Krystal Biotech

Krystal Biotech updates on EMA's CHMP requesting written responses for B-VEC, no major objections to full approval. Anticipated CHMP opinion in Q1 2025, launch in Germany in Q2 2025. B-VEC, FDA-approved in May 2023, treats DEB with non-invasive, redosable gene therapy. Company reports strong financial health and Q3 2024 earnings, focusing on Europe and Japan expansion.

Krystal Biotech updates on EMA's review of B-VEC for DEB, with CHMP cancelling Oral Explanation and requesting written responses. No Major Objections for full approval; CHMP opinion expected in 1Q 2025, with German launch planned for Q2 2025.

Krystal Biotech's CHMP Oral Explanation for B-VEC was cancelled, with written responses to remaining issues requested. No Major Objections are outstanding, and a CHMP opinion is now expected in 1Q 2025, with a Germany launch planned for 2Q 2025.

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Krystal Biotech's CEO, Krish S. Krishnan, will participate in fireside chats at Citi’s 2024 Global Healthcare Conference (Dec 3) and the 7th Annual Evercore HealthCONx Conference (Dec 4). Both events will be webcast live and archived on the company's website.

Samuel Simmons, with ichthyosis, spends hours daily scrubbing dry, scaly skin. Research for a cure is slow, with dermatologists repurposing drugs for other skin conditions. Ichthyosis, a family of genetic skin diseases, causes skin cell turnover issues, leading to dry, cracked skin. Despite some progress with retinoids, a cure remains elusive. Dermatologists like Amy Paller and Joyce Teng are exploring immunomodulatory drugs used for psoriasis, revealing shared immune signatures. Clinical trials with IL-17-targeting drugs have shown mixed results, highlighting the need for personalized treatments due to ichthyosis's diversity. Simmons, frustrated but hopeful, keeps an eye on ongoing research.

Global Organoids Market to grow at a CAGR of ~14% by 2030, driven by personalized medicine, organoid use in drug discovery, and cancer research. North America leads the market, with key players including Thermofisher Scientific Inc., StemCell Technologies Inc., and others. Organoids mimic human organs, aiding in disease modeling and drug testing, but face challenges like high costs and limited vascularization.

Abeona Therapeutics' BLA for pz-cel, an RDEB treatment, was accepted by the FDA with a PDUFA date of April 29, 2025. This resubmission follows a CRL in April 2024, addressing CMC issues. The BLA is supported by phase 3 VIITAL study data showing significant wound healing and pain reduction in RDEB patients. If approved, pz-cel would be the second advanced therapeutic for RDEB, following Krystal Biotech's B-VEC approval in 2023.

Non-cystic fibrosis bronchiectasis market driven by rising prevalence, early diagnosis, and drug development. Over 15 companies, including Zambon SpA, Insmed, and AstraZeneca, are developing 15+ pipeline therapies. Key drugs in trials include Colistimethate sodium, Benralizumab, and Brensocatib.