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        CRISPR-based gene editing company Mammoth Biosciences and biopharmaceutical company Regeneron Pharmaceuticals have partnered to develop in vivo CRISPR-based gene editing therapies. Mammoth will receive a total of USD 100 million upfront, including USD 95 million in equity, from Regeneron, with a potential additional USD 370 million in milestone payments.
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        The collaboration will combine Regeneron's adeno-associated viral vectors (AAV)-based delivery technology with Mammoth&rsquo;s gene editing systems to enhance the delivery of gene editing treatments to tissues beyond the liver. Regeneron will lead the development and commercialization of the drug targets; Regeneron&rsquo;s access to Mammoth&rsquo;s technology will be limited to the targets specified in the agreement. However, the partnership can be extended for another two years upon payment of the research extension fee.
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  <p>
    <a href="https://www.globenewswire.com/en/news-release/2024/04/25/2869495/0/en/Regeneron-and-Mammoth-Biosciences-Collaborate-to-Pursue-Next-Generation-CRISPR-Based-Gene-Editing-for-Multiple-Diseases.html">
      Press release
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  <p>
    <a href="https://www.fiercebiotech.com/biotech/regeneron-signs-mammoth-gene-editing-deal-worth-100m-upfront">
      Fierce Biotech
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  <p>
    <a href="https://www.biospace.com/article/regeneron-pays-mammoth-100m-to-access-crispr-enzymes-for-aav-delivery/">
      BioSpace
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Human Gene Editing

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

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 <p>Gene editing firm Mammoth Biosciences has teamed up with biopharma company Regeneron Pharmaceuticals to create in vivo (within the body) CRISPR-based gene editing treatments. Regeneron will provide Mammoth with an initial $100 million, including $95 million in equity, and possibly a further $370 million based on accomplishments.</p>
 <p>This partnership will merge Regeneron's Adeno-Associated Viral (AAV) vectors - a delivery technology - and Mammoth’s gene editing systems, improving the delivery of gene editing therapies beyond the liver. Regeneron will take the reign on developing and commercializing the drug targets while its use of Mammoth’s technology will be confined to specified targets in the deal. However, by paying a research extension fee, the collaboration can be extended for an additional two years.</p>
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Mammoth Biosciences signs USD 100 million gene editing deal with Regeneron

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 <p>NYU Langone Health reported a successful surgical procedure involving a heart pump implantation and a genetically modified pig kidney transplant on a 54-year-old woman. The woman had been battling heart failure and end-stage kidney disease and was previously not a candidate for conventional heart or kidney transplants. The procedure was carried out in two separate surgeries over 12 days.</p>
 <p>The initial stage of the procedure involved the insertion of a left ventricular assist device (LVAD), a type of heart pump. The second stage consisted of a xenotransplant of a pig kidney. The kidney used for the transplant came from United Therapeutics and was a genetically modified version dubbed "UThymoKidney", possessing an attached pig thymus.</p>
 
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NYU Langone Health surgeons perform first combined heart pump and pig kidney transplant

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 <p>Seamless Therapeutics, a gene editing biotech company, has secured USD 25 million in a seed funding round. The round was led by Forbion and Wellington Partners.</p>
 <p>The investment will be used to progress the company's unique technology of programmable recombinases, which can precisely edit a 138-kilobase fragment. This capability opens possibilities for gene editing in non-dividing cell types. The company aims to develop a variety of product candidates that may prove beneficial for various human diseases, regardless of specific genetic alteration.</p>
 
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Seamless Therapeutics raises USD 25 million in seed funding

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 <p>Profluent Bio, an AI-first protein design company, has revealed the world's first AI-based open-source gene editor, OpenCRISPR-1. This new product is designed to precisely edit the human genome with customizable gene editors and can be licensed for both ethical research and commercial use. </p>
 <p>OpenCRISPR-1 is an AI-developed gene editor, designed with a structure that is analogous to the Cas9 protein and guide RNA. It is made by leveraging Profluent's extensive language models (LLMs) and has the capability to generate numerous varieties of CRISPR-like proteins that do not naturally occur.</p>
 <p>Profluent Bio combines state-of-the-art AI technology with thorough experimental validation in order to pioneer advancements in protein design, focusing particularly on CRISPR gene editing technologies. </p>

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Profluent debuts first-ever open-sourced AI gene editor

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 <p>CRISPR-based gene editing firm SNIPR Biome has raised USD 5.48 million from global non-profit group Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X).</p>
 <p>The funds will support the Phase Ib/IIa clinical trial for SNIPR001, a CRISPR-enhanced phage therapy aiming to prevent E. coli bloodstream infections in hematological cancer patients receiving hematopoietic stem-cell transplants. The trial, scheduled to begin later this year, will include approximately 24 patients from 10 locations in Europe and the U.S.</p>
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SNIPR Biome secures USD 5.5 million from CARB-X to advance clinical progress

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 <p>North Carolina-based gene editing company Precision BioSciences has taken control over three therapeutic programs from Prevail Therapeutics, a subsidiary of Eli Lilly and Company. This follows Prevail's decision to end the collaboration that was started in <a href="https://investor.lilly.com/news-releases/news-release-details/lilly-and-precision-biosciences-announce-genome-editing-research"> November 2020 </a>.</p>
 <p>The partnership originally intended to develop Precision’s ARCUS nucleases for the research and development of potential in vivo therapies for genetic disorders, including Duchenne muscular dystrophy. In light of promising data from these programs, Precision BioSciences is looking into continuing the development of these programs either independently or in partnership. </p>
 
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Precision BioSciences regains rights for gene editing programs from Prevail Therapeutics following partnership closure

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 <p>Chinese medical researchers from Xijing Hospital of the Air Force Medical University have successfully transplanted a genetically modified pig kidney into a brain-dead person. This accomplishment follows a successful pig liver transplantation earlier in the year. </p>
 <p>Dr. Qin Weijun led the surgical team performing the kidney transplant, which was placed in the patient's right iliac fossa. The operation took over six hours and resulted in the immediate function of the kidney, as demonstrated by the production of urine. There was no evidence of hyperacute rejection. Research suggests that this technique could provide new solutions for end-stage renal disease and help to address organ shortages. This success largely owes to advancements in gene editing technology and immunology.</p>
 
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Chinese researchers successfully transplant genetically modified pig kidney to human body

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 <p>SOHM, a biopharmaceutical company, has developed a partnership with Coastar Therapeutics to refine genome editing technology. This partnership will utilize SOHM's ABBIE (A Binding Based Integrase Enzyme) genome-editing tool in conjunction with Coastar's red blood cell membrane-derived carriers.</p>
 <p>The objective of their collaboration is to augment the effectiveness of genome editing through improved delivery of therapeutic cargo. Their aim is to achieve greater precision by targeting specific cells, and to establish a flexible, scalable platform for efficient genome editing applicable to various cell types and tissues.</p>
 <p>SOHM's work is focused on researching and developing gene editing technologies for their utilization in therapeutic applications, concentrating on precision medicine. Its product portfolio has been expanded to include gene-editing technologies such as ABBIE, a gene-editing platform procured in September 2023, thus strengthening its proficiency in the biotech sector.</p>
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SOHM, Inc. partners with Coastar Therapeutics to advance genome-editing technologies

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 <p>Sangamo Therapeutics, a gene editing company from California, has inked a securities purchase and sale deal with institutional investors. Under the agreement, approximately 24.7 million shares of common stock and pre-funded warrants to acquire around 3.8 million shares of common stock will be purchased and sold. This deal also includes warrants to purchase up to 28.5 million shares of common stock.</p>
 <p>Each common stock and its corresponding warrant are priced at USD 0.84. Each pre-funded warrant plus an accompanying warrant carry a price of USD 0.83. These warrants can be exercised six months after issue and are valid for five and a half years, with an exercise price of USD 1.00 per share. The close of the offering is anticipated on March 26, 2024.</p>
 <p>The funds raised from this offering will be used for various purposes. Primarily, they will be employed to develop a genomic medicines pipeline focused on neurology, promote the AAV capsid delivery technology, and cover other general corporate needs.</p>
 
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Sangamo Therapeutics announces USD 24 million in registered direct offering to develop neurology pipeline

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 <p>Profluent, an AI-based protein engineering company, has raised an additional USD 35 million in its latest venture funding round. This round was led by Spark Capital and saw participation from existing investors Insight Partners, Air Street Capital, and several angel investors. This additional funding followed the company's seed round in August 2022, which raised USD 9 million, bringing the total funds raised to USD 44 million.</p>
 <p>These funds are set to propel the company into its next growth phase. This includes enhancing its unique proprietary LLMs and datasets, broadening wet lab capabilities, and improving the efficiency of CRISPR gene-editing systems.</p>
 <p>Founded recently in 2022, Profluent uses GenAI models to understand the genetic code's language. This know-how aids in designing and validating new and functional proteins to make advancements in biomedicine. Their key aim lies in designing unique, functional proteins that can address limitations in the existing industry.</p>
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