Familial amyloid polyneuropathy

Intellia Presents Updated Interim Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis at the American Heart Association Scientific Sessions 2022

Retrieved on: 
Saturday, November 5, 2022
American Heart Association, Private Securities Litigation Reform Act, Form 10-K, Adult, Intellia Therapeutics, NYHA, Part Two, U.S. Securities and Exchange Commission, Clinical trial, TTR, NTLA, Form, CRISPR, Cardiomyopathy, SEC, Polyneuropathy, Heart failure, Protein, Regeneron Pharmaceuticals, GLOBE, Amyloid, Cas9, Familial amyloid polyneuropathy, Security (finance), Degenerative disease, Nerve Damage, Patient, Cancer, Nobel Prize, Human, Science, Heart, Cardiology, Amyloidosis, Physician, ATTR, Genome, Observation, AHA, Annual report, Therapy, III, Serum, Tissue, Safety, New York Heart Association Functional Classification, NASDAQ, Risk, Liver, Pharmaceutical industry, Vaccine

Results were presented in a Late-Breaking Science oral presentation at the American Heart Association (AHA) Scientific Sessions 2022, held November 5 7 in Chicago, Illinois.

Key Points: 
  • Results were presented in a Late-Breaking Science oral presentation at the American Heart Association (AHA) Scientific Sessions 2022, held November 5 7 in Chicago, Illinois.
  • The interim data from the dose-escalation portion of the Phase 1 study include 12 adult patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA) Class I III heart failure.
  • These data highlight NTLA-2001s potential as a one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein in people with ATTR-CM.
  • We believe a single dose of NTLA-2001 has the potential to halt and potentially reverse this life-threatening disease.

SOM Biotech signs letter of intent with Megapharm to commercialize SOM3355 in Israel and the Palestinian Authority

Retrieved on: 
Wednesday, December 15, 2021
Data mining, Company, Huntington's disease, SOM Biotech, Amyloidosis, Parkinsonism, Toxicity, Growth, ENISA, CNS, Glioblastoma, PRO, Time, Tardive dyskinesia, Nib Health Funds, Drug discovery, Research, Marketing, Familial amyloid polyneuropathy, Suicide, Disease, VMAT2, CDTI, SOM, Depression, Technology, Pharmaceutical industry, Science, Phenylketonuria, Industry, Hematology

The Company has received partial funding for a number of its programs from the Spanish Ministries of Science and Innovation (CDTI) and Industry (ENISA).

Key Points: 
  • The Company has received partial funding for a number of its programs from the Spanish Ministries of Science and Innovation (CDTI) and Industry (ENISA).
  • SOM3355 is potentially the only safe VMAT2(-) not related to serious adverse events such as depression, suicide, parkinsonism, or neuroleptic syndrome.
  • Megapharm provides its partners with a full set of commercial capabilities, including registration, market access and sales and marketing.
  • Megapharm has demonstrated dynamic sales growth by developing a strong company presence and expertise in selected therapeutic areas (i.e.

SOM Biotech granted Orphan Drug Designation by the FDA for SOM3355

Retrieved on: 
Monday, October 18, 2021
SOM Biotech, SOM, Bevantolol, Huntington's disease, Lists of diseases, Chorea, Hypertension, Central nervous system tumor, Marketing, Glioblastoma, Amyloidosis, FDA, Movement, Familial amyloid polyneuropathy, Patient, Tardive dyskinesia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, VMAT2, Food, ODD, Company, HD, Orphan, Central nervous system, ENISA, Research, Drug discovery, CDTI, Pharmaceutical industry

SOM Biotech (SOM or the Company), a clinical-stage drug discovery and development company focused on orphan diseases of the Central Nervous System, today announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SOM3355, currently in clinical development for the treatment of chorea movements in Huntingtons Disease (HD).

Key Points: 
  • SOM Biotech (SOM or the Company), a clinical-stage drug discovery and development company focused on orphan diseases of the Central Nervous System, today announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SOM3355, currently in clinical development for the treatment of chorea movements in Huntingtons Disease (HD).
  • Receiving ODD may qualify SOM Biotech for a seven year period of US marketing exclusivity upon approval of SOM3355.
  • Ral Insa, Chief Executive Officer of SOM Biotech highlights: Receiving Orphan Drug Designation for SOM3355 is a significant step forward for Huntingtons Disease patients and confirms the potential of our AI-powered approach to accelerate drug discovery and development.
  • About SOM Biotech: SOM Biotech (www.sombiotech.com) established in 2009 is a biopharmaceutical company based in Barcelona, Spain.

