HTT

Wave Life Sciences Reports First Quarter 2024 Financial Results and Provides Business Update

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torsdag, maj 9, 2024
Genetics, Liver disease, Coronary artery disease, Research, N-Acetylgalactosamine, Weight loss, Doctor of Philosophy, Webcast, SVP, DSM-IV codes, CSO, Biomarker, Drug discovery, AATD, RNA editing, Activin and inhibin, Facial muscles, Pharmacokinetics, LNP, SAD, GSK, Serum, Duchenne muscular dystrophy, HTT, Cerebrospinal fluid, MASH, Weight, Safety, Health, RNA, Obesity, Human, Therapy, HD, Central nervous system, Clinical trial, ED50, Small RNA, Stanford University, Lof, Genomics, Exon, Science, Senior, Patient, Cardiovascular disease, MBA, PD, Lipolysis, Q&A, Pharmaceutical industry

ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., May 09, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Revenue was $12.5 million for the first quarter of 2024, as compared to $12.9 million in the first quarter of 2023.
  • Net loss was $31.6 million for the first quarter of 2024, as compared to $27.4 million for the first quarter of 2023.
  • ET to review the first quarter 2024 financial results and pipeline updates.

Design Therapeutics Announces First Quarter 2024 Financial Results and Highlights Upcoming Program Milestones

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onsdag, maj 8, 2024
Patient, Design, Research, DMPK, IND, Observational study, HD, GLP, FA, DM1, HTT, Therapy, Biotechnology, G&A, Pharmaceutical industry

CARLSBAD, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced first quarter 2024 financial results along with recent business highlights and upcoming milestones.

Key Points: 
  • Starting Phase 1 Development for Fuchs Endothelial Corneal Dystrophy (FECD) in 2024; Observational Study Currently Enrolling Patients
    CARLSBAD, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced first quarter 2024 financial results along with recent business highlights and upcoming milestones.
  • Design is on track to complete GLP studies for DT-216P2 by year-end 2024 to start patient trials in 2025.
  • R&D Expenses: Research and development (R&D) expenses were $9.8 million for the quarter ended March 31, 2024.
  • G&A Expenses: General and administrative (G&A) expenses were $4.6 million for the quarter ended March 31, 2024.

Latus Launches with State-of-the-Art Gene Therapy Development Technologies, Two Lead Product Candidates, and an Initial $54 Million in Series A Financing

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torsdag, maj 2, 2024
Biotechnology, Professional Services, Health, General Health, Pharmaceutical, Other Science, Research, Genetics, Science, Clinical Trials, Finance, Doctor of Philosophy, CNS, SVP, Translational medicine, MIT, Genzyme, Cochlea, Hereditary Disease Foundation, Harvard Medical School, Toxicity, HTT, Therapy, Merck, CLN2, Hospital, Safety, Biomedical Research, Samsung Biologics, CHOP, Samsung C&T Corporation, AAV, Koch Institute, Gene expression, Central nervous system, NHP, Program management, Cell, DSM-IV codes, Patient, Gene, Society, Einstein Medical Center Philadelphia, Annual general meeting, Massachusetts General Hospital, Pharmaceutical industry

Latus Bio, Inc. (“Latus”), a biotechnology company developing novel gene therapy candidates for disorders of the central nervous system (CNS), today announced its launch and an initial close of $54 million in Series A financing.

Key Points: 
  • Latus Bio, Inc. (“Latus”), a biotechnology company developing novel gene therapy candidates for disorders of the central nervous system (CNS), today announced its launch and an initial close of $54 million in Series A financing.
  • These limitations have led to the reliance on high-dose injections that cause off-target toxicity and difficulties in scalable manufacturing.
  • The Company’s management team is composed of experienced and respected leaders in gene therapy and neuroscience.
  • Her address will showcase the Company's progress and the vast potential of its technology to solve unmet needs in CNS gene therapy.

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

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tisdag, mars 19, 2024
Research, Observational study, Cerebellar ataxia, Biotechnology, Gene expression, FXN, Disease, Webcast, Patient, G&A, DM1, Design, IND, Treatment, Clinical trial, Investigational New Drug, Therapy, Completeness, Evaluation, DMPK, FA, HD, Food, FDA, GLP, HTT, Pharmaceutical industry

R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.

