www.fiercebiotech.com Open in urlscan Pro
104.18.0.165  Public Scan

Form analysis
2 forms found in the DOM

GET /search-results

<form action="/search-results" method="get">
  <fieldset class="d-flex align-items-center position-relative input-search"><input type="text" placeholder="Search" name="fulltext_search" class="search-text w-100"><button class="search-submit d-flex align-items-center" id="search-submit"
      type="submit"><svg aria-hidden="true" focusable="false" data-prefix="far" data-icon="search" class="svg-inline--fa fa-search fa-w-16" role="img" xmlns="http://www.w3.org/2000/svg" viewBox="0 0 512 512">
        <path fill="currentColor"
          d="M508.5 468.9L387.1 347.5c-2.3-2.3-5.3-3.5-8.5-3.5h-13.2c31.5-36.5 50.6-84 50.6-136C416 93.1 322.9 0 208 0S0 93.1 0 208s93.1 208 208 208c52 0 99.5-19.1 136-50.6v13.2c0 3.2 1.3 6.2 3.5 8.5l121.4 121.4c4.7 4.7 12.3 4.7 17 0l22.6-22.6c4.7-4.7 4.7-12.3 0-17zM208 368c-88.4 0-160-71.6-160-160S119.6 48 208 48s160 71.6 160 160-71.6 160-160 160z">
        </path>
      </svg></button><input type="hidden" placeholder="Search" name="dns" value="fiercehealthcare_com,fiercebiotech_com,fiercepharma_com"></fieldset>
</form>

GET /search-results

<form class="w-100" action="/search-results" method="get">
  <fieldset class="d-flex justify-content-between position-relative input-search"><input type="text" placeholder="Search" name="fulltext_search" class="search-text w-100"><button class="search-submit d-flex align-items-center" id="search-submit"
      type="submit"><svg aria-hidden="true" focusable="false" data-prefix="far" data-icon="search" class="svg-inline--fa fa-search fa-w-16" role="img" xmlns="http://www.w3.org/2000/svg" viewBox="0 0 512 512">
        <path fill="currentColor"
          d="M508.5 468.9L387.1 347.5c-2.3-2.3-5.3-3.5-8.5-3.5h-13.2c31.5-36.5 50.6-84 50.6-136C416 93.1 322.9 0 208 0S0 93.1 0 208s93.1 208 208 208c52 0 99.5-19.1 136-50.6v13.2c0 3.2 1.3 6.2 3.5 8.5l121.4 121.4c4.7 4.7 12.3 4.7 17 0l22.6-22.6c4.7-4.7 4.7-12.3 0-17zM208 368c-88.4 0-160-71.6-160-160S119.6 48 208 48s160 71.6 160 160-71.6 160-160 160z">
        </path>
      </svg></button></fieldset>
</form>

Text Content

 * Fierce Pharma
 * Fierce Biotech
 * Fierce Healthcare
 * Fierce Life Sciences Events

 * Advertise
 * About Us



 * Research
 * Biotech
   
   * Clinical Data
   * Venture Capital
   * Deals
 * Medtech
   
   * Devices
   * Diagnostics
   * AI and Machine Learning
 * CRO
 * Special Reports
 * Trending Topics
   
   * Cell & Gene Therapy
   * COVID-19
 * Podcasts

 * Resources
   
   * Fierce Events
   * Industry Events
   * Webinars
   * Podcasts
   * Whitepapers
 * Events
   
   
 * Subscribe

Subscribe

 * Research
 * Biotech
   
   * Clinical Data
   * Venture Capital
   * Deals
 * Medtech
   
   * Devices
   * Diagnostics
   * AI and Machine Learning
 * CRO
 * Special Reports
 * Trending Topics
   
   * Cell & Gene Therapy
   * COVID-19
 * Podcasts

 * Resources
   
   * Fierce Events
   * Industry Events
   * Webinars
   * Podcasts
   * Whitepapers
 * Events
   
   
 * Subscribe

 * Fierce Pharma
 * Fierce Biotech
 * Fierce Healthcare
 * Fierce Life Sciences Events

 * Advertise
 * About Us




Biotech


PATIENTS ATTACK-FREE A YEAR AFTER RECEIVING INTELLIA'S HEREDITARY ANGIOEDEMA
CRISPR MED

By Annalee ArmstrongJun 11, 2023 08:30am
Intellia Therapeuticshereditary angioedemaCRISPRinflammatory diseases
The CRISPR therapeutic is designed to inactivate the kallikrein B1 (KLKB1) gene.
(YinYang/ iStock/Getty Images Plus)

Patients with the inflammatory disorder hereditary angioedema who took Intellia
Therapeutics’ CRISPR medicine have been attack free for a year or more after
receiving just one dose.




The gene editing company has provided a bit more data on NTLA-2002, its second
candidate to enter the clinic, providing a look at durability a year or more out
from treatment in a presentation at the European Academy of Allergy and Clinical
Immunology Hybrid Congress in Germany this weekend. NTLA-2002 is being tested in
a phase 1/2 trial of patients with type I or II HAE, which is a rare inherited
disorder that causes rapid swelling all over the body.




Intellia is presenting NTLA-2002 as a “functional cure” for the disorder and the
latest updated interim results add more weight to the question of how long the
single-dose treatment lasts.

Across the 10 patients in the study, a single dose led to a 95% mean reduction
in monthly attack rate through the latest follow up, which had a February 17 cut
off.


RELATED

Intellia is ready to talk 'functional cure' for HAE with interim data in hand
for 2nd CRISPR candidate

The study is looking at the safety, tolerability and pharmacokinetics of
NTLA-2002. One measure is levels of plasma kallikrein protein, which is a serine
protease known to be involved in many processes in the body, including blood
coagulation and the classical complement pathway. Approved medicines for HAE
inhibit this mechanism to try to address attacks as they happen or prevent them.



All patients who received a greater than 60% reduction of plasma kallikrein,
which is nine of the patients, have remained attack-free following the 16-week
observation period and through the latest follow up. Intellia said that the
longest attack-free span observed so far is 13 months.

The single patient that did not achieve the 60% plasma kallikrein reduction, who
received the lowest study dose, experienced a mild HAE attack with minor
swelling in the hand following a sports injury. But that was after more than a
year without an attack. The event did not require any medical intervention and
the patient has not had an attack since.

“We are thrilled to see that the earliest-dosed patients are attack free for
approximately a year or longer, with NTLA-2002 demonstrating a very favorable
safety profile,” CEO John Leonard said in a statement. “These remarkable attack
rate reductions have been consistent, even in patients with the most severe
symptoms.”


RELATED

Fierce Biotech Summit: Gene editing execs reflect on promises and challenges as
CRISPR celebrates 10-year anniversary

Three of the patients who had the highest rate of attacks per month—one
experiencing up to 16 or more—all became attack-free by the end of the 16-week
primary observation period, and are still without attacks through the February
cut off. The longest duration without an attack in this group is currently 11.5
months and counting.



Safety was consistent with earlier data drops, with only grade 1 events such as
infusion-related reactions and fatigue.

Intellia is hoping that the CRISPR therapeutic, which is designed to inactivate
the kallikrein B1 (KLKB1) gene, will be a one-and-done therapy for the disease.

Dosing has kicked off for the phase 2 portion of the study already, with
completion of enrollment expected in the second half. The company previously
reported plasma kallikrein reduction numbers, showing that NTLA-2002 reduced the
biomarker and that patients were attack-free up to 10.6 months after receiving
the therapy.

Clinical DataBiotechClinical Data

Our Newsletter







RELATED ARTICLES

After FDA rebuke, Owlet relaunches baby-monitoring ‘sock’ with agency’s blessing
Jun 21, 2023 10:16am
Pieris stock deflates as AstraZeneca stops trials of asthma drug in response to
toxicology signal
Jun 21, 2023 09:10am
Ex-Novartis NASH drug clears clinical test, fueling Boston's pursuit of Akero,
89bio
Jun 21, 2023 07:15am
Flagship-launched Empress aims to be a small molecule speedster, with up to 4
INDs over next 2 years
Jun 21, 2023 06:00am
See more articles
 * Connect
   
   * The Team
   * Advertise
 * Join Us
   
   * Newsletters
   * Resources
   * RSS Feeds
 * Our Brands
   
   * Fierce Pharma
   * Fierce Biotech
   * Fierce Healthcare
 * Our Events
   
   * Life Sciences Events

©2023 Questex LLC All rights reserved.
Terms of use
Privacy Policy



Close

In order to deliver a personalized and enhanced site navigation, we store
information on how you use the site. By continuing to use this site and access
it's features, you are consenting to our use of cookies. For more information,
please see the Questex Privacy Policy.