Intellia Therapeutics

Acquired Hemophilia A Pipeline Expands with 10+ Novel Therapies in Development

6 days ago

• Global Acquired Hemophilia A pipeline comprises over 10 companies developing innovative therapies across various clinical stages, according to DelveInsight's 2025 report. • Recent clinical advances include promising results for rituximab as a potential first-line therapy and Pfizer's positive Phase 3 AFFINE trial results for giroctocogene fitelparvovec gene therapy. • Key pharmaceutical players including Novo Nordisk, Sanofi, Pfizer, and Belief Biomed are advancing treatments ranging from gene therapies to monoclonal antibodies targeting this rare autoimmune bleeding disorder.

Acadia Pharmaceuticals Wins Patent Infringement Case Against Aurobindo for Parkinson's Drug Nuplazid

6 days ago

• A U.S. District Court ruled that Aurobindo Pharma infringes on two key patent claims held by Acadia Pharmaceuticals for its Parkinson's disease drug Nuplazid. • The court rejected Aurobindo's arguments that Acadia's patent claims were invalid, providing significant intellectual property protection for Acadia's flagship product. • Following the favorable ruling, Acadia's stock soared by over 34% to a one-year high of $23.61, reflecting investor confidence in the company's market position.

Novel Gene-Editing Therapy Shows Promise for Rare Fatal Heart Disease ATTR-CM

8 days ago

• Singapore researchers are leading Asia's first clinical trial of Nexiguran Ziclumeran (nex-z), a groundbreaking gene-editing therapy targeting transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and potentially fatal heart disease. • The MAGNITUDE study, led by Assistant Professor Lin Weiqin from the National University Heart Centre, Singapore, uses CRISPR-Cas9 technology to alter DNA and reduce production of disease-causing proteins in patients with ATTR-CM. • Early phase clinical trials have shown "consistent, rapid, and durable reductions" in abnormal proteins with minimal side effects, offering new hope to approximately 150 diagnosed patients in Singapore and potentially many more undiagnosed cases.

Stylus Medicine Emerges with $85M to Revolutionize In Vivo Genetic Medicines

9 days ago

• Stylus Medicine has emerged from stealth with $85 million in financing to develop its platform combining sequence-specific genome integration with cell-targeted lipid nanoparticle delivery. • The company's technology aims to overcome limitations of ex vivo cell therapy manufacturing by enabling durable, flexible, and scalable in vivo cell engineering, with initial focus on CAR-T therapies. • Led by industry veterans Emile Nuwaysir (CEO) and Jason Fontenot (CSO), Stylus has secured backing from prominent investors including RA Capital Management, Khosla Ventures, Chugai Venture Fund, Eli Lilly, and Johnson & Johnson Innovation.

Vertex Pharmaceuticals Abandons AAV Vector Technology Amid Broader Industry Retreat from Gene Therapy

15 days ago

• Vertex Pharmaceuticals has discontinued all research on adeno-associated virus (AAV) vector technology, impacting partnerships with Affinia Therapeutics and Tevard Biosciences focused on Duchenne muscular dystrophy treatments. • The retreat from AAV vectors follows a broader industry trend, with Pfizer, Roche, Takeda, and Biogen all scaling back gene therapy programs due to safety concerns, limited payload capacity, and high manufacturing costs. • Despite industry pullback, companies like Affinia Therapeutics continue developing next-generation AAV vectors, while others explore alternative delivery systems such as Herpes simplex virus-1 vectors with larger genetic payloads.

Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions

2 months ago

• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors. • Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing. • Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.

Epicrispr Biotechnologies Secures $68 Million to Advance First Epigenetic Therapy for FSHD

2 months ago

• Epicrispr Biotechnologies has raised $68 million in Series B funding to develop EPI-321, a first-in-class epigenetic therapy targeting facioscapulohumeral muscular dystrophy (FSHD). • New Zealand's Medsafe has approved Epicrispr's clinical trial application, with the first-in-human study of EPI-321 expected to begin in 2025 to evaluate its safety and biological activity in adults with FSHD. • EPI-321 uses CRISPR technology to silence the DUX4 gene that drives FSHD progression and has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations.

CRISPR-Cas9 Market Set to Surge: Over 25 Therapies in Clinical Trials Expected to Launch Within 5-7 Years

2 months ago

• The global CRISPR-Cas9 therapy market is rapidly expanding with over 25 therapies currently in clinical trials, many of which are expected to receive regulatory approval and enter the market within the next 5-7 years. • Casgevy, the first approved CRISPR-Cas9 therapy for sickle cell anemia and beta thalassemia, is currently available in the US, EU, UK, Saudi Arabia, and Bahrain at approximately $2 million per treatment course. • Applications of CRISPR-Cas9 technology are expected to expand beyond genetic disorders and cancers to include cardiovascular, neurodegenerative, and CNS diseases, potentially transforming into a multi-billion-dollar industry.

