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        Allogeneic CAR T therapy developer Allogene Therapeutics has announced the price for an underwritten offering of 37.9 million common shares at a rate of USD 2.90 per share. Mutual funds, large institutional investors, healthcare specialists, and certain members of Allogene's board and executive management team will also participate in the offering.
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        The offering, which is expected to close on or about May 16, 2024, is expected to raise USD 110 million in overall gross proceeds.&nbsp;
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        Allogene focuses on offering off-the-shelf CAR T therapies. It develops allogeneic chimeric antigen receptor T-cell (AlloCAR T) products to treat cancers and autoimmune diseases. The company leverages multiplex gene-engineering and gene-editing technologies to offer off-the-shelf T-cell therapies designed to be more scalable and cost-effective than autologous counterparts.&nbsp;
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    <a href="https://www.globenewswire.com/en/news-release/2024/05/13/2880920/0/en/Allogene-Therapeutics-Announces-Pricing-of-110-million-Offering-of-Common-Stock.html">
      Press release
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Human Gene Editing

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 <p>Allogene Therapeutics, a company specializing in allogeneic CAR T therapy, has announced an underwritten offering of 37.9 million common shares at USD 2.90 each. This offering, which includes participation from mutual funds, large institutional investors, healthcare specialists, and some of Allogene's board and executive management team, is set to close around May 16, 2024, and is anticipated to generate an overall gross profit of USD 110 million.&nbsp;</p>
 <p>The company is known for developing allogeneic chimeric antigen receptor T-cell (AlloCAR T) products, aiming to treat cancers and autoimmune diseases. By exploiting multiplex gene-engineering and gene-editing technologies, Allogene offers more scalable and cost-effective off-the-shelf T-cell therapies compared to autologous counterparts.&nbsp;</p>
 
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Allogene Therapeutics to raise USD 110 million in underwritten offering

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 <p>Arbor Biotechnologies, an AI-powered gene-editing company based in Massachusetts, has acquired gene-editing technology provider Serendipity Biosciences. This acquisition aims to enhance the development of novel gene-editing treatments and to expand Arbor's genomic medicine portfolio. However, the financial aspects of the acquisitions have not been disclosed.</p>
 <p>The gene-editing assets of Serendipity, including genome editing that utilize RNA-guided endonuclease proteins like Fanzor, IsrB, and others, will assist in the discovery of new therapeutic applications for Arbor’s genomic medicine portfolio. Arbor Biotechnologies plans to leverage these technologies from Serendipity to enhance and diversify its existing nuclease technologies post-acquisition. This will potentially enable therapeutic applications involving reverse transcriptase (RT) editing and the in vivo insertion of exons or entire genes.</p>
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Arbor Biotechnologies acquires Serendipity Biosciences to expand gene-editing capabilities

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 <p>iECURE has been granted Fast Track designation by the FDA for its project, ECUR-506, intended to tackle neonatal onset Ornithine Transcarbamylase (OTC) deficiency. This status allows iECURE to have frequent meetings with the FDA, aiming to speed up the development and approval process of the remedy.</p>
 <p>ECUR-506 is created through in vivo gene insertion, utilizing Precision BioSciences’ ARCUS platform. The treatment comprises of two vectors: An ARCUS nuclease vector to aim at gene editing in the PCSK9 gene locus, and a donor vector to insert an operational OTC gene. The intended function of this treatment is to offer a possible route to the lengthy expression of a healthy gene. Before this, the FDA had given the treatment Rare Pediatric Disease and Orphan Drug designations, and it also received Orphan designation from the European Commission for the cure of OTC deficiency.</p>

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iECURE receives FDA Fast Track designation for Gene Insertion program 'ECUR-506'

