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dydo last won the day on July 21

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About dydo

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    Biotech SGMO, PTLA

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  1. dydo

    Genomic Index

    I have adjusted the index to be an equal weight investment with $20K for each stock
  2. dydo


    uniQure Announces Development of a Highly Potent, Next-Generation Promoter for Liver-Directed Gene Therapies ~ New Data Presented at the European Society of Gene and Cell Therapy Annual Meeting Demonstrates up to 40 Times Higher Liver Expression with Optimized Promoter ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 18, 2018 (GLOBE NEWSWIRE) -- uniQure N.V. (QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today presented non-clinical data demonstrating that a next-generation synthetic promoter developed for liver-directed gene therapy is able to drive gene expression at increased levels and with very high specificity, allowing uniQure to tailor expression levels required for a specific therapeutic transgene. These data were featured in an oral session at the 26th Congress of the European Society of Gene and Cell Therapy (ESGCT), being held from October 16 – 19, in Lausanne, Switzerland. A “promoter” is an essential component of a gene therapy construct that controls expression of a therapeutic protein. Most gene therapies incorporate natural promoters, which have limitations and may not optimize the expression of genes in specific target cells. Consequently, natural promoters may not be appropriate for gene therapies that require higher levels of gene expression and tissue specificity. In collaboration with Synpromics Ltd, a U.K.-based developer of gene expression solutions, uniQure has developed synthetic promoters designed to enhance gene expression in a highly specific manner. Two promoter libraries and several data-driven rational designs were analyzed in-vitro for strength and specificity. Selected promoters then underwent additional design optimizations and were further validated through non-clinical testing. Non-Clinical Data Findings: The first in-vivo comparison study was performed in a mouse model injected with AAV incorporating a reporter gene and an optimized promoter candidate. The expression of the reporter gene using the optimized promoter was compared to that using a standard reference promoter. Data from this study showed the optimized promoter was capable of generating up to a 40-fold increase in expression compared to the reference promoter. A second in-vivo comparison study was performed in non-human primates (NHPs) and demonstrated that the optimized promoter was able to express a therapeutically relevant protein in NHPs, maintaining 8-fold higher protein levels over the reference promoter at 8 weeks post-injection. Additional data from a total of 15 validated promoter designs show that each performed better than the reference promoter in expressing the reporter gene both in in-vitro and in-vivo studies. “The data from these studies underscore the potential value of uniQure’s gene therapy technology platform built over the last two decades,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “These novel promoters result in a significant increase of the expression of a therapeutically relevant gene, which may prove to be critical to optimizing clinical outcomes for many liver-directed disorders. We believe this proprietary technology will be important in the development of next generation, liver-targeted gene therapy programs, and we look forward to sharing additional details on the applicability of these promoters at our investor-focused R&D Day next month.”
  3. Rocket Pharmaceuticals Presents Updated Long-Term Phase 1/2 Clinical Data of RP-L102 in Fanconi Anemia Patients at the 2018 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) NEW YORK--(BUSINESS WIRE)-- - RP-L102 Continues to Demonstrate Increasing and Durable Engraftment (Peripheral Vector Copy Number) In Fanconi Anemia Patients for Up to Thirty Months with First-Generation Process – Rocket Pharmaceuticals, Inc. (RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, presents updated long-term clinical data from its ongoing Phase 1/2 clinical trial of RP-L102 for Fanconi Anemia (FA) at the 2018 Annual Congress of the ESGCT in Lausanne, Switzerland. Title: Gene Therapy Trial in Non-Conditioned Fanconi Anemia Patients Session: Blood Disorders I Presenter: Paula Rio, Ph.D., Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)/ CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz Four patients have met the requisite 12-month follow-up time for long-term stem-cell engraftment analysis. As evidenced by progressively increasing vector copy number (VCN) in peripheral blood mononuclear cells, RP-L102 continues to demonstrate durable engraftment in all four patients, ranging from 18 to 30 months since gene therapy administration. Patients also demonstrated improved resistance of bone marrow CD34+ cells in the presence of mitomycin-C (MMC) and durable increases in peripheral T-lymphocyte chromosomal stability in the presence of diepoxybutane (DEB). MMC- and DEB-resistance are two key diagnostic measures of functional and phenotypic correction in FA. Trends for continued stabilization of previously-declining blood counts and the progressive increase of corrected versus non-corrected peripheral blood leukocytes continued for all patients. These patients were treated with “Process A”—the first one developed for RP-L102. Of note, the first patient in this cohort (Patient 2002)—whose stem cells were collected at an early age and is representative of the target demographic—experienced improvements in peripheral VCN from 0 at month 0, to 0.43 at month 24, and to 0.55 at month 30. Patient 2002 also demonstrated improvements in bone marrow MMC-resistance from 0% to more than 70% at month 24, approaching the normal range. “These data are very encouraging,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “The improvements highlight the natural selective advantage that uniquely exists in FA for gene corrected stem cells over diseased stem cells and obviates the need for conditioning. Furthermore, the data reflect the potential of RP-L102 to restore the functionality of bone marrow hematopoietic stem cells.” In the first half of 2019, Rocket plans to treat patients in the U.S.