. The collaboration could also generate $305 million for BioNTech in milestone payments from Pfizer, which joined several other biopharma giants in partnering with BioNTech, including Genentech, a member of the Roche Group, Eli Lilly, Sanofi, Genmab, and Bayer Animal Health.
What is the purpose of Onpattro?
On August 30, the European Commission followed suit and approved Onpattro, which is designed to interfere with RNA production of an abnormal form of the protein transthyretin (TTR).
What is Tegsedi inotersen?
In July, the two companies won European Commission approval of their Tegsedi™ (inotersen), an RNA-targeted treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. The FDA followed suit three months later. 3. Alnylam Pharmaceuticals.
Where is Gradalis manufactured?
Gradalis has been on an expansion track this year, closing on a $55 million Series C financing and upgrading its current Good Manufacturing Practice (cGMP) manufacturing facility in Carrollton, TX, to support the launch of a Phase III registration trial of its immunotherapy platform Vigil™ plus chemotherapy drugs Irinotecan and Temozolomide in Ewing’s sarcoma ( NCT03495921 ). Ewing’s sarcoma is one of several advanced cancer indications for which Vigil is being developed; the platform is also in combination studies with checkpoint inhibitors targeting advanced gynecological and other women’s cancers. Gradalis raised $10 million in a Series B round in 2013, and $10 million in a Series A round six years earlier.
Is Moderna Therapeutics an IPO?
Earlier this month, Moderna Therapeutics carried out the largest-ever initial public offering (IPO) for a biotech. And in August, Alnylam won the first-ever FDA approval for an RNAi drug in the U.S. Both companies appear on GEN’s first-ever list of top biopharmas focused on leveraging RNA-based treatments. Yet at least one company that didn’t make ...
What is AstraZeneca's specialty?
AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal and Metabolism, and Respiratory.
What is Silence Therapeutics?
AstraZeneca will collaborate with Silence Therapeutics to discover, develop and commercialise small interfering RNA (siRNA) therapeutics for the treatment of Cardiovascular, Renal, Metabolic and Respiratory diseases.
Where is AstraZeneca located?
Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @ AstraZeneca.
What is the role of small interfering RNA in the cell?
Small interfering RNA (siRNA) are double-stranded RNA molecules that offer new opportunities for therapeutic intervention because they act inside the cell to influence protein production. They do this by targeting RNA to prevent the production of disease-causing proteins. 1
How many RNA targeted therapies are there?
About a hundred investigational RNA-targeted therapies are in the pipelines today. A series of events, including the approval of RNAi drug Patisiran (Onpatto) and antisense oligonucleotide Nusinersen (Spinraza), and especially development of Milasen drug -- all have marked the point when RNA targeted therapies are taking off. The RNA medicines are an alluring technology for several reasons: the potential possibility of shortening the drug development time down to months instead of years; it could be applied for intractable diseases that are hardly treatable with conventional therapies; and in opposite to gene editing, the action of RNA targeted therapies are reversible that largely mitigates potential risks of side effects.
How much money did Triplet Therapeutics raise?
One of them is Triplet Therapeutics, which has raised $59 Million at the end of 2019 for development of transformative treatment for Huntington’s Chorea along with other triplet repeat disorders.
What is the first antisense drug?
Let's start with the first approval of antisense drug for treating Spinal Muscular Atrophy (SMA), Spinraza, announced in 2016. SMA is a fatal neurodegenerative disease caused by a malfunctioning of SMN2 protein -- it affects on average 1 in every 10.000 people globally. Researchers from Ionis and Biogen managed to reverse the effect of the genetic error at the stage of RNA splicing. The drug works through binding to the target mRNA sequence forcing exon, which is being skipped by an error in the genome, to be translated into a full-length protein. Approval of Spinraza has changed the situation with RNA-targeted therapies, resurging the field which was largely abandoned by investors in the mid-2000s. In 2016 the market was also replenished with Exondys 51, a drug by Sarepta Therapeutics, targeting Duchenne Muscular Dystrophy (DMD).
What are the two categories of RNA-targeting medicines?
Alongside the topic of gene editing technologies that keeps making headlines these days, there is also a wave of breakthroughs in the field of RNA-targeting medicines, primarily falling into the following two categories: antisense oligonucleotides (ASO), and RNA interference (RNAi) technologies. (another promising approach, dealing with editing RNA itself by ADAR enzymes, is not covered in this post).
