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CRISPR Therapeutics to Present Preclinical Data on Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase Gene Editing Technology at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress
Globenewswire· 2025-10-01 12:00
Core Insights - CRISPR Therapeutics announced the acceptance of an abstract for oral presentation at the ESGCT 2025 Annual Congress, focusing on its SyNTase gene editing technology for treating Alpha-1 Antitrypsin Deficiency (AATD) [1][5] - The SyNTase platform represents a significant advancement in gene editing, combining compact Cas9 proteins with engineered polymerases for improved efficiency and precision [2] - The technology demonstrated high editing levels (up to 95%) in human hepatocyte cell models with minimal off-target effects (<0.5%) and showed promising results in humanized mouse and rat models [4] Company Overview - CRISPR Therapeutics has evolved from a research-stage company to a leader in gene editing, having achieved the first-ever CRISPR-based therapy approval [6] - The company has a diverse portfolio targeting various diseases, including hemoglobinopathies, oncology, and rare diseases, with a notable product, CASGEVY, approved for sickle cell disease and beta thalassemia [6] - The company has formed strategic partnerships, including with Vertex Pharmaceuticals, to enhance its research and development efforts [6] Presentation Details - The oral presentation titled "Single-dose in vivo gene correction of AATD via LNP-delivered SyNTase editors" is scheduled for October 10, 2025, at the ESGCT conference [5] - The abstract is available online for congress registrants, with updated data to be presented during the session [5]
Precision BioSciences Announces Late-Breaking Poster Presentation at the 30th Annual International Congress of the World Muscle Society
Businesswire· 2025-09-30 11:01
Core Insights - Precision BioSciences, Inc. is a clinical stage gene editing company focused on developing in vivo gene editing therapies for diseases with high unmet needs [1] - The company will present late-breaking preclinical data for its product PBGENE-DMD at the 30th Annual International Congress of the World Muscle Society in Vienna, Austria, from October 7-12, 2025 [1]
Cibus Announces the Election of Kimberly A. Box to Board of Directors
Globenewswire· 2025-09-25 20:05
Core Insights - Cibus, Inc. has elected Kimberly A. Box to its Board of Directors, enhancing its governance and commercialization strategy as it prepares for the launch of advanced trait technologies aimed at improving crop productivity and sustainability [1][2] Company Overview - Cibus is a leading agricultural technology company focused on developing and licensing advanced plant traits that enable higher yields, lower input costs, and more sustainable farming practices [4] - The company utilizes proprietary precision gene editing and breeding systems to develop traits more quickly and accurately than conventional methods, transforming agricultural practices globally [4] Leadership Appointment - Kimberly A. Box brings extensive leadership experience in technology and commercialization, which is expected to strengthen Cibus' strategic governance and long-term value creation efforts [2][3] - Box's previous roles include President and CEO of Gatekeeper Innovation, Inc., and nearly three decades at Hewlett-Packard, where she held various executive positions [2][3] - Her expertise in guiding innovation into scalable businesses is seen as critical for Cibus as it prepares for initial product launches [2][3] Strategic Focus - Cibus aims to improve productivity and sustainability for farmers worldwide through its gene editing platform, which is viewed as a breakthrough in agricultural trait development [3] - The company is focused on enhancing customer adoption and delivering innovative solutions to meet market demands [2][3]
Intellia Therapeutics Announces Positive Longer-Term Phase 1 Data for Nexiguran Ziclumeran (nex-z) in Patients with Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy
Globenewswire· 2025-09-25 13:52
Core Insights - Intellia Therapeutics announced promising long-term follow-up data from the Phase 1 study of investigational gene therapy nexiguran ziclumeran (nex-z) for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) [1][2] - The results indicate significant and durable reductions in serum TTR levels, suggesting potential for improved patient outcomes [2][3] Group 1: Clinical Results - A one-time dose of nex-z led to a mean serum TTR reduction of 92% at 24 months and 90% at 36 months among patients [3][4] - Among 18 patients assessed at 24 months, 72% showed clinically meaningful improvements in the modified Neuropathy Impairment Score (mNIS+7) [5] - Secondary endpoints such as modified body mass index (mBMI), Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN), and neurofilament light chain (NfL) also trended towards improvement [5] Group 2: Safety Profile - Nex-z has been generally well tolerated, with mild to moderate infusion-related reactions being the most common adverse events [6] - No new drug-related events were reported during the follow-up period, and previously reported liver enzyme elevations resolved without intervention [6][8] Group 3: Future Developments - The Phase 3 MAGNITUDE-2 trial is progressing rapidly, with patient enrollment expected to complete in the first half of 2026 [7][8] - Intellia anticipates submitting a biologics license application (BLA) for nex-z by 2028 [7][8] - The trial aims to evaluate the efficacy and safety of nex-z in approximately 50 patients with ATTRv-PN [7][9] Group 4: About Nex-z and ATTR Amyloidosis - Nex-z is based on CRISPR/Cas9 technology and aims to inactivate the TTR gene, potentially becoming the first one-time treatment for ATTR amyloidosis [11] - ATTR amyloidosis is a rare and progressive disease affecting an estimated 50,000 people with hereditary forms and between 200,000 to 500,000 with wild-type forms [12]
Know Thyself 2.0: When Technology Rewrites Life | Aristides G. Eliopoulos | TEDxNTUA
TEDx Talks· 2025-09-19 15:15
[μουσική] [χειροκρότημα] [χειροκρότημα] Καλησπέρα σας. Έχετε κάνει ποτέ μια εξέταση και φοβηθήκατε να δείτε το αποτέλεσμα Μπορεί να ήτανε κάτι απλό, μια γενική αίματος ή κάτι λίγο πιο σοβαρό, μια μικρή βιοψία. Εκείνη στιγμή που ο φόβος να μάθεις ξεπερνάει την ανάγκη να ξέρεις.Τώρα φανταστείτε πως η εξέταση αυτή δεν αφορά το παρόν, αλλά το μέλλον σας. Πώς θα νιώθατε αν ξέρετε ότι έχετε για παράδειγμα 60% αυξημένο κίνδυνο να εμφανίσετε καρδιακιακή νόσο ή 40% μεγαλύτερη πιθανότητα για αλzχάμε στα 58 σας θα θέλ ...