Key Points: 
  • R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $4.1 million for the quarter ended December 31, 2023, and $21.1 million for the year ended December 31, 2023.
  • Net Loss: Net loss was $11.8 million for the quarter ended December 31, 2023, and $66.9 million for the year ended December 31, 2023.
  • Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $281.8 million as of December 31, 2023.

Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

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onsdag, mars 6, 2024
Therapy, Facial muscles, Pharmacology, Central nervous system, Research, GSK, MD, Liver disease, Dystrophin, Obesity, Cerebrospinal fluid, N-Acetylgalactosamine, HD, Weight loss, DMD, Duchenne muscular dystrophy, Lof, Exercise, Lipolysis, Exon, MBA, Conference, Wave, Activin and inhibin, AATD, Health, Safety, Biomarker, RNA, Greenshoe, Genetics, Pharmacokinetics, Webcast, Patient, Clinical trial, HTT, Q&A, Small RNA, Serum, Episcopal conference, Human, Coronary artery disease, Pharmaceutical industry

In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.

Key Points: 
  • In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.
  • Revenue was $29.1 million for the fourth quarter of 2023 as compared to $1.2 million in the prior year quarter.
  • Net loss was $16.3 million for the fourth quarter of 2023 as compared to $43.7 million in the prior year quarter.
  • ET to review the fourth quarter and full year 2023 financial results and pipeline updates.

Hydrothermal Model Predicts Russian Thistle Seedling Emergence

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onsdag, februari 21, 2024
Doctor of Philosophy, PNW, Farmer, Pollution, Salsola, HTT, Oregon State University, Pacific Northwest, Pressure, Risk, Agriculture

WESTMINSTER, Colo., Feb. 21, 2024 /PRNewswire-PRWeb/ -- Recently published research in the journal Weed Science highlights the first predictive model to use hydrothermal time (HTT) unit data to predict Russian thistle (Salsola tragus) seedling emergence in the Pacific Northwest (PNW). The predictive model will be especially helpful in timing efforts to control Russian thistle populations in fallow and spring-wheat no-till systems that are vulnerable to becoming herbicide resistant.

Key Points: 
  • Recently published research in the journal Weed Science highlights the first predictive model to use hydrothermal time (HTT) unit data to predict Russian thistle (Salsola tragus) seedling emergence in the Pacific Northwest (PNW).
  • WESTMINSTER, Colo., Feb. 21, 2024 /PRNewswire-PRWeb/ -- Recently published research in the journal Weed Science highlights the first predictive model to use hydrothermal time (HTT) unit data to predict Russian thistle (Salsola tragus) seedling emergence in the Pacific Northwest (PNW).
  • During the study, Dr. Oreja and collaborating researchers were able to develop and validate an HTT unit model to successfully predict Russian thistle seedling emergence patterns under PNW field conditions.
  • In addition, the model can help farmers and agronomists control Russian thistle seedling populations with fewer herbicide applications than the current, three to four standard application practice used in wheat-fallow rotations.

The macroeconomic effects of global supply chain reorientation

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lördag, februari 10, 2024
Bank, Control, Quarterly Journal of Economics, Literature, Deutsche Bundesbank, Reconstruction, COVID-19, Monetary policy, Medical classification, Aggregate, Interest, Hail, Motion, Organization, WT, Policy, Smith, Elasticity, American Economic Review, Information, CHiPs, Journal of Economic Perspectives, Reproduction, Tagliapietra, Culture, Journal of International Economics, Section 3, European Commission, Communication, B16, Shock, NTM, European Chips Act, SSC, PHT, B17, Classification, Common, Tradability, Bank of Italy, Congressional Research Service, NT, Central bank, Private, Exercise, NIU, Labour, PDF, Website, European Parliament, Terrorism, Employment, B10, SUBST, Agricultural economics, F62, RTK, Bank of England, European Central Bank, Calibration, Agriculture, Foreign policy, Semiconductor, International Monetary Fund, Research Papers in Economics, Outline, Council, Openness, Bias, Economic system, European Council, Public policy, Deutsch, Statistics, GDP, Real, American Economic Journal, Table, Journal, YT, EAGLE, Household, Grossman, Science, Conference, Journal of Comparative Economics, Horse, SSRN, TC, Consumption, REA, F13, Section 2, University, Section 5, Legislation, Money, NTD, Central Bank of Ireland, Language, Capital, University of Limerick, Intermediate, CBI, Caselli, Macroeconomics, Crowding, Technical report, B14, Tax, Civil service commission, Growth, Commission, UNCTAD, Optimism, Politics, PIM, PX, Work, Social science, JEL, Government, Automation, HTT, Quarterly Journal, Canadian International Council, ECB, XT, METRO, ELAS, Credit, Bolt, Research, European Communities, American Journal, ArXiv, Unilateralism, Lerner, Motivation, International, C6, Committee, Security (finance)

