Haploinsufficiency

Renovacor Announces Upcoming Presentation at Keystone Symposia’s Heart Failure: Mechanisms and Therapies Conference

Retrieved on: 
Monday, September 26, 2022
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Keystone, U.S. Securities and Exchange Commission, CO, Securities & Exchange Commission of Pakistan, COVID-19, NYSE, FDA, IND, Company, Poster, Intellectual property, DCM, Heart failure, Forward-looking statement, Patient, Quality of life, MST, Private Securities Litigation Reform Act, Heart, BAG3, Conference, RCOR, Mortality, Haploinsufficiency, Disease, Pharmaceutical industry

The conference is taking place from September 28 October 1, 2022, at the Beaver Run Resort in Breckenridge, CO.

Key Points: 
  • The conference is taking place from September 28 October 1, 2022, at the Beaver Run Resort in Breckenridge, CO.
  • The posters presenting author is Valerie Myers, Ph.D., Senior Director of Preclinical Science at Renovacor.
  • Renovacor is a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases.
  • The companys lead program in BAG3-associated dilated cardiomyopathy (DCM) uses gene transfer technology to address the monogenic cause of this severe form of heart failure.

Stoke Therapeutics Enrolls First Patient in a Natural History Study of People Living with Autosomal Dominant Optic Atrophy (ADOA)

Retrieved on: 
Thursday, August 25, 2022
Health, Biotechnology, Security (finance), ADOA, COVID-19, Asos, Central nervous system, Therapy, Severe cognitive impairment, Founder effect, Disease, Tissue, Incidence, Diagnosis, TANGO, Oregon Health & Science University School of Dentistry, U.S. Securities and Exchange Commission, Targeted molecular therapy for neuroblastoma, Stoke, Medicine, Patient, Neurology, Nasdaq, FDA, FALCON, Twitter, Oregon Health Plan, Dravet syndrome, Mutation, Haploinsufficiency, Risk, Annual report, OPA1, Private Securities Litigation Reform Act, Eye, ASO, Life, Pharmaceutical industry, Vaccine, Ophthalmology

FALCON is a two-year prospective natural history study in patients who have a confirmed diagnosis of ADOA that is caused by an OPA1 mutation.

Key Points: 
  • FALCON is a two-year prospective natural history study in patients who have a confirmed diagnosis of ADOA that is caused by an OPA1 mutation.
  • Autosomal dominant optic atrophy (ADOA) is the most common inherited optic nerve disorder.
  • STK-002 is a proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of Autosomal Dominant Optic Atrophy (ADOA).
  • Stoke is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates

Retrieved on: 
Monday, August 8, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, FDA, Dravet syndrome, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Movement, Death, Government budget balance, CSF, Asos, SUDEP, ASO, Retinal ganglion cell, Risk, IVT, Diagnosis, Technology, NHP, Cognition, Targeted molecular therapy for neuroblastoma, FALCON, Sleep, Haploinsufficiency, Research, Caregiver, Cerebrospinal fluid, TANGO, Language, Plasma, Patient, Quality of life, Therapy, Company, Contract, Prognosis, License, Seizure, RGC, Autonomic nervous system, Intellectual disability, RNA, Safety, Lists of diseases, Epilepsy, Collaboration, OLE, ADOA, Protein, Nasdaq, SCN1A, Food, EMA, Pharmacokinetics, Disease, Vaccine, Pharmaceutical industry, Cryptocurrency, Online gambling, Animal, Stoke, OPA1

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the second quarter of 2022 and provided business updates.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the second quarter of 2022 and provided business updates.
  • In the second half of 2022, the Company expects to begin enrollment in a prospective natural history study (FALCON) of people living with ADOA.
  • Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
  • Stoke is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.