The DNA Dilemma: Shaping Bioethical Technologies Together | Andrea Lei | TEDxSHSID Youth
TEDx Talks· 2025-09-19 14:57
I want you guys to all first picture yourselves walking into a salon here. Instead of choosing a new hairstyle, you're choosing new jeans. Would you buy an enhanced memory gene pack that has a coupon for Ivy students.Perhaps a perfect athleticism combo. The case where we can edit our genes easily is a future that we are quickly approaching. And with these emergent technologies, we are facing questions that no challenges before have ever had to answer.Today, I'm not here to assert whether genetic editing is ...
Is Autopiloting possible for NanoRobot | Dr.Ali Al Hasan | TEDxRiyadh
TEDx Talks· 2025-09-18 16:36
اللي تشوفونه شريحه فيها 7 مليون روبوت نانو ما نقدر نشوفها بالعين المجرده كي ولكن في كل واحد فيكم يمشي في دمه ملايين الروبوتات اليوم ويمكن اغلبكم ما يدري كيف راح اقول لكم بعدين ولكن اتمنى في نهايه اليوم انه تكونوا مستعدين للتحديث الجديد [تصفيق] كليكر ورك اوكي جود روبوتات النانو مو شيء بسيط ابدا شيء جدا اقل من تصميمه لانه في الاساس يشتغل على مستوى الذرات ومستوى الجينات يحتاج ل خبرات كبيره يحتاج لعلوم كبيره لتصميمها الجميل في في روبوتات النمو انها ممكن تدخل جسم الانسان وممكن تسبح بين خلايانك وممكن ممكن توصل لا ...
CRISPR stock wins new overweight at J.P. Morgan (CRSP:NASDAQ)
Seeking Alpha· 2025-09-18 12:58
Group 1 - CRISPR Therapeutics (NASDAQ:CRSP) saw an increase in premarket trading following J.P. Morgan's initiation of coverage with an Overweight recommendation [5] - The company is recognized for its strong position in the gene editing sector, attributed to its promising pipeline potential [5]
Precision BioSciences, Inc. (DTIL) Presents At H.C. Wainwright 27th Annual Global Investment Conference Transcript
Seeking Alpha· 2025-09-16 09:52
Core Viewpoint - Precision BioSciences is a clinical stage gene editing company utilizing its proprietary ARCUS platform to develop in vivo gene editing therapies for diseases with high unmet needs [1][2]. Group 1: Company Overview - Precision BioSciences focuses on gene editing and is currently in the clinical stage of development [1]. - The company leverages its novel ARCUS platform, which is designed to address diseases that have significant unmet medical needs [1]. Group 2: Technology Differentiation - The ARCUS platform distinguishes itself from other gene editing technologies through its unique cutting mechanism, smaller size, and simpler structure [2]. - These characteristics enable ARCUS nucleases to achieve more defined and intended therapeutic outcomes [2].
Precision BioSciences Presents Data from the Phase 1 ELIMINATE-B Trial of PBGENE-HBV at the 6th International Coalition to Eliminate HBV Cure Symposium
Businesswire· 2025-09-12 11:01
Core Insights - Precision BioSciences, Inc. is a clinical stage gene editing company focused on developing in vivo gene editing therapies for diseases with high unmet needs [1] - The company presented clinical data from the Phase 1 ELIMINATE-B trial of PBGENE-HBV targeting chronic Hepatitis B patients [1] - The presentation took place at the International Coalition to Eliminate HBV (ICE-HBV) "HBV Cure Symposium" in Berlin [1]