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News and Press Releases

AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)

AB-1009 program advances to Phase 1/Phase 2; clinical trial initiated in the United States Therapy granted United States Food and Drug Administration (FDA) Fast Track and Orphan Drug designations Pompe...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: January 8, 2026

AskBio
info@askbio.com
20 T.W. Alexander Drive Suite, 110 Research Triangle Park, NC 27709

MapLight Therapeutics Receives Fast Track Designation for ML-007C-MA for Alzheimer’s Disease Psychosis

5 January 2026 -- California and Massachusetts, US -- MapLight Therapeutics, Inc today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ML-007C-MA, an...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: January 5, 2026

MapLight Therapeutics
info@maplightrx.com
800 Chesapeake Drive, Redwood City, California 9406

FDA Clears NUZ-001 for Entry into HEALEY ALS Platform Trial

FDA has completed their review of the Neurizon NUZ-001 regimen to the HEALEY ALS Platform Trial Master Protocol This marks the official entry of NUZ-001 as Regimen I in the...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: December 11, 2025

Neurizon Therapeutics
enquiries@neurizon.com
Level 4, 96-100 Albert Road South Melbourne, VIC 3205 Australia

Xlife Sciences AG Delivers Breakthrough in Early Alzheimer’s Detection, Unlocking Major Market Opportunity

1 December 2025 -- Zurich, Switzerland -- Xlife Sciences AG, together with saniva diagnostics GmbH, today announced a landmark achievement: NeuroMex is the world’s first certified and clinically validated medical device for...

Category: Biotechnology, Clinical Trials, Other, Pharmaceutical
Posted: December 1, 2025

Xlife Sciences
info@xlifesciences.ch
Talacker 35 8001 Zürich Switzerland

Sanofi and Regeneron’s Dupixent approved as the first targeted medicine in the EU in over a decade for chronic spontaneous urticaria

Sanofi and Regeneron’s Dupixent approved as the first targeted medicine in the EU in over a decade for chronic spontaneous urticaria Approval based on phase 3 studies showing Dupixent significantly...

Category: Drug Discovery, Other, Pharmaceutical
Posted: November 25, 2025

Regeneron
ukinfo@regeneron.com
The Charter Building, Vine Street, Uxbridge, Middlesex, United Kingdom, UB8 1JG

Annovis Announces FDA Meeting to Discuss Parkinson’s Disease Dementia Program; Reaffirms FDA Alignment on Pivotal Phase 3 Alzheimer’s Disease Study

18 November 2025 -- Pennsylvania, US -- Annovis Bio, Inc, a late-stage clinical drug platform company pioneering transformative therapies for neurodegenerative diseases such as Alzheimer's disease (AD) and Parkinson's disease...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: November 18, 2025

Annovis
info@annovisbio.com
101 Lindenwood Drive, Suite 225 Malvern, PA 19355

ONWARD Medical Receives FDA 510(k) Clearance Expanding ARC-EX System Indication for Home Use

US FDA clearance now allows use of the ARC-EX System both in clinics and homes ARC-EX is the first and only FDA-cleared technology demonstrated to improve hand strength and sensation...

Category: Biotechnology, Other, Pharmaceutical
Posted: November 17, 2025

ONWARD Medical
investors@onwd.com
Schimmelt 2. 5656 AE, Eindhoven, the Netherlands

Heidelberg Pharma’s Lead ADC Candidate HDP-101 Granted Fast Track Designation by US FDA for the Treatment of Multiple Myeloma

Recognizes the potential of HDP-101 to address a serious or life-threatening condition with high unmet medical needs Enables more frequent engagement with FDA and eligibility for rolling review to support...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: October 23, 2025

Heidelberg Pharma
info@hdpharma.com
Gregor-Mendel-Straße 22 68526 Ladenburg Deutschland

SOM Biotech secures clear registrational path for SOM3355 in Huntington’s disease after FDA End‑of‑Phase 2 Meeting

20 October 2025 -- Barcelona, Spain -- SOM Biotech, a clinical-stage company dedicated to the discovery and development of innovative therapies for rare central nervous system disorders, announces today that it...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: October 20, 2025

SOM Biotech
Baldiri Reixac, 4 (08028) Barcelona, Spain

Data from GNT-018-IDES trial supports feasibility of imlifidase as pretreatment in gene therapy treatment for patients with Crigler–Najjar syndrome who are immune to AAV

Results presented today at ESGCT 2025 10 October 2025 -- Paris, France and Lund, Sweden -- Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: October 10, 2025

Genethon
1 bis, rue de l’Internationale 91000 EVRY-COURCOURONNES

Relief Therapeutics and NeuroX, Successor to MindMaze, Sign Definitive Agreement for Business Combination

Companies sign definitive agreement following previously announced binding term sheet Transaction to create an expanded, SIX-listed, commercial-stage, AI-driven, scalable health tech company Combined company to be renamed MindMaze Therapeutics Holding...

Category: Biotechnology, Drug Discovery, Other, Pharmaceutical
Posted: October 8, 2025

Relief Therapeutics
contact@relieftherapeutics.com
Avenue de Sécheron 15 1202 Geneva Switzerland

Pharming Group announces U.S. FDA acceptance and Priority Review of supplemental New Drug Application forleniolisib in children with APDS aged 4 to 11 years

If approved, leniolisib will be first and only treatment indicated for children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency Decision based on positive data from...

Category: Biotechnology, Drug Discovery, Other, Pharmaceutical
Posted: October 1, 2025

Pharming Group NV
info@pharming.com
Pharming Group N.V. Vondellaan 47 2332 AA Leiden The Netherlands

Terumo Blood and Cell Technologies Supports Expanded Access to Rare Disease Therapies in Latin America  

Regulatory approvals in 14 countries expand treatment options for sickle cell disease, Guillain-Barré syndrome and myasthenia gravis Approvals align with the historic World Health Assembly resolution on rare diseases. 16...

Category: Biotechnology, Drug Delivery, Other, Pharmaceutical
Posted: September 16, 2025

Terumo Blood and Cell Technologies
Old Belfast Road Millbrook Larne BT40 2SH

Sanofi’s SAR402663 earns fast track designation in the US for neovascular age-related macular degeneration

Designation earned for a one-time intravitreal gene therapy with the potential to eliminate treatment burden for people living with neovascular age-related macular degeneration Neovascular or “wet” age-related macular degeneration can...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: September 11, 2025

Sanofi
One Onslow Street, Guildford, Surrey, GU1 4YS, UK

Iksuda Therapeutics receives FDA IND clearance for IKS014

FDA decision will enable Iksuda to expand its ongoing clinical trial in the US, Australia and Singapore Preliminary data from an ongoing dose-escalation study of IKS014 has shown meaningful clinical activity across...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: July 1, 2025

Iksuda
The Biosphere, Draymans Way, Newcastle Helix Newcastle upon Tyne NE4 5BX