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Pfizer partners with Beam to advance in vivo base editing therapy
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Pfizer partners with Beam to advance in vivo base editing therapy.
Over $1.3 billion, Pfizer partners with base editing company Beam to advance in vivo base editing therapy.
On January 10, 2022, pharmaceutical giant Pfizer and leading base editing company Beam announced an exclusive four-year partnership that will see Beam receive an upfront payment of $300 million from Pfizer and milestone payments of up to $ 1.05 billion .
The four-year partnership will combine Pfizer’s extensive experience in mRNA, LNP, gene therapy and other drug development and Beam’s leading edge in base editing and mRNA/LNP delivery to jointly advance the liver , muscle and central nervous system . Systematic in vivo base editing therapies for three rare genetic diseases that are not included in Beam’s existing R&D pipeline.
In this collaboration, Beam delivers its proprietary base-editing technologies through LNP-wrapped mRNA to target organs.
These technologies, combined with Pfizer’s proven experience in developing and manufacturing vaccines and drugs, have the potential to bring benefits to patients with rare genetic diseases. Revolutionary treatment.
In addition to the $300 million upfront payment and milestone payment of $1.05 billion paid to Beam by Pfizer,
Beam has the right to enter into a global development and commercialization agreement after the completion of the Phase 1/2 clinical trial. %/35% share the development and commercialization costs and share the net profit.
Since the outbreak of the COVID-19 epidemic, only Pfizer/BioNTech and Moderna have successfully launched mRNA vaccines, which has also brought them huge cash flows of tens of billions of dollars.
Prior to this, Moderna had announced its official entry into the field of gene editing.
In November 2021, Moderna announced a partnership with the gene editing startup Metagenomi to combine the CRISPR-based new generation gene editing system and other gene editing systems owned by Metagenomi with Moderna’s Combining mRNA technology and LNP delivery technology to develop the next generation of in vivo gene editing therapies to develop treatments for patients with serious genetic diseases .
Pfizer chose to cooperate with the base editing company Beam. Unlike other CRISPR gene editing, Beam’s Base Editing is more precise and can target and repair a single base in the genome without causing DNA damage.
Double-strand breaks (DSBs) , and therefore do not lead to the associated potential risks, are considered safer.
Beam was co-founded in May 2018 by authoritative experts in the field of gene editing, including David Liu , J. Keith Joung and Zhang Feng , and was listed on Nasdaq in February 2020.
Zhang Feng (left), Liu Ruqian (middle), J. Keith Joung (right)
Advantages and Directions of Base Editing
Beam is based on single base editing technology, by rewriting a single base in the genome, producing precise, predictable and efficient genetic modifications on the target DNA sequence without causing DNA double-strand breaks (DSBs) , so it is considered safer. Controllable.
Currently, base editors can provide a variety of gene editing effects for therapeutic gene intervention, including: correcting disease-causing point mutations (Gene Correction) , modifying genes to generate protective mutations (Gene Modification) , activating gene expression ( Gene Activation) , Gene Silencing , Multiplex Editing .
How Beam is Delivered
Beam’s delivery strategies are comprehensive and include electroporation , lipid nanoparticles (LNP), and adeno-associated virus (AAV) .
Beam believes that no single delivery strategy can efficiently deliver therapeutic drug ingredients to different organs and tissues.
Therefore, Beam’s plans are to use electroporation for in vitro blood and immune cell delivery; LNP for in vivo delivery to the liver and other organs; and AAV for eye and central nervous system delivery.
Beam’s R&D pipeline
Beam’s current R&D pipeline is divided into four categories: in vitro edited hematopoietic stem cell therapy , in vitro edited T cell therapy , in vivo LNP targeting liver genetic diseases , and AAV treatment of eye diseases .
Among them, BEAM-101 , a therapy for the treatment of sickle cell disease and β-thalassemia by in vitro editing of hematopoietic stem cells , is the most advanced.
It has passed the IND application and will start Phase 1/2 human clinical trials in the second half of this year.
BEAM-102 for the treatment of sickle cell disease will also submit an IND application in the second half of this year.
In addition, the CAR-T therapy (BEAM-201) for the treatment of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) with in vitro edited T cells will submit an IND application in the second half of this year.
Beam will also initiate the IND study of the first in vivo base editing therapy BEAM-301 this year .
The base editing component is delivered to the liver in the form of mRNA through LNP to repair glycogen storage disease type Ia (GSDIa) The R83C point mutation of the G6PC gene.
The R&D pipelines in the above picture are all Beam’s own pipelines, excluding the project in cooperation with Pfizer.
In addition, in July 2021, Beam also reached a 5-year cooperation with Apellis Pharmaceuticals to jointly develop base editing therapies for complement -driven genetic diseases. driving eye, liver and brain disease .
Beam will receive an upfront payment of $50 million from Apellis and subsequent annual payments of $25 million.
Pfizer partners with Beam to advance in vivo base editing therapy
(source:internet, reference only)