We analyse the macroeconomic

Key Points: 
    • We analyse the macroeconomic
      effects of supply chain reorientation through localisation policies, using a global dynamic
      general equilibrium model.
    • While arguments about comparative advantage, the potential forgone benefits of international specialisation and industry- and product-specific disruptions are familiar, there is less
      analysis on the macroeconomic effects of supply chain changes resulting from localisation policies.
    • The large sensitivity of the global economy to the recent supply chain shocks suggests that
      the international trade reconfiguration implied by localisation policies could also have sizable
      impacts on key macroeconomic variables such as output, employment and inflation.
    • Thus, localisation focuses on the
      goods in our model most closely related to global supply chains.
    • Retaliation also attenuates any positive effects from
      reshoring on output and implies a reduction in the volume of overall international trade.
    • This finding calls for limiting the scope of reshoring, such as by focusing on vital goods that are
      most susceptible to supply chain disruptions.
    • Either that, or the economic costs are considered a worthwhile trade-off for an increase
      in security of supply, for example.
    • While arguments about comparative advantage, the potential forgone benefits of international specialisation and industry- and product-specific disruptions are familiar, there is less
      analysis on the macroeconomic effects of supply chain changes resulting from localisation policies.
    • Recent supply chain shocks have had large effects, with disruptions in 2021 estimated
      to have reduced euro area GDP by around two percent and doubled the rate of manufacturing producer inflation (Celasun et al., 2022).
    • To analyse this issue, we simulate a (partial) reshoring of production back to Europe in
      a global dynamic general equilibrium framework.
    • Thus,
      localisation focuses on the goods in our model most closely related to global supply chains.3 We
      model reshoring through a direct change to the export goods? production-function parameters.
    • Since reshoring
      effectively shortens the supply chain, the sum of markups along the chain falls.
    • This means that imports that are at the end of the supply chain (i.e.
    • In particular, our work relates to papers examining the potential for countries to reduce
      their exposure to global supply chains.
    • (2021) demonstrate that reduced reliance on foreign inputs does not mitigate pandemicinduced contractions in labour supply.
    • (2021) find no evidence of a relationship
      between global value chain integration and macroeconomic volatility.
    • This dynamic, along with factors such as natural disasters, climate-change
      induced volatility and terrorism mean that supply chain disruptions could be a new normal
      (Grossman et al., 2021).
    • Our work contributes to the literature providing dynamic general equilibrium analyses of
      protectionist policies, in particular those using global macroeconomic models to quantify trade
      policy changes.
    • (2008) analyse the effect of a rise in protectionism in response
      to rising global trade imbalances.
    • Linde? and Pescatori (2019) find that although the macroeconomic costs of a
      trade war are substantial, a fully symmetric retaliation is the best response.
    • (2020) consider a rich input-output structure and demonstrate that closer integration amplifies
      the adverse effects of protectionist trade policies.
    • Several recent studies have also examined the economic effects of a global trade fragmentation.
    • First, we modify a dynamic general
      equilibrium model of the global economy in order to analyse the transmission of localisation
      policies.
    • This allows for a comprehensive treatment of cross-border macroeconomic interdependences and spillovers between the different regions.
    • 4

      There is, however, substantial cross-country heterogeneity in terms of impact, with small open economies
      (SOEs) reliant on global supply chains more affected.

    • ECB Working Paper Series No 2903

      7

      Second, we are able to assess both long-run effects and the transition dynamics of localisation
      policies.

