DM1

Entrada Therapeutics Reports Third Quarter 2022 Financial Results

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월요일, 11월 7, 2022
G&A, Company, Central nervous system, Investigational New Drug, Therapy, Food, DMD, Tissue, Myotonic dystrophy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, HSA, GLOBE, Form 10-K, Entrada (Fringe), Congress, Mouse, Private Securities Litigation Reform Act, Degenerative disease, IND, DM1, Duchenne, Terminology, Biomarker, General Administration of Sport of China, Clinical endpoint, COVID-19, HIV disease progression rates, Research and development, U.S. Securities and Exchange Commission, Duchenne muscular dystrophy, Myotonia, R, Therapeutic index, Security (finance), SEC, Smart contract, Patient, Administration, EEV, Pharmaceutical industry, Fine chemical

BOSTON, Nov. 07, 2022 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEV™) therapeutics as a new class of medicines, today reported financial results for the third quarter ended September 30, 2022 and highlighted recent business updates.

Key Points: 
  • We are well positioned to execute on our strategic initiatives to create value for patients and shareholders alike, said Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics.
  • Entrada expects to file its first Investigational New Drug (IND) application for ENTR-601-44 for patients with Duchenne who are amenable to exon 44 skipping in the fourth quarter of 2022.
  • Research & Development (R&D) Expenses: R&D expenses for the third quarter of 2022 were $19.0 million, compared to $10.5 million for the same period in 2021.
  • Net Loss: Net loss for the third quarter of 2022 was $25.1 million, compared to $14.4 million for the same period of 2021.

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

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목요일, 11월 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.

Dyne Therapeutics Reports Third Quarter 2022 Financial Results and Business Highlights

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목요일, 11월 3, 2022
G&A, Jude the Apostle, DMD, FDA, Investor, Adult, FSHD, University, Research, U.S. Securities and Exchange Commission, R, SEC, CA, FORCE, Food, MAD, GLOBE, Congress, University of Milan, Science, Dystrophin, Nucleic Acids Research, Duchenne muscular dystrophy, Security (finance), Patient, Muscle, Pharmacokinetics, DM1, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Degenerative disease, Private Securities Litigation Reform Act, Terminology, COVID-19, Review, National Institutes of Health Clinical Center, Hospital, Fast Track, Clinic, Nucleic acid, Therapy, Tissue, Safety, Conference, Facioscapulohumeral muscular dystrophy, Nasdaq, Form 10-Q, Credit, Credit Suisse, DYN, Center, Pharmaceutical industry, Medical imaging, Air cargo, Lease, Neurology, Dyne, Facebook

WALTHAM, Mass., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the third quarter of 2022 and business highlights.

Key Points: 
  • WALTHAM, Mass., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the third quarter of 2022 and business highlights.
  • In the third quarter of 2022 we marked incredibly important milestones for Dyne with the initiation of patient clinical trials for our two lead product candidates, DYNE-101 and DYNE-251.
  • In October 2022, Dyne announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for DYNE-251.
  • Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases.

Dyne Therapeutics to Present at Investor Conferences in November

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수요일, 11월 2, 2022
DM1, Credit, Duchenne muscular dystrophy, Conference, Dyne, GLOBE, Nasdaq, Facioscapulohumeral muscular dystrophy, DMD, Degenerative disease, Investor, Therapy, DYN, FSHD, Credit Suisse, CA, Pharmaceutical industry, Facebook

WALTHAM, Mass., Nov. 02, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc .

Key Points: 
  • WALTHAM, Mass., Nov. 02, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc .
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that management is scheduled to present at the following investor conferences in November:
    Credit Suisse 31st Annual Healthcare Conference, presentation on November 9, 2022 at 11:00 a.m. PT (2:00 p.m.
  • ET) in Rancho Palos Verdes, CA
    Jefferies London Healthcare Conference, fireside chat on November 16, 2022 at 12:20 p.m. GMT (7:20 a.m.
  • ET in New York
    Piper Sandler 34th Annual Healthcare Conference, fireside chat on November 29, 2022 at 11:00 a.m.

Avidity Biosciences to Participate in Upcoming Investor Conferences

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화요일, 11월 1, 2022
DM1, RNA, Duchenne muscular dystrophy, Conference, Nasdaq, Facioscapulohumeral muscular dystrophy, DMD, Antibody-oligonucleotide conjugate, AOCS, FSHD, SAN, Therapy, Science, Vaccine, Avidity

ET

Key Points: 
  • ET
    5th Annual Evercore ISI HealthCONx Conference on November 29 at 7:30 a.m. PT/10:30 a.m.
  • Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs).
  • Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution.
  • Avidity is headquartered inSan Diego, CA.For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter .

Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy

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월요일, 10월 31, 2022
DMD, U.S. Securities and Exchange Commission, Marketing, Safety, PMO, Diaphragm, Time, Transferrin, Clinical trial, Accelerated approval (FDA), Food, Dystrophin, Priority review, Degenerative disease, Therapy, Muscle, FSHD, Private Securities Litigation Reform Act, Heart, Second Half, Patient, SEC, Terminology, DM1, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Mortality, Security (finance), FORCE, Duchenne muscular dystrophy, Disease, Company, Form 10-Q, DYN, GLOBE, Myopathy, Nasdaq, FDA, MPH, Exon, Plasma protein binding, COVID-19, Facioscapulohumeral muscular dystrophy, Review, MAD, Fast Track, Cardiac muscle, Pharmacokinetics, Pharmaceutical industry, Dyne, Facebook

WALTHAM, Mass., Oct. 31, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping. DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial.