Bayer and CRISPR Therapeutics Form $300 Million Joint Venture to Advance Gene-Editing Therapies

2 months ago

• Bayer is investing $300 million over five years in a new joint venture with CRISPR Therapeutics, plus $35 million for a minority stake in the gene-editing pioneer. • The collaboration will focus on developing potential curative treatments for hemophilia, congenital heart disease, and Stargardt disease using CRISPR-Cas9 gene-editing technology. • This partnership represents Bayer's first investment through its newly established Bayer LifeScience Centre and joins other major pharmaceutical companies including Vertex, Novartis, and Celgene in backing CRISPR-Cas9 technology.

Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments

3 months ago

• Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027. • KalVista Pharmaceuticals' sebetralstat receives Orphan Drug Designation in Japan, marking progress toward the first oral on-demand treatment for HAE attacks. • The global angioedema pipeline includes 20+ companies developing innovative therapies, with several promising candidates in late-stage development from companies like ADARx, BioMarin, and Astria Therapeutics.

RNA Editing Breakthroughs Show Clinical Promise with Wave Life Sciences Leading First Human Results

3 months ago

• Wave Life Sciences achieves first-ever RNA editing results in humans for alpha-1 antitrypsin deficiency, demonstrating therapeutic protein level increases with a single dose treatment. • RNA editing emerges as a potentially safer alternative to DNA editing, with companies like Ascidian Therapeutics and Korro Bio advancing clinical trials for rare genetic disorders. • The field has evolved significantly over the past decade, with new editing capabilities and delivery methods opening potential treatment opportunities for over 10 million patients.

IDEAYA Biosciences Reports Strong Pipeline Progress and $1.1B Cash Position for 2024

3 months ago

• IDEAYA Biosciences advances multiple oncology programs, with over 230 patients enrolled in potential registration-enabling trial for darovasertib in metastatic uveal melanoma, targeting key readouts by end of 2025. • Company strengthens its position with approximately $1.1 billion cash reserve as of December 2024, expected to fund operations into at least 2028. • Pipeline expansion includes progress on DLL3 TOP1i ADC IDE849 showing 73% response rate in early SCLC data, and advancement of three new candidates targeting solid tumors.

Intellia Therapeutics Prioritizes Late-Stage CRISPR Therapies, Announces Workforce Reduction

4 months ago

• Intellia Therapeutics is prioritizing NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis, focusing on late-stage development. • The company plans to complete enrollment for the Phase 3 HAELO study of NTLA-2002 in HAE in the second half of 2025, with a BLA submission expected in 2026. • Intellia anticipates enrolling over 550 patients in the MAGNITUDE study for nex-z in ATTR amyloidosis with cardiomyopathy (ATTR-CM) by the end of 2025. • A strategic reorganization, including a 27% workforce reduction, will extend the company's cash runway into the first half of 2027, supporting commercial readiness.

Retifanlimab Plus Chemotherapy Shows Promise in Advanced Squamous Cell Anal Carcinoma

5 months ago

• Phase 3 POD1UM-303 trial shows retifanlimab combined with chemotherapy significantly improves progression-free survival in patients with locally recurrent or metastatic squamous cell anal carcinoma (SCAC). • The combination therapy demonstrated a median PFS of 9.3 months compared to 7.4 months with chemotherapy alone, marking a potential new standard of care. • Interim overall survival data also suggest a trend towards improvement with the addition of retifanlimab, with manageable immune-related adverse effects. • The study's success supports a planned supplemental biologics license application (sBLA) for retifanlimab, offering hope for addressing unmet needs in SCAC treatment.

Acoramidis Gains Global Momentum: FDA Approval, EU Recommendation, and Promising Clinical Data

6 months ago

• Acoramidis (Attruby), developed by Stanford Medicine and BridgeBio, receives FDA approval for transthyretin amyloid cardiomyopathy (ATTR-CM) treatment, marking a significant milestone. • The European Medicines Agency's CHMP recommends acoramidis for EU marketing authorization based on positive Phase 3 ATTRibute-CM trial results. • Clinical trials demonstrate acoramidis' efficacy in reducing cardiovascular-related hospitalizations and improving survival rates for ATTR-CM patients. • Bayer and BridgeBio collaborate to commercialize acoramidis, with Bayer holding EU rights and plans for a launch in Europe in early 2025.

Intellia Therapeutics Initiates Phase 3 Trial of NTLA-2002 for Hereditary Angioedema

8 months ago

• Intellia Therapeutics has dosed the first patient in its Phase 3 HAELO trial evaluating NTLA-2002 for hereditary angioedema (HAE). • The HAELO trial is a global, randomized, double-blind, placebo-controlled study involving 60 adults with Type I or Type II HAE. • NTLA-2002, a single-dose CRISPR-based therapy, targets the _KLKB1_ gene to reduce plasma kallikrein activity and prevent HAE attacks. • Intellia anticipates completing enrollment in the second half of 2025 and plans for a potential U.S. launch in 2027, pending regulatory approval.