—under a Rocket-sponsored Investigational New Drug (IND) application—and in the EU with “Process B” RP-L102. This new optimized process incorporates higher cell doses, transduction enhancers, and commercial-grade vector. Data from this presentation will be available in the Company’s corporate presentation at: www.rocketpharma.com/pipeline/. About RP-L102 (LVV-based gene therapy for Fanconi Anemia) RP-L102 is Rocket’s lentiviral vector (LVV)-based gene therapy in development for patients with FA with Rocket’s collaboration partners at Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain, CIBER-Rare Diseases and IIS-Fundación Jiménez Díaz. The International Fanconi Anemia Gene Therapy Working Group helped develop the structure of RP-L102, which begins with a HIV-1-derived, self-inactivating lentiviral vector. RP-L102’s lentiviral vector carries the FANC-A gene as part of the PGK-FANCA-WPRE expression cassette which includes a phosphoglycerate kinase (PKG) promoter and an optimized woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The ex vivo administration process begins with the removal and isolation of hematopoietic stem cells using a CD34+ selection process. Autologous genetically modified CD34+ enriched hematopoietic cells (fresh or cryopreserved) are infused back into patients to restore function. RP-L102 is currently being studied in a Phase 1/2 clinical trial in the European Union with an Investigational Medicinal Product Dossier (IMPD) in place with the Spanish Agency for Medicines and Health Products. RP-L102 has been granted Orphan Drug designation for the treatment of Fanconi Anemia type A in the United States and in Europe. About Fanconi Anemia Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning, which is highly toxic for the patient. HSCT is frequently complicated by graft versus host disease and also increases the risk of many solid organ malignancies. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for FA diagnosis. The DEB assay can further differentiate FA patients from somatic mosaic patients. Somatic mosaicism occurs when there is a spontaneous reversion mutation that can lead to a mixed chimerism of corrected and uncorrected bone marrow cells leading to stabilization or correction of an FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism provides strong rationale for the development of FA gene therapy and demonstrates the selective advantage of gene-corrected hematopoietic cells in FA1. 1Soulier, J. ,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336
  4. BEDFORD, Mass., Oct. 18, 2018 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (FIXX), a genetic medicines company, announced today long-term efficacy data of a single dose of its lead gene therapy candidate for the treatment of phenylketonuria (PKU), an inborn error of metabolism. The presentation at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress showed sustained reduction of phenylalanine (Phe), the surrogate biomarker for PKU. Homology plans to begin and report initial clinical data from a Phase 1/2 gene therapy trial in adults with PKU in 2019. “We are excited to share this long-term data demonstrating that our gene therapy development candidate can address and correct the genetic cause of PKU in a well-established animal disease model,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our approach, which delivers functional copies of the human PAH gene, may offer a one-time potential cure for PKU that would restore the natural biochemical pathway. We look forward to starting our Phase 1/2 trial and sharing initial patient data next year. In preparation for the clinic, GMP manufacturing capabilities are sufficient to support our planned clinical program, and we expect to produce supply for other pipeline programs in our new GMP Phase 1/2 manufacturing facility, which is on track to be completed this year.” The data presented at ESGCT demonstrate that a single injection of Homology’s gene therapy candidate resulted in sustained reduction of phenylalanine to normal levels for 48 weeks post-treatment in the preclinical disease model. Importantly, levels of tyrosine increased; tyrosine is a natural byproduct of phenylalanine metabolism and is required for the production of neurotransmitters. In addition, coat color changed, which is indicative of melanin production, a byproduct of phenylalanine metabolism. About Phenylketonuria (PKU) PKU is a rare, inherited inborn error of metabolism caused by a mutation in the PAH gene. The current standard of care is a highly restrictive diet, but it is not always effective. If left untreated, PKU can result in progressive and severe neurological impairment. PKU is estimated to affect approximately 15,000 people in the U.S. and there are currently no treatments available that address the genetic defect in PKU.
  5. It is fascinating I recommend observe for time being. There are three editing methods, CRISPR, Zinc Finger, and Talen. I believe in SGMO's ZFN as most able, safe and furthest in the clinic. I will be posting on occasion. It is a tough if not tougher than solar sector
  6. dydo

