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Former Pfizer CSO Mikael Dolsten Joins Two Biotech Boards, Advancing Gene Editing and Mast Cell Therapeutics

Gene EditingRare Disease
  • Dr. Mikael Dolsten, former Pfizer Chief Scientific Officer, has joined the boards of Arbor Biotechnologies and MC Sciences as both companies advance novel therapeutic platforms.
  • Dolsten's 16-year tenure at Pfizer included advancing over 150 drug candidates into clinical studies and leading regulatory approval of 36 medicines and vaccines.
  • Arbor Biotechnologies is progressing its lead gene editing program ABO-101 for primary hyperoxaluria type 1 into clinical trials.
  • MC Sciences is developing first-in-class mast cell-targeting therapeutics for diseases including chronic urticaria, systemic mastocytosis, and asthma.

First-in-Human iPSC-CAR-NK Cell Therapy Shows Promise for Autoimmune Disease Treatment

Gene Editing
  • Qihan Biotech's QN-139b represents the first-ever application of iPSC-CAR-NK cells in treating autoimmune disease, successfully inducing immune reset in a patient with refractory systemic sclerosis.
  • The dual-targeting therapy eliminates pathogenic B cells and plasma cells by targeting both CD19 and BCMA, incorporating nine gene edits and advanced safety features including a tEGFR safety switch.
  • After six months of treatment, the patient showed significant reduction in autoantibodies, dramatic improvement in skin scores, and histological evidence of B cell clearance and fibrosis suppression.
  • The groundbreaking clinical results were published in Cell, marking a potential breakthrough for treating severe autoimmune diseases with off-the-shelf cell therapies.

CRISPR Base Editing Shows Promise for Treating Fatal Pediatric Vascular Disease MSMDS

Gene EditingRare Disease
  • Researchers from Mass General Brigham and UT Southwestern have developed customized CRISPR-Cas9 base editing therapies that successfully treat multisystemic smooth muscle dysfunction syndrome (MSMDS) in preclinical models.
  • The bespoke gene-editing approach extended survival four-fold in mouse models and prevented disease symptoms when administered early, targeting the ACTA2 gene mutation that causes this lethal pediatric condition.
  • Both research teams have engaged with the FDA for clinical trial preparation, with Mass General Brigham securing rare disease designations to accelerate development toward human testing.
  • The breakthrough represents the first CRISPR-based therapeutic specifically designed to target vascular smooth muscle cells, potentially opening new treatment avenues for other genetic vascular diseases.

Eli Lilly Acquires Gene-Editing Biotech Verve Therapeutics for $1.3 Billion to Expand Cholesterol Treatment Pipeline

Gene Editing
  • Eli Lilly will acquire gene-editing startup Verve Therapeutics for up to $1.3 billion, including an upfront payment of almost $1 billion and $300 million in milestone-based payments.
  • The acquisition strengthens Lilly's pipeline beyond its blockbuster weight-loss and diabetes drugs, focusing on one-time gene-editing therapies for high cholesterol in heart disease patients.
  • Verve's lead therapies use base editing technology to target PCSK9, ANGPTL3, and LPA genes responsible for regulating blood cholesterol levels.
  • The companies were already partnering on cholesterol-lowering treatments, with Verve's VERVE-102 therapy currently in early-stage trials for familial hypercholesterolemia.

Intellia's CRISPR Gene Therapy Shows 98% Reduction in HAE Attacks After Three Years

Gene EditingOrphan DrugRare Disease
  • Intellia Therapeutics reported three-year follow-up data showing lonvoguran ziclumeran achieved a 98% mean reduction in monthly hereditary angioedema attack rates across all 10 Phase 1 patients.
  • All patients remained attack-free and treatment-free for a median of 23 months, demonstrating the potential for lonvo-z to become the first one-time therapy for HAE patients.
  • The company's Phase 3 HAELO trial completed screening ahead of schedule, with results expected in the first half of 2026 and a planned U.S. launch in 2027.
  • The CRISPR-based therapy targets the KLKB1 gene to prevent HAE attacks and has received multiple regulatory designations including FDA Orphan Drug and RMAT status.