SOM Biotech presents positive Phase 2a data with SOM3355 at the 34th ECNP Congress 2021

Retrieved on: 
Monday, October 4, 2021
Vomiting, Parkinsonism, Somnolence, Huntington's disease, SOM Biotech, Central nervous system, ECNP, Congress, Central nervous system tumor, Fatigue, Tardive dyskinesia, Depression, Effectiveness, Patient, Amyloidosis, Tetrabenazine, Glioblastoma, Nausea, Bevantolol, Chorea, Toxicity, Data mining, SOM, Suicide, CEO, Lists of diseases, Company, Risk, Familial amyloid polyneuropathy, TMC, HD, Technology, Research, Brain, Drug discovery, BID, Safety, Movement, Time, Headache, VMAT2, Pharmaceutical industry, Fine chemical

The molecules chemical structurediffers considerablyfrom Xenazine and SOM3355 is thought to reduce chorea by inhibiting VMAT2 by a different mechanism.

Key Points: 
  • The molecules chemical structurediffers considerablyfrom Xenazine and SOM3355 is thought to reduce chorea by inhibiting VMAT2 by a different mechanism.
  • The Phase 2a study was a double-blind, randomized, placebo-controlled study designed to assess the efficacy and safety of SOM3355 in HD patients with chorea.
  • About SOM Biotech: SOM Biotech (www.sombiotech.com) established in 2009 is a biopharmaceutical company based in Barcelona, Spain.
  • The main strength of the technology is its high success rate with two of the Companys programs already achieving positive Phase 2a results.

Prothena and Novo Nordisk Announce Acquisition Agreement for Prothena’s ATTR Amyloidosis Programme

Retrieved on: 
Monday, July 12, 2021
Amyloidosis, Branches of biology, Proteins, Anatomy, Amyloid, Novo Nordisk, Transthyretin, Familial amyloid polyneuropathy, Organ-limited amyloidosis, Cardiac amyloidosis

PRX004 is a phase 2 ready anti-amyloid immunotherapy designed to deplete the amyloid deposits that are associated with the disease pathology of ATTR amyloidosis.

Key Points: 
  • PRX004 is a phase 2 ready anti-amyloid immunotherapy designed to deplete the amyloid deposits that are associated with the disease pathology of ATTR amyloidosis.
  • Novo Nordisk will initially focus on the clinical development of PRX004 in ATTR cardiomyopathy - an underdiagnosed and potentially fatal form of ATTR amyloidosis characterised by build-up of amyloid deposits in cardiac tissue.
  • Under the terms of the definitive purchase agreement, Novo Nordisk acquires Prothenas wholly-owned subsidiary and gains full worldwide rights to the intellectual property and related rights of Prothenas ATTR amyloidosis business and pipeline.
  • This acquisition is a testament to Prothenas pioneering work in ATTR amyloidosis and Novo Nordisks dedication to advancing new disease-modifying therapies for the benefit of people with cardiovascular diseases which are the worlds leading cause of death.

Alnylam Announces U.S. Food and Drug Administration Acceptance of New Drug Application for Investigational Vutrisiran for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis

Retrieved on: 
Thursday, June 24, 2021
Health, FDA, Genetics, Other Health, Clinical trials, Pharmaceutical, Biotechnology, Amyloidosis, Branches of biology, Organ systems, Nervous system, Neurological disorders, Protein folding, Familial amyloid polyneuropathy, Orphan drug, Peripheral neuropathy, Polyneuropathy, Wild-type transthyretin amyloid, PTC Therapeutics, Vutrisiran, the HELIOS-A Phase 3 Study, hATTR Amyloidosis, RNAi, Alnylam Pharmaceuticals, VUTRISIRAN, THE HELIOS-A PHASE 3 STUDY, HATTR AMYLOIDOSIS, RNAI, ALNYLAM PHARMACEUTICALS

If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.

Key Points: 
  • If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.
  • Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis.
  • Vutrisiran has also been granted a Fast-Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults.
  • Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis.

Ionis' antisense therapies in ATTR amyloidosis to be featured at 2021 PNS Annual Meeting

Retrieved on: 
Thursday, June 10, 2021
Branches of biology, Biology, Therapeutic gene modulation, Antisense RNA, Life sciences, Spinal muscular atrophy, Huntington's disease, Ionis Pharmaceuticals, Ionis, Antisense therapy, Familial amyloid polyneuropathy, Amyloidosis

CARLSBAD, Calif., June 10, 2021 /PRNewswire/ --Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies,today announced that multiple presentations highlighting advances in its ATTR amyloidosis programs will be featured during the 2021 Peripheral Nerve Society (PNS) Annual Meeting to be held virtually on June 12-13, and 25-27, 2021.

Key Points: 
  • CARLSBAD, Calif., June 10, 2021 /PRNewswire/ --Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies,today announced that multiple presentations highlighting advances in its ATTR amyloidosis programs will be featured during the 2021 Peripheral Nerve Society (PNS) Annual Meeting to be held virtually on June 12-13, and 25-27, 2021.
  • Ionis' participation highlights the advancements it's made in developing novel antisense therapies for ATTR and underscores Ionis' commitment to this patient community.
  • A founding member of Ionis, Dr. Bennett is responsible for continuing to advance antisense technology and expand Ionis' drug discovery platform.
  • He will discuss the therapeutic potential of antisense therapies to address an unprecedented range of neurological diseases, with a focus on TTR amyloidosis.

Prothena to Participate in JMP Securities Life Sciences Conference on June 16

Retrieved on: 
Thursday, June 10, 2021
Amyloidosis, Branches of biology, Proteins, Biology, Amyloid, Familial amyloid polyneuropathy, Transthyretin, Alzheimer's disease, AL amyloidosis

Following the live presentation, a replay of the webcast will be available on the Company's website for at least 90 days following the presentation date.

Key Points: 
  • Following the live presentation, a replay of the webcast will be available on the Company's website for at least 90 days following the presentation date.
  • Prothena Corporation plc is a late-stage clinical company with a robust pipeline of novel investigational therapeutics, built on protein dysregulation expertise, with the potential to change the course of devastating rare peripheral amyloid and neurodegenerative diseases.
  • Prothenas pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including AL amyloidosis, ATTR amyloidosis, Alzheimers disease, Parkinsons disease and a number of other neurodegenerative diseases.
  • For more information, please visit the Companys website at www.prothena.com and follow the Company on Twitter @ProthenaCorp.

Alnylam Announces New Data from ATTR Amyloidosis Programs at the Peripheral Nerve Society’s 2021 Annual Meeting

Retrieved on: 
Monday, June 7, 2021
Research, Surgery, Clinical trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Amyloidosis, Branches of biology, Health sciences, Health, Alnylam Pharmaceuticals, Familial amyloid polyneuropathy, Patisiran, Subcutaneous injection, Amyloid, ONPATTRO® (Patisiran), Vutrisiran, the HELIOS-A Phase 3 Study, hATTR Amyloidosis, RNAi, Alnylam Pharmaceuticals, ONPATTRO® (PATISIRAN), VUTRISIRAN, THE HELIOS-A PHASE 3 STUDY, HATTR AMYLOIDOSIS, RNAI, ALNYLAM PHARMACEUTICALS

These data were presented as posters during the 2021 Peripheral Nerve Societys Annual Meeting, and the HELIOS-A results will also be featured during the Richard A.C. Hughes - Clinical Science Highlights Presentation on Sunday, June 27th.

Key Points: 
  • These data were presented as posters during the 2021 Peripheral Nerve Societys Annual Meeting, and the HELIOS-A results will also be featured during the Richard A.C. Hughes - Clinical Science Highlights Presentation on Sunday, June 27th.
  • Patisiran also demonstrated an encouraging safety and tolerability profile after 12 months of treatment and there were no drug-related study discontinuations or deaths.
  • Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis.
  • Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

Intellia Therapeutics to Present Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis at the 2021 Peripheral Nerve Society Annual Meeting

Retrieved on: 
Friday, June 4, 2021
Amyloidosis, Branches of biology, Proteins, Anatomy, Neurological disorders, Protein folding, Histopathology, Structural proteins, Familial amyloid polyneuropathy, Transthyretin, NTLA, Amyloid

The presentation will include interim data from the ongoing dose-escalation portion of Intellias Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN).

Key Points: 
  • The presentation will include interim data from the ongoing dose-escalation portion of Intellias Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN).
  • Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader population of people with ATTR amyloidosis, including those with cardiomyopathy.
  • Intellia will host a live webcast on Monday, June 28, 2021 at 8:00 a.m. E.T.
  • Intellias global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).