FXN

Prime Medicine Announces Recent Progress and Highlights 2023 Strategic Priorities

Retrieved on: 
Monday, January 9, 2023
Disease, FRDA, Genome, Cystic fibrosis transmembrane conductance regulator, Swelling, CGD, Safety data sheet, Respiratory tract, GAA, Tissue, Cystic fibrosis, CD34, CMC, AAV, Heart, Failure rate, CFTR, Mitochondrion, Culture, DNA, LNP, Safety, Mutation, IPSC, Gene editing, Central nervous system, Hepatocyte, Patient, Degenerative disease, CF, Bronchiole, Neuron, PCSK9, Ataxia, Liver, Human, FXN, Inflammation, Chronic granulomatous disease, Eye, Investment, Prime editing, Vaccine, Pharmaceutical industry, Medical device, IND, WD, Frataxin

CAMBRIDGE, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today provided an update on recent advancements of its Prime Editing technology and progress across its initial pipeline of eighteen programs, and outlined its strategic priorities and expected milestones for 2023.

Key Points: 
  • Today, Prime Medicine announced new preclinical data demonstrating that Prime Editing-mediated removal of pathological repeats in vitro results in correction of hypermethylation at the FXN gene, restoring genetic function back to wild-type levels.
  • Off-target Safety Data: Prime Medicine is progressing a comprehensive suite of assays to evaluate the potential off-target activity of its Prime Editors.
  • Prime Medicine is using PCSK9 as a model system for developing its modular LNP delivery platform to the liver.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Nominate first development candidate for CGD in 1Q 2023.

EQS-News: Gerresheimer AG: Gerresheimer to expand significantly in High Value Solutions and further accelerates its sustainable profitable growth

Retrieved on: 
Sunday, December 18, 2022
Contract manufacturer, Capital market, Medical device, Syringe, Incidence, FXN, Sustainability, Book, Medicine, EBITDA, Health, Administration, Environment, Diabetes, Pharma, EZ, EUR, Cosmetics, USD, Growth, Obesity, Digitalization, Investment, FY, Patient, Pharmaceutical industry

With new orders for High Value Solutions and Medical Devices, we have further reinforced our leading positions in attractive markets.

Key Points: 
  • With new orders for High Value Solutions and Medical Devices, we have further reinforced our leading positions in attractive markets.
  • Gerresheimer’s expertise and strong track record are enabling the Company to accelerate sustainable, profitable growth by consistently increasing the revenue share of biologics solutions and injectables.
  • In particular “ready-to-fill” solutions in syringes and vials are driving growth in High Value Solutions while Medical Devices such as auto injection devices and pens are boosting growth in the area of Contract Manufacturing.
  • The order book for Medical Devices is growing significantly, and Gerresheimer recently won two large contracts that will consistently increase its profitable organic growth.

Design Therapeutics Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Friedreich Ataxia and Portfolio Progress

Retrieved on: 
Wednesday, December 7, 2022
RNA, Degenerative disease, Friedreich's ataxia, Security (finance), MAD, Webcast, Cerebellar ataxia, Research, CTG, Cohort, Plasma, Liver function tests, Clinical trial, Safety, CUG, Treatment, Female, Neurology, SAD, Vital signs, Therapy, Myotonic dystrophy, Investigational New Drug, FXN, UCLA, RNA transfection, Disease, Mutation, Caregiver, Division, Patient, GAA, Friedreich's Ataxia Research Alliance, PD, Nature, AES, AE, Pharmacokinetics, EST, DM1, IND, PK, Risk, Lead, Pharmaceutical industry, Vaccine, Mineral wool, FA, Design, Fuchs

CARLSBAD, Calif., Dec. 07, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today reported progress across its portfolio of novel GeneTAC™ small molecules. Today’s updates include initial results on DT-216 from the company’s single-ascending dose (SAD) Phase 1 clinical trial in patients with Friedreich ataxia (FA). The results show that DT-216 was generally well-tolerated and able to overcome the frataxin (FXN) transcription impairment that causes FA, with a greater than two-fold increase in FXN mRNA in the cohort with the highest response. These data support the continued advancement of DT-216 in the ongoing multiple-ascending dose (MAD) Phase 1 trial and the anticipated Phase 2 clinical trial in FA patients, which is on track to begin in 2023.

Key Points: 
  • Today’s updates include initial results on DT-216 from the company’s single-ascending dose (SAD) Phase 1 clinical trial in patients with Friedreich ataxia (FA).
  • The primary and secondary study objectives were to evaluate safety and tolerability, and pharmacokinetics (PK) of DT-216 in FA patients.
  • Patients in Cohorts 1-4 were dosed with a single bolus dose of DT-216 at increasing levels from 25 mg to 200 mg.
  • Design plans to dose at least three cohorts and report data from the MAD trial in mid-2023.