    Genomic Index

    Results of the index October 18, 2018
  7. Intellia scientists present first robust demonstration of CRISPR-mediated insertion of transgenes in the liver Non-human primate data show high correlation achieved between liver edit and reduction of TTR protein High rate and specificity of acute myeloid leukemia cell killing observed with genome-edited, WT1-targeting T cells CAMBRIDGE, Mass., Oct. 18, 2018 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, presented new data from three of its programs, including the company’s first data on complex edits, at the 26th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), in Lausanne, Switzerland. “We are extremely pleased to present an outstanding compilation of data today reflecting progress in our preclinical genome editing programs,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We showed that we can efficiently introduce complex edits in mice by inserting genes to express proteins that are deficient in some genetic diseases. By using our LNP delivery system in combination with AAV to deliver template DNA, we are opening the door for the development of therapies for a wide range of genetic diseases that require stable gene insertion and expression. In parallel, we are driving forward our ex vivo programs and other in vivo programs. Our researchers are gaining further insights into our ATTR program through our ongoing NHP studies, as well as working with our collaborators at Ospedale San Raffaele (OSR) to make excellent progress in our quest to advance the next generation of engineered cell therapy.” CRISPR-mediated, Targeted Gene Insertion Data In a collaboration between Intellia and Regeneron Pharmaceuticals, Inc., researchers combined Intellia’s modular lipid nanoparticle (LNP) delivery system of CRISPR/Cas9 with a modular adeno-associated viral (AAV) insertion template to achieve supratherapeutic levels (levels higher than those required in a clinical setting) of gene expression in mice. Using Factor 9 (F9) as a model gene, the team demonstrated the first robust, efficient CRISPR-mediated targeted insertion into the liver. F9 is a gene that encodes Factor IX (FIX), a blood-clotting protein that is often missing or defective in hemophilia B patients. Using Intellia’s proprietary bi-directional template, researchers detected hybrid mAlb-hF9 transcripts in >50 percent of hepatocytes following a single dose. Circulating human FIX protein levels of >30,000 ng/mL were achieved, which are predicted to correspond to levels 40-300 times higher than those capable of preventing bleeding episodes in hemophilia B patients, when using a wildtype or hyperfunctional version of F9 (sources: George, et al, NEJM, 2017; Simioni et al, NEJM, 2009). Researchers were able to vary FIX levels by modulating either the LNP or the AAV dose, and expression levels remained stable and ongoing in all cases throughout 12 weeks of observation. This approach was repeated with Intellia’s wholly owned preclinical in vivo program in alpha-1 antitrypsin deficiency (AATD), another genetic disease of the liver associated with a mutation in the SERPINA1 gene that causes liver and lung dysfunction. Researchers used the LNP-AAV delivery combination of CRISPR/Cas9 components to insert donor template DNA encoding the SERPINA1 gene for AATD. The insertion resulted in blood protein levels in mice that corresponded to a range of SERPINA1 systemic levels required for normal lung function in humans. Today’s presentation, titled “Supra-therapeutic levels of transgene expression achieved in vivo by CRISPR/Cas9 mediated targeted gene insertion,” was made by Jonathan Finn, Ph.D., executive director, platform biology, Intellia. This presentation will be accessible through the Events and Presentations page of the Investor Relations section of Intellia’s website at www.intelliatx.com. New Non-Human Primate Data from Intellia’s ATTR Program Intellia also presented new data from non-human primate (NHP) studies in its transthyretin amyloidosis (ATTR) program further demonstrating a high correlation between liver editing and reduction of the transthyretin (TTR) protein. ATTR