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1
Intellia Therapeutics
Posted 12/10/2021

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

NCT06634420Active, Not RecruitingPhase 3
Intellia Therapeutics
Posted 1/15/2025

Federal Circuit Upholds Invalidation of Agilent's CRISPR Guide RNA Patents in Synthego Victory

Gene Editing
  • The U.S. Court of Appeals for the Federal Circuit affirmed PTAB decisions invalidating Agilent Technologies' patents on chemically modified CRISPR guide RNAs, following challenges by Synthego Corp.
  • The court clarified that anticipation under patent law requires only one operable embodiment, not the full scope enablement mandated for patent validity under Section 112.
  • The ruling removes patent barriers for chemically modified guide RNAs featuring 2'-O-methyl and phosphonoacetate modifications, potentially accelerating CRISPR therapeutic development.
  • Synthego's CEO emphasized the decision keeps the path clear for broader innovation in the CRISPR therapeutics market and supports advancement of curative cell and gene therapies.

MaxCyte and Ori Biotech Partner to Enhance CAR-T Cell Manufacturing Through Platform Integration

CAR-TGene Editing
  • MaxCyte and Ori Biotech announced a strategic collaboration integrating their ExPERT™ and IRO® platforms to improve gene-edited T cell yields and reduce manufacturing timelines.
  • The partnership will evaluate CD19 CAR expression via CRISPR knock-in in activated T cells as the initial test system to optimize manufacturing processes.
  • MaxCyte's Flow Electroporation® technology, used in over 19 active clinical and commercial programs, will be combined with Ori's automated manufacturing platform.
  • The collaboration aims to provide therapy developers with enhanced tools to achieve clinically relevant quantities of gene-edited T cells more rapidly and efficiently.

Taconic Biosciences Secures Exclusive CRISPR License from Helmholtz Munich to Enhance Animal Model Generation

Gene Editing
  • Taconic Biosciences has entered into an exclusive license agreement with Helmholtz Munich to acquire patented CRISPR-based gene editing technology for improved genetically modified animal model generation.
  • The partnership enhances Taconic's ExpressMODEL® platform to deliver high-quality, precise CRISPR/Cas9-generated custom animal models on an accelerated timeline to preclinical researchers.
  • The collaboration aims to accelerate the creation of precise models that uncover the genetic basis of diseases, supporting scientific discovery and paving the way for future medical breakthroughs.
  • Taconic has been a pioneer in CRISPR/Cas9 gene editing services for over a decade and strengthens its position as a leader in commercializing cutting-edge CRISPR gene editing services.

Biocytogen Secures Japan Patent for RenMab Platform, Strengthening Global IP Portfolio for Fully Human Antibody Discovery

Monoclonal AntibodyGene Editing
  • Biocytogen Pharmaceuticals has received a Japan Patent Office invention patent for its RenMab fully human antibody mouse platform technology, marking a significant milestone in global intellectual property expansion.
  • The RenMab platform uses proprietary SUPCE technology to replace murine immunoglobulin genes with human counterparts, enabling generation of fully human antibodies without additional humanization steps.
  • The company has established licensing agreements with over 20 companies including Merck KGaA, Janssen/Johnson & Johnson, and BeiGene, generating over 1,000,000 fully human antibody sequences against more than 1,000 therapeutic targets.
  • Biocytogen's RenMice platform has secured patent grants in nearly 10 countries with nearly 40 patent applications under examination across 15 countries and regions.

Beam Therapeutics Receives FDA Orphan Drug Designation for BEAM-101 Sickle Cell Disease Therapy

Gene Editing
  • The FDA has granted orphan drug designation to BEAM-101, Beam Therapeutics' investigational base-edited cell therapy for treating sickle cell disease affecting approximately 100,000 Americans.
  • Clinical data from seven patients in the BEACON Phase 1/2 trial demonstrated robust increases in fetal hemoglobin and reductions in sickle hemoglobin with normalized hemolysis markers.
  • The orphan drug designation provides potential benefits including tax credits for clinical trials, user fee exemptions, and seven years of market exclusivity upon approval.
  • Beam expects to dose 30 patients in the ongoing BEACON trial by mid-2025, with updated clinical data accepted for presentation at the European Hematology Association Congress in June 2025.

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880RecruitingPhase 1
Beam Therapeutics Inc.
Posted 8/30/2022

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