Who is paying attention to RNAi?
Biotech investors, naturally, are also paying close attention to RNAi. To help in that effort, read on for more about seven drug and biotech companies focused on developing RNAi drugs.
How does RNAi work?
They potentially work by silencing specific messenger RNAs that are responsible for making disease-causing proteins. If you can prevent these proteins from being made, you can potentially stop or cure the disease.
What is the ISIS pipeline?
Status: Isis focuses primarily on developing drugs using antisense technology, which is a cousin to RNAi. The company's drug pipeline includes drugs targeting metabolic, cancer and inflammatory diseases in early and mid-stage human clinical trials.
Is Sangamo a partner?
Partners: Sangamo's human drugs are not partnered. The company has a plant agriculture partnership with
Is RNAi still in development?
RNAi drugs are still in the early stages of clinical development, but the field is drawing a lot of attention from Big Pharma and smaller biotech startups, companies racing to develop the first successful RNAi-based drug. The 2006 Nobel Prize for Medicine was awarded to two RNAi research pioneers.
What is CRISPR therapeutics?
Emmanuelle Charpentier. It is associated with developing transformative gene-based medicines for cellular therapy of genetically-defined diseases. The company, in partnership with Vertex Pharmaceuticals, has developed CTX001, a therapeutic program in a clinical trial phase, which is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy for patients suffering from β-thalassemia and sickle cell disease with an ex vivo approach. Two of the immuno-oncology cell therapy programs CTX110 and CTX120 are in the pre-clinical phase. CTX110 targets CD19, an antigen expressed in various B-cell malignancies, while CTX120 targets BCMA, an antigen expressed in multiple myeloma. Other CRISPR/Cas9 gene-edited programs being researched include CTX130 that targets CD70 for the treatment of both solid tumors and hematologic malignancies, regenerative medicine approach in partnership with ViaCyte, combining CRISPR and stem cells to treat type 1 diabetes, as well as, in vivo approaches for the treatment of Glycogen storage disease type Ia, Duchenne muscular dystrophy, cystic fibrosis, and Hurler syndrome.
What is CRISPR technology?
CRISPR gene-editing technology is driving every aspect of biotechnology, including molecular biology, genetics, oncology, immunology, agricultural and industrial biotechnology, and even food technology. Since its discovery, new companies have been founded to deliver advantages of CRISPR to various sectors, and many of the existing gene-editing ...
What is Agenovir Corporation?
Agenovir Corporation is associated with the research and development of antiviral therapeutics against diseases caused by latent or persistent viral reservoirs. The company utilizes computationally engineered nuclease technologies, including CRISPR/Cas9 gene-editing system, to target and disrupt pathogenic viral genomes. The company’s first target is the Human Papilloma Virus (HPV) and plans to target hepatitis B, cytomegalovirus, Epstein-Barr virus, and Herpes simplex virus.
Who is Jennifer Doudna?
Jennifer Doudna, a co-discoverer of CRISPR gene editing, and colleagues co-founded Intellia Therapeutics aiming to develop therapeutic programs in areas of substantial unmet medical need with the tool of CRISPR/Cas9 gene editing. The company has a partnership with Regeneron Pharmaceuticals to treat Transthyretin Amyloidosis (ATTR) by an in vivo approach, which is already in the latter stage of preclinical development. Other in vivo pipeline includes programs targeting alpha-1 antitrypsin deficiency (AATD), and Primary Hyperoxaluria Type 1. The ex vivo programs involve developing engineered therapies to treat various oncological and autoimmune diseases and are also focused on chimeric antigen receptor T (CAR-T) cells and hematopoietic stem cells (HSCs).
Who is the founder of Editas Medicine?
Editas Medicine. Co-founded by one of the pioneers in CRISPR gene editing Dr. Feng Zhang, Editas Medicine has its lead program EDIT-101 accepted by FDA as an Investigational New Drug (IND) and is set to be the first in vivo CRISPR medicine administered to people anywhere in the world.
Where is Inari located?
Headquarters: Cambridge, MA, United States. Inari Agriculture aims to enhance the agricultural biotechnology sector with CRISPR gene-editing technology. Inari develops customized seeds to fit local conditions, such as humidity, day and night temperatures, and soil type of a farm.