Intellia Therapeutics

In 2024, Angioedema News highlighted top advancements in angioedema treatment, including deals for donidalorsen, NTLA-2002's 95% attack reduction, Takeda's partnerships, Takhzyro's efficacy in adolescents, deucrictibant's Phase 3 trials, FDA's lift on deucrictibant hold, Ionis' FDA application for donidalorsen, garadacimab's safety, Haegarda's real-world effectiveness, and Orladeyo's long-term benefits in Japan.

In 2024, FAP News Today highlighted advancements in FAP treatment, including disease-modifying drugs, gene-editing therapies, and liver transplants. Key findings showed treatments like Onpattro, Tafamidis, and Wainua effectively slow disease progression, with some therapies showing benefits in early stages or reducing heart damage.

Layoffs have slowed but companies like Bayer, Bristol Myers Squibb, and Johnson & Johnson plan significant cuts in 2024. Firms are shifting focus, e.g., licensing out therapies to concentrate on in vivo platforms. Hiring for cell and gene therapy roles emphasizes more than technical skills, seeking well-rounded candidates.

The CAR-T cell therapy market, valued at USD 7.31 billion in 2024, is expected to grow at a CAGR of 36.8% to USD 188.84 billion by 2034. This growth is driven by R&D advancements and the therapy's effectiveness against cancers like lymphoma and leukemia, even when other treatments fail.

The CAR-T cell therapy market, valued at USD 7.31 billion in 2024, is expected to grow to USD 188.84 billion by 2034, with a CAGR of 36.8%. This growth is driven by advancements in cancer treatment, particularly for lymphoma, leukemia, and multiple myeloma, and the rising prevalence of cancers like DLBCL. North America leads the market due to advanced healthcare infrastructure and significant R&D investments.

Intellia Therapeutics' CRISPR-based therapy, nex-z, received FDA's RMAT designation for treating ATTRv-PN, following promising Phase 1 results. Nex-z, a single-dose gene editing therapy, inactivates the TTR gene to prevent TTR protein production. The RMAT designation expedites therapy development, offering benefits like early FDA engagement and potential priority review.

CRISPR Therapeutics, a pioneer in gene therapy, faces challenges despite its first-mover advantage and FDA approval of Casgevy. Profitability remains elusive, with slow commercialization and significant financial losses projected. Intense competition from other biotech firms adds to the uncertainty, making cautious investment advisable.

The CRISPR and Cas gene market was valued at $3.3 billion in 2023, with a CAGR of 20.38% since 2018. It's projected to reach $8.8 billion by 2028 and $24.6 billion by 2033. Growth drivers include personalized medicine, genetic disorders, and synthetic biology. Challenges include regulatory complexities and healthcare access in developing countries. The market is segmented by type, application, product type, end use, and service type, with various segments showing significant growth potential. North America leads the market, but Asia-Pacific and Africa are expected to grow fastest. The market is dominated by large players like Danaher Corporation and Agilent Technologies Inc.

The global genome editing market, valued at USD 5.5 billion in 2022, is projected to grow at a CAGR of 13.9% to USD 17.5 billion by 2031, driven by advancements in CRISPR and other gene-editing technologies, increasing applications in therapeutics and agriculture, and rising investments in genetic research and development.