Uniqure, Inc.

Stifel Nicolaus analyst Paul Matteis maintains a Buy rating on GHRS stock, citing GH Research's positive phase 2a study results in postpartum depression and bipolar II disorder, favorable safety profile of GH001, and regulatory progress. H.C. Wainwright also reiterated a Buy rating with a $40 target.

2024 was a challenging year for the biopharma industry, with companies like Bayer, Bristol Myers Squibb, and Johnson & Johnson cutting hundreds to thousands of jobs. Cassava Sciences announced layoffs of 10 employees, 33% of its workforce, following a failed Phase III trial for its Alzheimer's drug. CytomX Therapeutics cut 40% of its staff to focus on clinical programs. Velia, a San Diego biotech, is shutting down, affecting 47 employees. Regeneron's acquisition of Oxular led to layoffs, with no Oxular employees joining Regeneron. Javara, Ring Therapeutics, Outlook Therapeutics, Editas, Bavarian Nordic, BenevolentAI, Chroma Medicine and Nvelop Therapeutics, Cellectar Biosciences, Carisma Therapeutics, Belharra Therapeutics, National Resilience, AmplifyBio, Agenus, Alligator Bioscience, Idorsia Pharmaceuticals, Kronos Bio, Novartis, Recursion Therapeutics, Medigene, Alector, Bristol Myers Squibb, Sonata Therapeutics, 23andMe, Johnson & Johnson, Merck, Gilead Sciences, Adaptimmune, Sensei Biotherapeutics, Marinus Pharmaceuticals, Orna Therapeutics, Thermo Fisher Scientific, Charles River Laboratories, Aurinia Pharmaceuticals, Viracta Therapeutics, Astellas Gene Therapies, Sana Biotechnology, Sage Therapeutics, Compass Pathways, Spero Therapeutics, ICON, Pfizer, Takeda, SalioGen Therapeutics, Evonik, Medtronic, CareFusion Resources, Turnstone Biologics, Leo Pharma, Astellas Pharma, Prime Medicine, Kaléo, Stryker, Relay Therapeutics, ImmunityBio, Shattuck Labs, Inventprise, bluebird bio, Athira Pharma, AGC Biologics, Oncternal Therapeutics, Biosense Webster, Vesigen Therapeutics, Connect Biopharma, BioMarin, IN8bio, Edwards Lifesciences, DermTech, Repare Therapeutics, Genentech, Tome Biosciences, Aadi Bioscience, Lykos Therapeutics, Evotec, Galera Therapeutics, Grail, Ovid Therapeutics, Lexicon Pharmaceuticals, Acelyrin, Boundless Bio, FibroGen, Ajinomoto Bio-Pharma Services, AN2 Therapeutics, Entero Therapeutics, Precigen, Sumitomo Pharma America, uniQure, Vir Biotechnology, Arbutus Biopharma, HilleVax, and Bayer also announced significant layoffs, reflecting a tough year for the industry.

The cell and gene therapy sectors are rapidly advancing, with significant developments like Capricor's BLA submission for DMD cardiomyopathy therapy, Arbor Biotechnologies' FDA clearance for a CRISPR-based therapy trial, Ultragenyx's BLA for MPSIII gene therapy, RESTEM's fast-tracked ULSC therapy for IIM, ViGeneron's rare pediatric disease designation for a retinitis pigmentosa gene therapy, and uniQure's public offering pricing shares at $17.

Centessa appoints new CMO; EyePoint expands board; Air Industries wins $5.9M Air Force contract; I-Mab Chairman to buy $2M ADS; FDA accepts Agios' sNDA; Butterfly Network projects 35% Q4 growth; uniQure prices $75M offering; Synopsys' Ansys acquisition plan accepted; Viking begins obesity drug trial; Jasper reports positive study data; Boston Scientific to acquire Bolt Medical; AMD invests $20M in Absci; Shutterstock appoints new CMO; EVgo secures $75M for expansion; Tiziana's foralumab shows promise; EDAP initiates pancreatic cancer trial; SDIC Power holds EGM; Global Petroleum delivers Lidar data; N2OFF granted Nasdaq extension; NW Natural completes SiEnergy acquisition; Cadogan Energy reports shareholder change; UK auctions £4.25B Gilt; KBN initiates pre-stabilization; Lufthansa launches subordinated debt; Morgan Stanley to stabilize Türkiye İş Bankası securities; Hon Hai sells assets for NTD 358.8M; OTE completes share buyback; Evonik launches EUR 500M bond.

In December 2024, the FDA approved Mesoblast’s remestemcel-L for steroid-refractory GvHD, placed a clinical hold on PepGen’s DMD trial, fast-tracked Sana Biotechnology’s SC291 for SLE, and agreed with uniQure on an accelerated approval pathway for Huntington disease gene therapy AMT-130.

In December 2024, the FDA made several key decisions: granted breakthrough therapy to STK-001 for Dravet syndrome, provided guidance for CNM-Au8 in ALS, aligned on AMT-130's accelerated approval for Huntington's, designated tolebrutinib for nrSPMS, placed a hold on PGN-EDO51 for DMD, and approved tirzepatide for OSA and obesity.

2024 marked significant progress in Huntington’s disease (HD) research, with breakthroughs in understanding somatic instability, advancements in drug development, and both challenges and triumphs in clinical trials. The HD community saw the power of collaboration and innovation, with new voices at HDBuzz and updates from global conferences. Despite setbacks, there's hope with ongoing trials and new drug approvals, moving closer to treatments that could slow or halt HD.

In 2024, Fabry Disease News highlighted key updates: gene therapy trials for Fabry disease showed promise, including AMT-191 and 4D-310 targeting heart and liver. Elfabrio slowed kidney decline. Studies found elevated inflammatory markers in patients and suggested small nerve fiber damage as a diagnostic aid. An algorithm using insurance data aims to speed up diagnosis. Chronic cough and pain were identified as potential Fabry symptoms.

FDA and uniQure agreed on an accelerated approval pathway for AMT-130, an AAV vector-based gene therapy for Huntington disease, using ongoing phase 1/2 study data compared to external control natural history data. The composite Unified Huntington’s Disease Rating Scale (cUHDRS) may serve as an intermediate clinical end point, with supportive evidence from cerebrospinal fluid neurofilament light chain (NfL) decreases. This aligns with uniQure's data and collaborative discussions, aiming to deliver a potentially life-changing therapy to Huntington disease patients.