Nu Holdings Ltd. Reports Third Quarter 2022 Financial Results

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Monday, November 14, 2022
Professional Services, Payments, Technology, Mobile, Wireless, Finance, Fintech, Result, NPL, Analysis, Asset quality, SME, International, FX, Prospectus, US, NYSE, Conference, International expansion of Netflix, Personal, NU, Average cost method, Acceleration, Financial condition report, Security (finance), Holding, Delinquent, Financial result, Annual report, Yoy, Growth, FXN, International Financial Reporting Standards, Risk, Webcast, Central Bank of Brazil, Video game, Drug, Bank, Real estate, Risk management, Online shopping, Audit

Nu Holdings Ltd. (NYSE: NU | B3: NUBR33), (Nu or the Company), one of the worlds largest digital financial services platforms, released today its Third Quarter 2022 financial results.

Key Points: 
  • Nu Holdings Ltd. (NYSE: NU | B3: NUBR33), (Nu or the Company), one of the worlds largest digital financial services platforms, released today its Third Quarter 2022 financial results.
  • Financial results are expressed in U.S. dollars and are presented in accordance with International Financial Reporting Standards (IFRS).
  • Below are Q322 performance highlights of Nu Holdings Ltd.:
    Customer growth: Nu added 5.1 million customers in the quarter reaching a total of 70.4 million customers in Brazil, Mexico and Colombia.
  • Nu has become the primary banking relationship for over 55% of the monthly active customers that have been with Nu for over a year.

Design Therapeutics Highlights Pipeline Progress and Upcoming Milestones and Reports Third Quarter 2022 Financial Results

Retrieved on: 
Thursday, November 3, 2022
G&A, Private Securities Litigation Reform Act, Science, Therapy, International Congress on Tuberculosis, MAD, Industry, GLOBE, Severe cognitive impairment, Degenerative disease, D, Adult, Safety, IND, Pharmacokinetics, Mutation, FA, Nasdaq, Patient, Gene expression, Nature, Program, Friedreich's ataxia, U.S. Securities and Exchange Commission, Clinical trial, ICAR, GAA, FXN, Risk, R, Fuchs, Disease, Security (finance), SEC, SAD, Treatment, Drug, DM1, Pharmaceutical industry, Vaccine, Design, Research

CARLSBAD, Calif., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted recent progress and anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported third quarter 2022 financial results.

Key Points: 
  • In addition, we recently initiated dosing in the multiple-ascending dose trial, keeping us on-track to complete Phase 1 mid next year.
  • With a solid cash position, expert team and strong science behind us, Im confident in our ability to execute our milestones.
  • Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC gene targeted chimera small molecules.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

Design Therapeutics to Present Preclinical Data Highlighting the Potential of its GeneTAC™ Small Molecule, DT-216, for the Treatment of Friedreich Ataxia at ICAR 2022

Retrieved on: 
Monday, October 17, 2022
Gene expression, FA, U.S. Securities and Exchange Commission, Cardiomyopathy, Arrhythmia, Disease, Therapy, SEC, Risk, IND, Primate, FXN, Patient, ICAR, GAA, RATS, International Congress on Tuberculosis, Research, Program, Investigational New Drug, Security (finance), Nasdaq, Private Securities Litigation Reform Act, Neuron, Mutation, Industry, Pharmacokinetics, Nature, Adult, Diabetes, Safety, CNS, GLOBE, Degenerative disease, Clinical trial, Heart, Friedreich's ataxia, Pharmaceutical industry, Vaccine, Nikolaus Friedreich, Design

The data were included in the companys Investigational New Drug (IND) application for DT-216, which is currently being evaluated in a Phase 1 clinical trial.

Key Points: 
  • The data were included in the companys Investigational New Drug (IND) application for DT-216, which is currently being evaluated in a Phase 1 clinical trial.
  • DT-216 is a GeneTAC small molecule designed to specifically target the GAA repeat expansion mutation and restore endogenous FXN transcription.
  • The preclinical data support the potential for DT-216 to restore FXN gene expression, improve mitochondrial function and address the root cause of FA.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

EQS-News: Gerresheimer AG: Gerresheimer reports double digit growth in revenue and earnings for the third quarter

Retrieved on: 
Wednesday, October 12, 2022
Administration, Vial, Government, Heart, Growth, FXN, Glass, RTF, GM High Value engine, Investment, EUR, Partnership, Pharma, Device, Solution, EPS, CEO, Plastic, EBITDA, Environment, Cosmetics, Engineering, Renewable energy, Fine chemical

Revenue rose organically by 17.4% in the third quarter, reflecting strong customer demand, leading market positions and the ongoing success of its High Value Solutions.