Key Points: 
  • A drug receiving Fast Track designation also may be eligible for Accelerated Approval and Priority Review if relevant criteria are met.
  • DYNE-251 is Dynes product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
  • Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases.
  • Dyne has a broad portfolio of programs for serious muscle diseases, including candidates for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Avidity Biosciences, Inc. - RNA

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목요일, 10월 27, 2022
Avidity, Company, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NASDAQ, AOC, Food, News, GLOBE, DM1, FDA, LLP, Pomerantz, Private investigator

NEW YORK, Oct. 27, 2022 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Avidity Biosciences, Inc.(Avidity or the Company) (NASDAQ:RNA).

Key Points: 
  • NEW YORK, Oct. 27, 2022 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Avidity Biosciences, Inc.(Avidity or the Company) (NASDAQ:RNA).
  • Such investors are advised to contact Robert S. Willoughby at [email protected] or 888-476-6529, ext.
  • The investigation concerns whether Avidity and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions.

Arthex Biotech Selected for EIC Accelerator Award of up to 14 Million Euros

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화요일, 10월 25, 2022
MicroRNA, Education, Risk, Growth, Science, Acceleration, Commission, Patient, Small Business Innovation Research, Program, DM1, Culture, Investment, Commissioner, Degenerative disease, EIC, European Innovation Council, Company, Mentorship, CEO, Innovation, Drug development, Accelerator, Clinical trial, Forward-looking statement, Toxicology, Pharmaceutical industry, Research

VALENCIA, Spain, Oct. 25, 2022 /PRNewswire/ -- Arthex Biotech S.L., a pre-clinical-stage biotechnology company pioneering the investigation of innovative microRNA modulating approaches to diseases with unmet medical needs, today announced it has been selected for an EIC Accelerator award of up to 14 million Euros.

Key Points: 
  • VALENCIA, Spain, Oct. 25, 2022 /PRNewswire/ -- Arthex Biotech S.L., a pre-clinical-stage biotechnology company pioneering the investigation of innovative microRNA modulating approaches to diseases with unmet medical needs, today announced it has been selected for an EIC Accelerator award of up to 14 million Euros.
  • From this pool, 232 were invited to a video-pitch, and from this 75 were chosen for awards, including Arthex Biotech.
  • The EIC Accelerator will provide Arthex substantial financial support with grant funding (non-dilutive) of 2.5 million for innovation development costs, as well as a direct equity investment of up to 11.5 million, managed by the EIC Fund ,for scale up and other relevant costs.
  • For more information on the EIC Accelerator Scheme, visit: https://eic.ec.europa.eu/eic-funding-opportunities/eic-accelerator_en
    For the EIC Accelerator Press release, click here: https://eic.ec.europa.eu/news/european-innovation-council-new-wave-deep-...
    For more information on Arthex Biotech, visit www.Arthexbiotech.com

Arthex Biotech Selected for EIC Accelerator Award of up to 14 Million Euros

Retrieved on: 
화요일, 10월 25, 2022
MicroRNA, Education, Risk, Growth, Science, Acceleration, Commission, Patient, Small Business Innovation Research, Program, DM1, Culture, Investment, Commissioner, Degenerative disease, EIC, European Innovation Council, Company, Mentorship, CEO, Innovation, Drug development, Accelerator, Clinical trial, Forward-looking statement, Toxicology, Pharmaceutical industry, Research

VALENCIA, Spain, Oct. 25, 2022 /PRNewswire/ -- Arthex Biotech S.L., a pre-clinical-stage biotechnology company pioneering the investigation of innovative microRNA modulating approaches to diseases with unmet medical needs, today announced it has been selected for an EIC Accelerator award of up to 14 million Euros.

Key Points: 
  • VALENCIA, Spain, Oct. 25, 2022 /PRNewswire/ -- Arthex Biotech S.L., a pre-clinical-stage biotechnology company pioneering the investigation of innovative microRNA modulating approaches to diseases with unmet medical needs, today announced it has been selected for an EIC Accelerator award of up to 14 million Euros.
  • From this pool, 232 were invited to a video-pitch, and from this 75 were chosen for awards, including Arthex Biotech.
  • The EIC Accelerator will provide Arthex substantial financial support with grant funding (non-dilutive) of 2.5 million for innovation development costs, as well as a direct equity investment of up to 11.5 million, managed by the EIC Fund ,for scale up and other relevant costs.
  • For more information on the EIC Accelerator Scheme, visit: https://eic.ec.europa.eu/eic-funding-opportunities/eic-accelerator_en
    For the EIC Accelerator Press release, click here: https://eic.ec.europa.eu/news/european-innovation-council-new-wave-deep-...
    For more information on Arthex Biotech, visit www.Arthexbiotech.com

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Avidity Biosciences, Inc. - RNA

Retrieved on: 
토요일, 10월 15, 2022
News, Pomerantz, AOC, NASDAQ, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, DM1, Company, Food, Avidity, GLOBE, FDA, LLP, Private investigator

NEW YORK, Oct. 14, 2022 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Avidity Biosciences, Inc.(Avidity or the Company) (NASDAQ:RNA).

Key Points: 
  • NEW YORK, Oct. 14, 2022 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Avidity Biosciences, Inc.(Avidity or the Company) (NASDAQ:RNA).
  • Such investors are advised to contact Robert S. Willoughby at [email protected] or 888-476-6529, ext.
  • The investigation concerns whether Avidity and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions.