CUG

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy

Retrieved on: 
Friday, March 8, 2024
Pharmaceutical, Genetics, Health, Clinical Trials, Wheelchair, Myotonia, Cataract, DMPK, Wasting, Exercise, Arrowhead, Conference, DM1, RNA, Muscle weakness, Patient, CUG, Primate, MDA, RNA interference, Episcopal conference, Vaccine

Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.

Key Points: 
  • Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.
  • Pathogenesis of DM1 is driven by an expanded CUG trinucleotide repeat in the 3’-untranslated region of DMPK transcripts.
  • These abnormal transcripts cause mis-regulated splicing, known as spliceopathy, for certain messenger RNAs which are directly linked to the clinical manifestations of DM1.
  • Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

Tata Communications brings JAMVEE™ - an integrated, simplified cloud-based calling solution for global enterprises

Retrieved on: 
Monday, March 27, 2023
IOS, CUG, Knowledge, Communication, Tata Communications, API, Users' group, Human, Android, Closed user group, Windows shell, Mobile phone

ORLANDO, Fla., and MUMBAI, India, March 27, 2023 /PRNewswire/ -- Tata Communications , a global digital ecosystem enabler, today brings JAMVEE™, a cloud-based application with an integrated and simplified voice calling solution, enhancing the employee experience and productivity with easy collaboration features.

Key Points: 
  • ORLANDO, Fla., and MUMBAI, India, March 27, 2023 /PRNewswire/ -- Tata Communications , a global digital ecosystem enabler, today brings JAMVEE™, a cloud-based application with an integrated and simplified voice calling solution, enhancing the employee experience and productivity with easy collaboration features.
  • With flexible plans for voice calling and data roaming, JAMVEE™ is a cost-efficient solution for employee collaboration needs of enterprises.
  • JAMVEE™, together with Tata Communications MOVE™ eSIM solution, empowers businesses to offer the most cost-effective collaboration solution to employees travelling overseas that ensures ease of use, high-quality voice calling, and improves employee efficiency.
  • "Hybrid working is here to stay and will continue to evolve," said Mysore Madhusudhan, Executive Vice President, Collaboration and Connected Solutions, Tata Communications.

Tata Communications brings JAMVEE™ - an integrated, simplified cloud-based calling solution for global enterprises

Retrieved on: 
Monday, March 27, 2023
IOS, CUG, Knowledge, Communication, Tata Communications, API, Users' group, Human, Android, Closed user group, Windows shell, Mobile phone

ORLANDO, Fla., and MUMBAI, India, March 27, 2023 /PRNewswire/ -- Tata Communications , a global digital ecosystem enabler, today brings JAMVEE™, a cloud-based application with an integrated and simplified voice calling solution, enhancing the employee experience and productivity with easy collaboration features.

Key Points: 
  • ORLANDO, Fla., and MUMBAI, India, March 27, 2023 /PRNewswire/ -- Tata Communications , a global digital ecosystem enabler, today brings JAMVEE™, a cloud-based application with an integrated and simplified voice calling solution, enhancing the employee experience and productivity with easy collaboration features.
  • With flexible plans for voice calling and data roaming, JAMVEE™ is a cost-efficient solution for employee collaboration needs of enterprises.
  • JAMVEE™, together with Tata Communications MOVE™ eSIM solution, empowers businesses to offer the most cost-effective collaboration solution to employees travelling overseas that ensures ease of use, high-quality voice calling, and improves employee efficiency.
  • "Hybrid working is here to stay and will continue to evolve," said Mysore Madhusudhan, Executive Vice President, Collaboration and Connected Solutions, Tata Communications.

PepGen Reports Fourth Quarter and Full Year 2022 Financial Results and Recent Corporate Developments

Retrieved on: 
Thursday, March 23, 2023
PGN, Heart, GLP, Myogenesis, Duchenne muscular dystrophy, Therapy, DM1, Muscular dystrophy, RCT, Clinical trial, SAD, American Academy, Research, Cell, CUG, Research and development, Development, CTG, DMD, Patient, EDO, Physician, Episcopal conference, Administration, DMPK, MAD, NHP, HV, American Academy of Neurology, IND, Conference, Caregiver, Disease, Pharmaceutical industry, Cryptocurrency, Neurology

In December 2022, PepGen announced IND-enabling preclinical data supporting the progression of PGN-EDODM1 for the treatment of DM1 into clinical studies.

Key Points: 
  • In December 2022, PepGen announced IND-enabling preclinical data supporting the progression of PGN-EDODM1 for the treatment of DM1 into clinical studies.
  • Net loss was $14.9 million for the three months ended December 31, 2022, compared to $7.1 million for the same period in 2021.
  • Net loss was $69.1 million for the year ended December 31, 2022, compared to $27.3 million for the same period in 2021.
  • PepGen had approximately 23.7 million shares outstanding on December 31, 2022

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.

PepGen Announces IND-Enabling Preclinical Data Supporting Progression of PGN-EDODM1 into Clinical Studies

Retrieved on: 
Wednesday, December 7, 2022
PGN, Liver, Peptide, Cell, RPLP0, Muscle, Clinical trial, GLP, CNS, Kidney, Marketing, Safety, Heart, CUG, DMPK, EDO, Disability, DM1, Toxicology, Research, Disease, IND, CTG, Risk, Myotonia, Patient, Therapy, Real-time, Fine chemical, Vaccine, Pharmaceutical industry

BOSTON, Dec. 07, 2022 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurologic diseases, today announced new preclinical data supporting the progression of PGN-EDODM1, PepGen’s product candidate in development for the treatment of myotonic dystrophy type 1 (DM1), into clinical trials. PGN-EDODM1 leverages PepGen’s Enhanced Delivery Oligonucleotide (EDO) technology and consists of the Company’s proprietary cell-penetrating peptide conjugated to a steric blocking oligonucleotide cargo. This well-characterized therapeutic oligonucleotide is designed to bind to the CUG repeat expansion hairpin loops in the DMPK transcripts of people with DM1, and thus subsequently correct the downstream transcript mis-splicing events that lead to myotonia and cardiac dysfunction.

Key Points: 
  • “We are now pleased to provide an update on the safety profile of PGN-EDODM1, having completed IND-enabling preclinical studies in multiple models.
  • Notably, we observed that this product candidate was well-tolerated in acute GLP studies conducted in rodents and NHPs at what we believe are clinically relevant doses.
  • We believe that the totality of the data obtained from these preclinical and IND-enabling studies highlights a potentially favorable safety profile for PGN-EDODM1.
  • PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Pacific Telecommunications Council’s 2023 Annual Conference on Track for a Record-Breaking Event

Retrieved on: 
Thursday, December 8, 2022
Telecommunications, Satellite, Internet, Metaverse, Technology, Carriers and Services, IOT (Internet of Things), Mobile, Wireless, Cloud, Xchange, Pacific Telecommunications Council, Young professional, Conference, Annual conferences, CtrlS, DE-CIX, Schneider Electric, CoreSite, GOLD, CMI, Telecommunication, ESG, PTC, Pacific Rim, ICT, City, Pacific Ocean, Digital Realty, Global Cloud Xchange, CTG, Equinix, PDG, Ciena, Telstra, CUG, KDDI, Video game console, Colt

PTCs Annual Conference, the industrys premier telecommunications and ICT event, serves as a strategic springboard, a significant yearly kick-off event, where companies effectively initiate approximately 50 to 80% of their business transactions for the upcoming year.

Key Points: 
  • PTCs Annual Conference, the industrys premier telecommunications and ICT event, serves as a strategic springboard, a significant yearly kick-off event, where companies effectively initiate approximately 50 to 80% of their business transactions for the upcoming year.
  • In addition, the conference program boasts exceptionally high-caliber speakers participating in presentations, interviews, and moderated discussion panels.
  • With more than 330 member companies and individuals, many conference attendees find it advantageous to become a PTC Member.
  • PTCs Annual Conference, held each January in Honolulu, is the Pacific Rims premier telecommunications event and serves as the strategic springboard for the global communications industry.

Entrada Therapeutics Reports Second Quarter 2022 Financial Results

Retrieved on: 
Thursday, August 11, 2022
Entrada (Fringe), Regulation of food and dietary supplements by the U.S. Food and Drug Administration, U.S. Securities and Exchange Commission, Myotonia, HIV disease progression rates, CUG, Investor relations, Private Securities Litigation Reform Act, GLOBE, R, Duchenne muscular dystrophy, NHP, SEC, Therapeutic index, Terminology, IND, Tissue, Corporate communication, Security (finance), General Administration of Sport of China, Patient, Therapy, COVID-19, Company, Twitter, Research and development, Form 10-K, Central nervous system, G&A, Myotonic dystrophy, DM1, DMD, Degenerative disease, Clinical endpoint, LinkedIn, Conference, Biomarker, Duchenne, Administration, Food, EEV, Investigational New Drug, Medical device, Cryptocurrency, Pharmaceutical industry, Fine chemical, Tutoring

BOSTON, Aug. 11, 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 second quarter ended June 30, 2022 and highlighted recent business updates.

Key Points: 
  • We continued to make significant progress in advancing our growing pipeline of EEV therapeutic candidates during the second quarter, said Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics.
  • Research & Development (R&D) Expenses: R&D expenses for the second quarter of 2022 were $16.2 million, compared to $6.8 million for the same period in 2021.
  • General & Administrative (G&A) Expenses: G&A expenses for the second quarter of 2022 were $7.3 million, compared to $3.1 million for the same period in 2021.
  • Net Loss: Net loss for the second quarter of 2022 was $23.2 million, compared to $9.9 million for the same period of 2021.

Locanabio Presents New Preclinical Data from Myotonic Dystrophy Type 1 Program at Society for Muscle Biology Frontiers in Myogenesis Conference 2021

Retrieved on: 
Thursday, November 18, 2021
Patient, Tissue, Relaxation, Gastrointestinal tract, Cardiac muscle, Myotonic dystrophy, Conference, DM1, DMPK, Retina, Central nervous system, SAN, Therapy, Autosome, CUG, RNA splicing, Weakness, Society, PUF, DNA, Mutation, Palilalia, Incidence, Muscle weakness, RNA, CTG, Genetic disorder, Skeletal muscle, Event, Vaccine

The data were presented at the Society for Muscle Biology's Frontiers in Myogenesis Conference 2021, taking place November 15-19 in Costa Rica.

Key Points: 
  • The data were presented at the Society for Muscle Biology's Frontiers in Myogenesis Conference 2021, taking place November 15-19 in Costa Rica.
  • Myotonic dystrophy type 1 (DM1) is an autosomal dominant genetic disorder affecting skeletal muscle, cardiac muscle, the gastrointestinal tract, and the central nervous system.
  • These toxic RNA repeats lead to disease symptoms including progressive muscle wasting, weakness and myotonia (delayed relaxation of skeletal muscle), a hallmark of DM1.
  • The incidence of myotonic dystrophy is estimated at one in 8,000 individuals worldwide or approximately 40,000 people in the United States.

Design Therapeutics Announces Positive Preclinical Data Highlighting Disease-Modifying Potential of its Novel DM1 GeneTACs as a Treatment for Myotonic Dystrophy Type-1

Retrieved on: 
Wednesday, September 8, 2021

There are currently no approved treatment options, said Joo Siffert, M.D., president and chief executive officer of Design Therapeutics.

Key Points: 
  • There are currently no approved treatment options, said Joo Siffert, M.D., president and chief executive officer of Design Therapeutics.
  • Our DM1 GeneTACs are small molecules designed to address the underlying root causes of DM1 by specifically blocking transcription of the mutant DMPK gene.
  • New preclinical data demonstrated the ability of our DM1 GeneTACs to potently and selectively block expression of the mutant DMPK gene in DM1 patient cells.
  • Design is leveraging its proprietary GeneTAC (gene targeted chimera) platform to develop therapeutic candidates for inherited diseases driven by nucleotide repeat expansions, such as DM1.