Stoke Therapeutics to Present at the 2022 Jefferies Healthcare Conference

Retrieved on: 
Wednesday, June 1, 2022
Health, Genetics, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Eye, Conference, Asos, Central nervous system, Haploinsufficiency, Therapeutic Targets Database, Webcast, Dravet syndrome, Disease, TANGO, Investor, Stoke, Nasdaq, Therapy, ADOA, Twitter, Tissue, Vaccine

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that management will present at the 2022 Jefferies Healthcare Conference on Wednesday, June 8, 2022, at 4:00 p.m.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that management will present at the 2022 Jefferies Healthcare Conference on Wednesday, June 8, 2022, at 4:00 p.m.
  • Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
  • Using Stokes proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.
  • Stoke is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates

Retrieved on: 
Tuesday, May 10, 2022
Research, FDA, Genetics, Clinical Trials, Biotechnology, Pharmaceutical, Health, Optical, Science, Other Science, Epilepsy, NHP, Targeted molecular therapy for neuroblastoma, European Medicines Agency, Safety, Seizure, Caregiver, Collaboration, Quality of life, Dravet syndrome, Language, IVT, Technology, SCN1A, Annual general meeting, Risk, Association for Research in Vision and Ophthalmology, Company, Plasma, Asos, ARVO, Patient, FDA, ASO, Disease, Haploinsufficiency, Research, Therapy, EMA, Pharmacokinetics, RNA, Autonomic nervous system, Medicine, Retinal ganglion cell, Cell, Cerebrospinal fluid, Contract, Cognition, ADOA, Sleep, Gene, Association, FALCON, American Society of Gene and Cell Therapy, The Association, Movement, Protein, Nasdaq, Government budget balance, Death, TANGO, Â, OPA1, Diagnosis, License, OLE, Prognosis, Intellectual disability, Society, SUDEP, Lists of diseases, Vaccine, Pharmaceutical industry, Online gambling, Ophthalmology, Stoke

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the first quarter of 2022 and provided business updates.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the first quarter of 2022 and provided business updates.
  • We believe we are in a strong financial position with sufficient capital to support our current research and clinical development efforts into 2025.
  • The European Medicines Agency (EMA) granted orphan drug designation for STK-001 as a potential new treatment for Dravet syndrome.
  • In the second quarter of 2022, the Company expects to initiate a prospective natural history study (FALCON) of people living with ADOA.

Stoke Therapeutics Presents New In-Vivo Data That Demonstrated Dose-Related Target Engagement and OPA1 Protein Upregulation in Retinal Tissue Following Administration of STK-002

Retrieved on: 
Monday, May 2, 2022
Research, Genetics, Clinical Trials, Other Health, Biotechnology, Pharmaceutical, Health, Optical, Science, Other Science, COVID-19, U.S. Securities and Exchange Commission, Mutation, Private Securities Litigation Reform Act, Twitter, Nasdaq, Asos, Targeted molecular therapy for neuroblastoma, RNA, Company, Retinal ganglion cell, Haploinsufficiency, Stoke, Research, Eye, TANGO, ARVO, ADOA, RGC, Injection, Patient, Security (finance), Association, Incidence, Association for Research in Vision and Ophthalmology, Severe cognitive impairment, Tissue, Significance, Potency (pharmacology), Founder effect, Dravet syndrome, ASO, Disease, NMD, Life, RNA transfection, IVT, Annual general meeting, Central nervous system, Therapy, Vaccine, Pharmaceutical industry, Ophthalmology, OPA1

Stoke Therapeutics, Inc. (Nasdaq: STOK), abiotechnologycompanydedicatedtoaddressingthe underlying causeof severe diseases by upregulating protein expression with RNA-based medicines, todayannounced new preclinical data that demonstrated dose-related target engagement and increases in OPA1 protein levels in retinal tissue of non-human primates (NHPs) following intravitreal (IVT) administration of STK-002.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), abiotechnologycompanydedicatedtoaddressingthe underlying causeof severe diseases by upregulating protein expression with RNA-based medicines, todayannounced new preclinical data that demonstrated dose-related target engagement and increases in OPA1 protein levels in retinal tissue of non-human primates (NHPs) following intravitreal (IVT) administration of STK-002.
  • Target engagement and OPA1 protein increases were sustained for at least eight weeks post-injection.
  • A dose-related increase in OPA1 protein was also detected in retinal ganglion cells (RGCs) of NHPs treated with STK-002.
  • An estimated 65% to 90% of cases are caused by mutations in the OPA1 gene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation.