    • Our model contains a detailed monetary block and captures inflation dynamics, which is a key
      concern for supply chain reorientation.
    • Overall, our paper contains a careful analysis of the key aspects of the localisation debate,
      including effects of localisation on domestic competition and efficiency.
    • Section 2 provides a brief overview of the model, the modifications to examine
      global supply chain reorientation, some key details on the calibration and a brief discussion of
      the nature of our exercise.
    • (2020) for discussions of the relative strengths and weaknesses of
      trade and macroeconomic models in assessing large economic shocks.
    • 2.1

      Supply chain reorientation

      Our analysis focuses on imported inputs used to produce goods for export, as the introduction
      of localisation policies is in response to recent disruptions to global supply chains.

    • Since reshoring
      effectively shortens the supply chain, the sum of markups along the chain falls.
    • Further to
      these effects, engagement with global firms provides an opportunity for knowledge spillovers to
      local firms (Criscuolo et al., 2017).
    • This finding calls for limiting the scope of reshoring, such as by focusing on vital goods that are
      most susceptible to supply chain disruptions.
    • (B12)

      Adjusting the share of local inputs in export goods, of course, affects prices and quantities all
      along the supply chain.

Orphan designation: Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene Treatment of Huntington’s disease, 16/08/2023 Positive

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söndag, februari 4, 2024
COMP, IRIS, European Commission, Cas9, HTT, Committee, EMA, Pharmaceutical industry

EU/3/23/2819 - orphan designation for treatment of Huntington’s disease

Key Points: 
  • EU/3/23/2819 - orphan designation for treatment of Huntington’s disease
    Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene
    OrphanHuman
    Laura Nae
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Wave Life Sciences Highlights Recent Achievements and Upcoming 2024 Milestones

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måndag, januari 8, 2024
Well, RNA, Conference, Science, Wave, Messenger, Research, AAT, Chemistry, Pizzicato, Small RNA, Lung, Weight, Health, Disease, MBA, MD, Mouse, HD, Human, Serum, GSK, Mutation, Genetics, Coronary artery disease, Obesity, Genomics, Concentration ratio, Central nervous system, Patient, Liver disease, Lof, HTT, Medicine, Therapy, Oligonucleotide, NHP, CTA, Vaccine

We expect 2024 will be an inflection year that will drive significant value for Wave, our shareholders, and most importantly, for the patients who will benefit from our research,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.

Key Points: 
  • We expect 2024 will be an inflection year that will drive significant value for Wave, our shareholders, and most importantly, for the patients who will benefit from our research,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.
  • Expected milestones: Wave plans to deliver proof-of-mechanism data in individuals with AATD in 2024.
  • Expected milestones: Wave expects to deliver data, including dystrophin protein expression from muscle biopsies at 24 weeks, in the third quarter of 2024.
  • A live webcast of this presentation will be available on the Investor Relations page of the Wave Life Sciences website at http://ir.wavelifesciences.com .

Ionis enters agreement with Roche for two novel RNA-targeted programs for Alzheimer's disease and Huntington's disease

Retrieved on: 
onsdag, september 27, 2023
Huntington's disease, HD, Learning, Partnership, Therapy, Ionis Pharmaceuticals, HTT, Systemic disease, Geographic atrophy, IONS, GA, Immunoglobulin A, RNA, Sympathetic nervous system, Huntingtin, Science, Neuroscience, IgA nephropathy, Patient, Knowledge, AD, Neurology, Alzheimer's disease, Fine chemical, Pharmaceutical industry, Ionis

"We are excited to expand our partnership with Roche, a global leader in developing and delivering innovative treatments to patients," said Brett P. Monia, Ph.D., Ionis' chief executive officer.

Key Points: 
  • "We are excited to expand our partnership with Roche, a global leader in developing and delivering innovative treatments to patients," said Brett P. Monia, Ph.D., Ionis' chief executive officer.
  • "With this new collaboration, we are joining forces to accelerate the discovery and development of novel therapies for people living with Alzheimer's disease and Huntington's disease globally.
  • Collaborating on these two programs enables Ionis to advance our wholly owned programs, including those in neurology, aligned with our strategic priorities."
  • IONIS-FB-LRx is currently in a Phase 2 study for GA, led by Ionis, and a Phase 3 study for IgAN led by Roche.