is a systemic, debilitating and fatal disease caused by one of approximately 136 different inherited mutations in the TTR gene. The company found that a liver editing rate of only ~35-40 percent in NHPs is needed to achieve a therapeutically meaningful reduction of TTR, specifically a TTR protein reduction of >60 percent. The data also demonstrated the transient nature of Intellia’s proprietary modular LNP delivery system, which was rapidly cleared from circulation, with all CRISPR/Cas9 components undetectable within five days of administration. Furthermore, rates of editing were durable over a six-month period without re-dosing the animals. These data included results from ongoing collaborations with researchers at Regeneron and the University of Porto in Portugal, where ATTR is endemic in certain populations. Today’s presentation, titled “Delivering on the therapeutic potential of CRISPR/Cas9: Development of an LNP-mediated genome editing therapeutic for the treatment of ATTR,” was made by Yong Chang, Ph.D., vice president, safety pharmacology, Intellia. This presentation will be accessible through the Events and Presentations page of the Investor Relations section of Intellia’s website at www.intelliatx.com. Data Update from Intellia’s Acute Myeloid Leukemia Program In a presentation titled “Hunting novel WT1-specific T cell receptors for immune gene therapy of acute myeloid leukemia,” Intellia and its research collaborator, OSR, led by Chiara Bonini, M.D., Ph.D., deputy director of the Division of Immunology, Transplantation and Infectious Diseases at San Raffaele Hospital and University, shared an update on the company’s lead ex vivo program in acute myeloid leukemia (AML). Researchers presented in vitro data showing that CRISPR/Cas9 editing resulted in over 90 percent knockout of endogenous T cell receptors (TCRs). Subsequent transduction of Wilms’ Tumor 1 (WT1)-specific transgenic TCRs led to high expression of the inserted TCR with over 95 percent purity in isolated cytotoxic T cells (CD8+ T cells). T cells were fully functional and specifically killed leukemic blast cells that expressed the WT1 antigen and HLA-A*02:01 allele. Several additional TCRs directed to multiple WT1 epitopes and human leukocyte antigen (HLA) alleles are under investigation, including undergoing in vitro and in vivo functional testing. Intellia and OSR are collaborating to develop best-in-class CRISPR-edited T cells directed to a specific epitope of WT1, a tumor-associated antigen overexpressed across a wide range of different tumor types and a known driver of leukocyte blasts in hematological cancers. Intellia’s first cell therapy tumor target is WT1 for the treatment of AML and other potential hematological malignancies, as well as for solid tumors. About Intellia Therapeutics Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases by replacing patients’ diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.
  8. dydo

    Canadian Solar (CSIQ)

    Isn't this a parent of SunPower buying CSIQ's modules? I know SPWR is off the project grid, but one would think the parent would buy still from it.
  9. A very knowledgeable author Marty has compiled details about SGMO clinical trials https://seekingalpha.com/instablog/400846-marty-chilberg/5224413-sangamo-clinical-trials-update
  10. dydo

    Genomic Index

    This is a structure of the index based on 1000 shares purchased per stock. Snapshot from today October 17th, 5 days old
  11. dydo

    Genomic Index

    This is the current index view in order of the market, based on today's prices
  12. dydo

    Genomic Index

    This is a selection of clinical trials
  13. dydo

    Genomic Index

    I am tracking genome edit and therapy companies as the next frontier for medicine and investment strategy. I have created an index with 18 companies which work with gene editing and therapies. I am planning to track them here separately and have created threads/forums for each. Below, is the recent market cap and the industry size
  14. I am actually invested in this company. Very interesting field. Very violent, as bad as solar. Could be a big award if it works.