Intellia Therapeutics, Inc NTLA

Intellia Therapeutics, Inc. es una empresa de edición genómica en etapa clínica, que se enfoca en desarrollar terapéuticas curativas utilizando la tecnología de Repeticiones Palindrómicas Cortas Agrupadas y Regularmente Interespaciadas/CRISPR asociada a 9 (CRISPR/Cas9). CRISPR/Cas9 es una tecnología para la edición del genoma, el proceso de alterar secuencias seleccionadas de ácido desoxirribonucleico genómico. Se enfoca en aprovechar su plataforma modular para avanzar en terapias in vivo y ex vivo para enfermedades con una alta necesidad no satisfecha. Su candidato principal in vivo, NTLA-2001, es para el tratamiento de la amiloidosis por transtiretina (ATTR), así como NTLA-2002 para el tratamiento del angioedema hereditario (HAE). Está desarrollando aplicaciones ex vivo para abordar la inmunooncología y enfermedades autoinmunes. Sus programas ex vivo avanzados incluyen un candidato de células T con receptor de antígeno quimérico (CAR-T) de propiedad exclusiva, NTLA-6001 dirigido a CD30 para el tratamiento de cánceres hematológicos que expresan CD30, incluido el linfoma de Hodgkin clásico recurrente o refractario.

• GlobeNewswire Inc. 17 sept

  Aerska Appoints Laura Sepp-Lorenzino to Scientific Advisory Board

  Aerska, a biotechnology company developing RNA medicines for CNS diseases through its antibody-oligonucleotide conjugate (AOC) platform, has appointed Laura Sepp-Lorenzino to its Scientific Advisory Board. Sepp-Lorenzino brings over 30 years of experience in genetic medicines and nucleic acid therapies, previously serving as Chief Scientific Officer at Intellia Therapeutics and leading nucleic acid programs at Vertex Pharmaceuticals.

  

• GlobeNewswire Inc. 4 sept

  Intellia Therapeutics Secures Non-Dilutive Debt Facility with OrbiMed for up to $400 Million

  Intellia Therapeutics announced a $400 million non-dilutive senior secured term loan facility with OrbiMed, consisting of $75 million upfront and $325 million tied to milestones. The financing provides strategic flexibility as the company advances toward U.S. approval and commercial launch of lonvo-z for hereditary angioedema treatment, while also supporting advancement of nexiguran ziclumeran in transthyretin amyloidosis.

  

• GlobeNewswire Inc. 4 sept

  Intellia Therapeutics to Present at Morgan Stanley 24th Annual Global Healthcare Conference

  Intellia Therapeutics reported positive Phase 3 HAELO clinical data for lonvo-z in HAE, with results presented at EAACI and published in the New England Journal of Medicine. The company anticipates FDA acceptance of a BLA (Biologics License Application) for lonvo-z in the second half of 2026.

  

• The Motley Fool 31 ago

  Cathie Wood Goes Bargain Hunting: 3 Stocks She Just Bought

  Cathie Wood's Ark Invest added to positions in Nvidia, Broadcom, and Intellia Therapeutics on Friday as these stocks declined. Nvidia exceeded earnings expectations with 106% revenue growth and raised guidance to 70% for fiscal 2028, now trading at 14x forward earnings. Broadcom is on track for accelerating growth with an upcoming earnings report. Intellia, a gene-editing biotech, has declined over 50% from recent highs but remains well-funded with analyst support.

  

• GlobeNewswire Inc. 3 jul

  Gene Editing Therapeutics: Transforming Biopharmaceuticals with 12.5% CAGR Growth Forecast

  The global gene editing therapeutics market is projected to grow from USD 0.36 billion in 2026 to USD 0.65 billion by 2031, driven by CRISPR advancements, precision medicine adoption, and increased pharma investments. Key applications include oncology and rare genetic disorders, though high development costs, regulatory complexity, and safety concerns remain significant challenges.