Key Points: 
  • Revenue rose organically by 17.4% in the third quarter, reflecting strong customer demand, leading market positions and the ongoing success of its High Value Solutions.
  • "Gerresheimer continues on its profitable growth path across all our divisions, business segments and regions reflecting our strong market positions.
  • In the third quarter of its financial year 2022, Gerresheimer generated revenues of EUR 473m, representing organic growth of 17.4%.
  • Gerresheimer reaffirms its guidance for the financial year 2022 for organic revenue growth of at least 10% and high single-digit organic growth in adjusted EBITDA and adjusted EPS.

Nu Holdings Ltd. Reports Second Quarter 2022 Financial Results

Retrieved on: 
Monday, August 15, 2022
Mobile, Wireless, Technology, Insurance, Payments, Finance, Fintech, Banking, Professional Services, Software, Internet, Crypto, U.S. Securities and Exchange Commission, Securities Act of 1933, Asset quality, International Financial Reporting Standards, SME, Investor relations, NYSE, CEO, Population, Technology, Credit card, Growth, Prospectus, Debt-to-GDP ratio, Security (finance), Annual report, Analysis, Yoy, NPS, International, Company, Mission statement, Financial result, NU, PIX, IFRS, International expansion of Netflix, Financial condition report, FXN, Webcast, Holding, Time, Conference, FX, Result, Adult, Video game, Bank, Real estate, Risk management, Online shopping, Audit, Insurance

Nu Holdings Ltd. (NYSE: NU | B3: NUBR33), (Nu or the Company), one of the worlds largest digital banking platforms, released today its Second Quarter 2022 financial results.

Key Points: 
  • Nu Holdings Ltd. (NYSE: NU | B3: NUBR33), (Nu or the Company), one of the worlds largest digital banking platforms, released today its Second Quarter 2022 financial results.
  • Financial results are expressed in U.S. dollars and are presented in accordance with International Financial Reporting Standards (IFRS).
  • Below are Q222 performance highlights of Nu Holdings Ltd.:
    Customer growth: Nu added 5.7 million customers in the quarter reaching a total of 65.3 million customers in Brazil, Mexico and Colombia.
  • Nu has become the primary bank for over 55% of the monthly active customers that have been with Nu for over a year.

Design Therapeutics Highlights Upcoming Milestones and Reports Second Quarter 2022 Financial Results

Retrieved on: 
Monday, August 8, 2022
U.S. Securities and Exchange Commission, Association, Risk, FA, Industry, GLOBE, R, Drug, Private Securities Litigation Reform Act, Gene expression, SEC, Clinical trial, Security (finance), Research, Gene editing, Patient, Therapy, Friedreich's ataxia, D, Program, Safety, RNA, Degenerative disease, G&A, Nature, Severe cognitive impairment, Lists of diseases, Biomedical Advanced Research and Development Authority, FXN, Mutation, Association for Research in Vision and Ophthalmology, DM1, Second Half, Nasdaq, GAA, ARVO, Adult, Pharmacokinetics, Disease, Pharmaceutical industry, Design, Ophthalmology, Fuchs

CARLSBAD, Calif., Aug. 08, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported second quarter 2022 financial results.

Key Points: 
  • CARLSBAD, Calif., Aug. 08, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC small molecules and reported second quarter 2022 financial results.
  • With a financial runway to support our current multi-year operating plan, we continue to be well-positioned to execute our milestones.
  • Design expects to initiate dosing of DT-216 in the multiple-ascending dose portion of the Phase 1 study in the second half of 2022.
  • The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting in May 2022.

Larimar Therapeutics Announces Presentation at the Upcoming Gordon Research Conference on Neurobiology of Brain Disorders

Retrieved on: 
Thursday, July 28, 2022
Gene expression, GLOBE, FXN, Discovery, Bioinformatics discovery of non-coding RNAs, Therapy, Conference, Expression, Friedreich's ataxia, Neuroscience, Neurodegeneration, Nasdaq, Poster, LRMR, Vaccine

BALA CYNWYD, Pa., July 28, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced that preclinical studies evaluating CTI-1601s effects on gene expression and neurodegeneration will be featured in a poster at the upcoming Gordon Research Conference on Neurobiology of Brain Disorders .

Key Points: 
  • BALA CYNWYD, Pa., July 28, 2022 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced that preclinical studies evaluating CTI-1601s effects on gene expression and neurodegeneration will be featured in a poster at the upcoming Gordon Research Conference on Neurobiology of Brain Disorders .
  • The conference will take place August 7 12, 2022 at the Rey Don Jaime Grand Hotel in Castelldefels, Spain.
  • The posters presenting author is David Bettoun, Ph.D., Vice President of Discovery and Non-clinical R&D, Larimar Therapeutics.
  • Larimar Therapeutics, Inc.(Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases.