Huntingtin

HD Immune GmbH Foundation and Publication of Latest Pre-Clinical Data

Retrieved on: 
Wednesday, January 24, 2024

HD Immune GmbH (HDI), a privately held biotechnology company developing novel antibody treatments for Huntington’s Disease, announces commencement of operations by its two founders Dr. Stefan Bartl and Dr. Lionel Wightman.

Key Points: 
  • HD Immune GmbH (HDI), a privately held biotechnology company developing novel antibody treatments for Huntington’s Disease, announces commencement of operations by its two founders Dr. Stefan Bartl and Dr. Lionel Wightman.
  • HDI has also closed a pre-seed financing round with an Austrian Business Angel and awarded an Austria Wirtschafts Service (AWS) pre-seed Grant.
  • HD Immune is developing an antibody to target the mutated protein and remove it from the body.
  • Based on these results, HD Immune intends to further advance the development of a human therapeutic antibody to reduce the symptoms of the disease in the patient.

US Neurologists Optimistic Regarding the Huntington’s Disease Treatment Pipeline, Emphasizing Halting Neurodegeneration as Leading Treatment Priority

Retrieved on: 
Wednesday, January 3, 2024

Thus, often without full awareness, patients lose motor control, experience psychiatric symptoms, and cognitive decline.

Key Points: 
  • Thus, often without full awareness, patients lose motor control, experience psychiatric symptoms, and cognitive decline.
  • A neurologist’s perspective from a recent research study suggests:
    “A Huntington’s patient is almost a combination of Alzheimer’s, tardive dyskinesia, Parkinson’s - all in one.
  • Recognizing the significant treatment unmet needs, Spherix Global Insights conducted a market landscape study, seeking insights from neurologists managing HD.
  • Additionally and unaided, neurologists also mentioned Annexon Biosciences’ ANX005, Azevan Pharmaceuticals’ SRX246, SOM Biotech’s SOM3355, and Vaccinex, Inc.’s pepinemab.

Annovis Bio Measures Novel Biomarkers in Plasma of Parkinson’s Patients

Retrieved on: 
Thursday, November 2, 2023

“We are pleasantly surprised to see a statistically significant drop in TDP43 levels in just 10 patients and to see a strong trend in GFAP and NfL.

Key Points: 
  • “We are pleasantly surprised to see a statistically significant drop in TDP43 levels in just 10 patients and to see a strong trend in GFAP and NfL.
  • To our knowledge this is the first time that a drug reduces the levels of TDP43 in humans, specifically here in PD patients,” said Dr. Maccecchini.
  • “These biomarker data not only corroborate the mechanism of actions of buntanetap, but also provide a new way to stratify patients and understand their disease pathology.
  • TDP-43 protein in plasma may index TDP-43 brain pathology in Alzheimer's disease and frontotemporal lobar degeneration.

Ionis enters agreement with Roche for two novel RNA-targeted programs for Alzheimer's disease and Huntington's disease

Retrieved on: 
Wednesday, September 27, 2023

"We are excited to expand our partnership with Roche, a global leader in developing and delivering innovative treatments to patients," said Brett P. Monia, Ph.D., Ionis' chief executive officer.

Key Points: 
  • "We are excited to expand our partnership with Roche, a global leader in developing and delivering innovative treatments to patients," said Brett P. Monia, Ph.D., Ionis' chief executive officer.
  • "With this new collaboration, we are joining forces to accelerate the discovery and development of novel therapies for people living with Alzheimer's disease and Huntington's disease globally.
  • Collaborating on these two programs enables Ionis to advance our wholly owned programs, including those in neurology, aligned with our strategic priorities."
  • IONIS-FB-LRx is currently in a Phase 2 study for GA, led by Ionis, and a Phase 3 study for IgAN led by Roche.

First Tunisian study examining age of onset for biallelic mutations in Huntington's disease

Retrieved on: 
Sunday, August 27, 2023

This Tunisian cohort study reported that the age of disease onset for patients with a homozygous mutation in the Huntingtin gene (HTT) did not differ significantly from that of heterozygous patients.

Key Points: 
  • This Tunisian cohort study reported that the age of disease onset for patients with a homozygous mutation in the Huntingtin gene (HTT) did not differ significantly from that of heterozygous patients.
  • The age of disease onset between the homozygous and heterozygous patients did not differ significantly.
  • Patients with smaller all were associated with a later age of onset, 62 years for homozygous and 74 years for heterozygous.
  • The number of CAG repeats and age of disease onset was shown to be inversely correlated for homozygous patients.

Life Edit Therapeutics to Present Data Demonstrating Therapeutic Potential of its Novel Gene Editing Platform

Retrieved on: 
Tuesday, April 25, 2023

Life Edit’s presentations underscore the ability of its gene editing platform to selectively target disease-associated genetic factors to treat life-threatening diseases.

Key Points: 
  • Life Edit’s presentations underscore the ability of its gene editing platform to selectively target disease-associated genetic factors to treat life-threatening diseases.
  • Wild-type HTT protein is purported to be physiologically critical, hence selective editing of the mutant HTT (mutHTT) protein is necessary for therapeutic benefit.
  • AAV5-delivered Life Edit gene editing systems enabling allele-specific targeting of mutant HTT demonstrated dose-response reductions in striatal mutHTT protein with clinically meaningful reductions of >50% observed.
  • Our recent technology advancements will help us to further power the Life Edit gene editing platform, support our partners, and selectively build out our pipeline of innovative therapeutic candidates."

Alchemab Therapeutics to present data profiling a novel antibody to Huntingtin Exon 1 at the CHDI Annual Huntington's Disease Therapeutics Conference

Retrieved on: 
Tuesday, April 25, 2023

CAMBRIDGE, England, April 25, 2023 /PRNewswire/ -- Alchemab Therapeutics, an antibody discovery company identifying naturally occurring antibodies from individuals resilient to disease, today announced that it will present data on a potential first-in-class disease modifying therapeutic for Huntington's Disease at the annual CHDI Huntington's Disease Therapeutics Conference in Dubrovnik, Croatia, 24-27 April 2023.

Key Points: 
  • CAMBRIDGE, England, April 25, 2023 /PRNewswire/ -- Alchemab Therapeutics, an antibody discovery company identifying naturally occurring antibodies from individuals resilient to disease, today announced that it will present data on a potential first-in-class disease modifying therapeutic for Huntington's Disease at the annual CHDI Huntington's Disease Therapeutics Conference in Dubrovnik, Croatia, 24-27 April 2023.
  • The data to be presented builds on a successful collaboration with Medicines Discovery Catapult and has been, in part, funded by two Innovate UK grants.
  • Young Kwon, Chief Executive Officer of Alchemab, said: "Huntington's Disease is a devastating neurodegenerative disorder and has proven to be very difficult to treat.
  • Presenter: Donna Finch, Head of Translational and Clinical Science, Alchemab Therapeutics
    Date and Time: Wednesday, April 26, Session III: Novel mechanisms, modalities, and candidates for HD therapeutics, 9:00am-12:20pm CEST

Alchemab Therapeutics to present data profiling a novel antibody to Huntingtin Exon 1 at the CHDI Annual Huntington's Disease Therapeutics Conference

Retrieved on: 
Tuesday, April 25, 2023

CAMBRIDGE, England, April 25, 2023 /PRNewswire/ -- Alchemab Therapeutics, an antibody discovery company identifying naturally occurring antibodies from individuals resilient to disease, today announced that it will present data on a potential first-in-class disease modifying therapeutic for Huntington's Disease at the annual CHDI Huntington's Disease Therapeutics Conference in Dubrovnik, Croatia, 24-27 April 2023.

Key Points: 
  • CAMBRIDGE, England, April 25, 2023 /PRNewswire/ -- Alchemab Therapeutics, an antibody discovery company identifying naturally occurring antibodies from individuals resilient to disease, today announced that it will present data on a potential first-in-class disease modifying therapeutic for Huntington's Disease at the annual CHDI Huntington's Disease Therapeutics Conference in Dubrovnik, Croatia, 24-27 April 2023.
  • The data to be presented builds on a successful collaboration with Medicines Discovery Catapult and has been, in part, funded by two Innovate UK grants.
  • Young Kwon, Chief Executive Officer of Alchemab, said: "Huntington's Disease is a devastating neurodegenerative disorder and has proven to be very difficult to treat.
  • Presenter: Donna Finch, Head of Translational and Clinical Science, Alchemab Therapeutics
    Date and Time: Wednesday, April 26, Session III: Novel mechanisms, modalities, and candidates for HD therapeutics, 9:00am-12:20pm CEST

uniQure Announces Third Quarter 2022 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Wednesday, November 2, 2022

LEXINGTON, Mass. and AMSTERDAM, Nov. 02, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2022 and highlighted recent progress across its business.

Key Points: 
  • and AMSTERDAM, Nov. 02, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2022 and highlighted recent progress across its business.
  • This followed the EMA validation of the marketing application authorization (MAA) in March 2022.
  • In July and August 2022, respectively, the EMA and the FDA conducted comprehensive, multi-day, pre-approval inspections of the Companys Lexington manufacturing site.
  • As of September 30, 2022, the Company had cash and cash equivalents of $440.3 million.

Annovis Bio Announces Publication of Patents Covering the Treatment of Amyloid Lateral Sclerosis, Huntington's Disease and Prion Diseases

Retrieved on: 
Tuesday, September 13, 2022

The patents cover methods of treating amyloid lateral sclerosis (ALS), Huntington's disease, and prion diseases by administering buntanetap.

Key Points: 
  • The patents cover methods of treating amyloid lateral sclerosis (ALS), Huntington's disease, and prion diseases by administering buntanetap.
  • A method of treating amyloid lateral sclerosis, a rare progressive neurodegenerative disorder, by administering buntanetap and inhibiting the synthesis of superoxide dismutase protein1 and TDP43.
  • A method of treating Huntington's disease, a rare progressive neurodegenerative disorder, by administering buntanetap and inhibiting the synthesis of Huntingtin protein.
  • For more information on Annovis Bio, please visit the Company's website www.annovisbio.com and follow us on LinkedIn and Twitter .