Modalis Therapeutics to Present Data Supporting of Development of Transformative Epigenetic Modulating Medicines for the Treatment of a Type of Muscular Dystrophy and the Other Genetic Disorders at the ASGCT Annual Meeting

Retrieved on: 
Wednesday, April 20, 2022
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The abstracts present preclinical data from the Companys Congenital Muscular Dystrophy type 1a (CMD1A) and the other rare disease programs in cardiovascular and neuroscience indications, as well as validation of our technology.

Key Points: 
  • The abstracts present preclinical data from the Companys Congenital Muscular Dystrophy type 1a (CMD1A) and the other rare disease programs in cardiovascular and neuroscience indications, as well as validation of our technology.
  • Abstracts can be accessed on the ASGCT website and the presentations will be posted on the Modalis website during the conference.
  • MDL-101 is an experimental, epigenetic modulation therapy under investigation for the treatment of Congenital Muscular Dystrophy type 1A (CMD1A).
  • Modalis Therapeutics develops precision genetic medicines using epigenetic gene modulation.

Stoke Therapeutics to Present at The Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting

Retrieved on: 
Thursday, April 7, 2022
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, ARVO, Therapy, Association for Research in Vision and Ophthalmology, Life, Haploinsufficiency, ASO, Founder effect, RNA-targeting small molecule drugs, Central nervous system, Peripheral neuropathy, Research, Incidence, TANGO, Dravet syndrome, RNA, Tissue, The Association, OPA1, Association, Technology, Annual general meeting, Twitter, Severe cognitive impairment, Nasdaq, Autosome, Gene, ADOA, Eye, Asos, Protein, Patient, Disease, Vaccine, Ophthalmology, Stoke

STK-002 is a proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of autosomal dominant optic atrophy (ADOA).

Key Points: 
  • STK-002 is a proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of autosomal dominant optic atrophy (ADOA).
  • The presentation details are as follows:
    TANGO (Targeted Augmentation of Nuclear Gene Output) is Stokes proprietary research platform.
  • In these cases, the mutated gene does not produce its share of protein, so the body does not function normally.
  • It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life.

Stoke Therapeutics to Present at the 21st Annual Needham Virtual Healthcare Conference

Retrieved on: 
Tuesday, April 5, 2022
Biotechnology, Genetics, Optical, Health, Therapy, Investor, Central nervous system, Conference, Webcast, TANGO, Dravet syndrome, Tissue, Twitter, Haploinsufficiency, Stoke, Nasdaq, Eye, ADOA, Asos, Therapeutic Targets Database, Disease, Vaccine

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that management will present at the 21st Annual Needham Virtual Healthcare Conference on Tuesday, April 12, 2022, at 8:00 a.m.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that management will present at the 21st Annual Needham Virtual Healthcare Conference on Tuesday, April 12, 2022, at 8:00 a.m.
  • Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
  • Using Stokes proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.
  • Stoke is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.

Tevard Biosciences licenses mRNA Amplifier technology from Johns Hopkins University

Retrieved on: 
Thursday, March 31, 2022
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CAMBRIDGE, Mass., March 31, 2022 /PRNewswire/ -- Tevard Biosciences, the pioneer of tRNA-based treatments for severe genetic diseases, today announced an agreement to exclusively license "mRNA Amplifier" technology from the Johns Hopkins University. The technology will help form the basis of Tevard's third platform for addressing diseases in which a mutation has caused a reduction in or loss of a vital protein.

Key Points: 
  • New platform provides additional avenue for treating severe genetic diseases, joins Tevard's tRNA-based platforms in broadly applicable suite of technologies
    CAMBRIDGE, Mass., March 31, 2022 /PRNewswire/ -- Tevard Biosciences , the pioneer of tRNA-based treatments for severe genetic diseases, today announced an agreement to exclusively license "mRNA Amplifier" technology from the Johns Hopkins University.
  • The technology will help form the basis of Tevard's third platform for addressing diseases in which a mutation has caused a reduction in or loss of a vital protein.
  • While the company's first two platforms employ RNA-based molecules called tRNAs, the new mRNA Amplifier platform is based around specially engineered proteins.
  • Tevard Biosciences is pioneering mRNA-modulating therapies to cure a broad range of genetic diseases.