PROMINENT_CATEGORY

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 <p>The Children's Hospital of Philadelphia (CHOP) and the Scheie Eye Institute at the University of Pennsylvania carried out a trial for a CRISPR-Cas9 gene editing treatment aimed at congenital blindness. The treatment, known as EDIT-101, was developed by Editas Medicine and relies on in vivo CRISPR/Cas9 genome editing to treat blindness.</p>
 <p>The trial required participants to receive a single injection of EDIT-101 in one eye. Their condition was then periodically evaluated over a period of at least six months. Key measures of effectiveness of the treatment were set out within four criteria. From their findings, the researchers reported that out of 11 participants, all showed improvements as per at least one of the criteria. Six of these participants showed enhancements according to more than one criterion. Moreover, six participants demonstrated noticeable improvements in their cone-mediated vision, as measured by FSTs. Out of these, five showed improvement in at least one other outcome. A significant note from this study is that there were no reported treatment-related adverse effects or toxicities.</p>
 
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Editas’ CRISPR treatment succeeds in CHOPS trial for improving congenital blindness

INCUMBENTS_PRESENT

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 <p>Gene-editing company, Cellectis, has received an additional $140 million in equity investment from AstraZeneca, raising AstraZeneca's stake in Cellectis to 44%. This transaction forms part of the ongoing collaboration between the two firms that began in <a href="https://sp-edge.com/updates/23389"> November 2023 </a>.</p>
 
<p>The investment will further support their joint development of up to ten new cell and gene therapy products. The main focus areas will be oncology, immunology, and rare diseases which have large unmet medical needs. Cellectis stands to gain an option fee and milestone payments ranging from $70 million to $220 million per program.</p>
   
<p>AstraZeneca will have worldwide exclusive license options for the developed candidate products through this collaboration.</p>

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Cellectis secures additional equity investment of USD 140 million from AstraZeneca

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    <p>MedStar Washington Hospital Center has enrolled the initial heart patient from the US in the Phase III MAGNITUDE clinical trial of NTLA-2001. This clinical trial is led by Intellia Therapeutics and focuses on in vivo CRISPR-based therapy for patients who are suffering from transthyretin amyloidosis with cardiomyopathy (ATTR-CM).</p>
    <p>NTLA-2001 involves a single-use, intravenous gene editing therapy that employs CRISPR/Cas9 technology. This technology focuses on preventing the formation of abnormal transthyretin (TTR) protein. This protein leads to ATTR-CM, a condition wherein the TTR protein amasses in the nerves and heart, often leading to heart failure. The preliminary results of the Phase I and II clinical trials suggest that NTLA-2001 can lead to a substantial reduction in TTR protein levels.</p>
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MedStar Health joins Intellia’s Phase III trial of CRISPR therapy for ATTR-CM

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 <p>Gene editing company, Poseida Therapeutics, has teamed up with Xyphos Biosciences, a subsidiary of Astellas Pharma, to develop two allogeneic cell therapies for solid tumors. Poseida will receive an upfront payment of USD 50 million from Astellas, with potential for an additional USD 550 million based on the fulfillment of certain objectives.</p>
 <p>This collaboration will combine Poseida's allogeneic CAR T platform with Xyphos' ACCEL technology. The outcome of this integration will be a CAR T construct and the development of two "convertibleCAR" programs.</p>
 <p>The partnership extends from an existing agreement in <a href="https://sp-edge.com/updates/20759"> August 2023 </a> that was aimed at supporting Poseida’s cancer cell therapy development efforts.</p>
 
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Poseida Therapeutics receives USD 50 million investment from Astellas Pharma

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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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All Updates

Allogene Therapeutics to raise USD 110 million in underwritten offering

Arbor Biotechnologies acquires Serendipity Biosciences to expand gene-editing capabilities

iECURE receives FDA Fast Track designation for Gene Insertion program 'ECUR-506'

Editas’ CRISPR treatment succeeds in CHOPS trial for improving congenital blindness

Cellectis secures additional equity investment of USD 140 million from AstraZeneca

MedStar Health joins Intellia’s Phase III trial of CRISPR therapy for ATTR-CM

Poseida Therapeutics receives USD 50 million investment from Astellas Pharma

Mammoth Biosciences signs USD 100 million gene editing deal with Regeneron

NYU Langone Health surgeons perform first combined heart pump and pig kidney transplant

Seamless Therapeutics raises USD 25 million in seed funding

Human Gene Editing

Allogene Therapeutics to raise USD 110 million in underwritten offering

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