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  </p>
  <ul>
    <li>
      <p>
        Johnson &amp; Johnson&rsquo;s subsidiary, Janssen-Cilag International, has secured a Type II variation approval from the European Commission for CARVYKTI (ciltacabtagene autoleucel or cilta-cel), a one-time infusion therapy for relapsed and refractory multiple myeloma (RRMM) patients who have received at least one prior therapy.
      </p>
    </li>
    <li>
      <p>
        Cilta-cel is a chimeric antigen receptor T-cell (CAR T) therapy that targets B-cell maturation antigen (BCMA), a protein commonly found on myeloma cells, and is approved for treating eligible patients as early as the first relapse. The company claims that the therapy can help lower the risk of RRMM progression or death by 74%.&nbsp;
      </p>
    </li>
    <li>
      <p>
        The drug has been approved in the EU (since May 2022) for patients who have had at least three prior therapies. Earlier this month, the FDA approved it for use on RRMM patients who have received at least one prior therapy.
      </p>
    </li>
  </ul>
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  </p>
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<div>
  <p>
    <a href="https://www.janssen.com/carvyktirv-ciltacabtagene-autoleucel-cilta-cel-first-bcma-targeted-treatment-approved-european">
      Press release
    </a>
  </p>
</div>


Cell & Gene Therapy

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

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 <p>Janssen-Cilag International, a subsidiary of Johnson &amp; Johnson, has gained Type II variation approval from the European Commission for CARVYKTI (ciltacabtagene autoleucel or cilta-cel). This therapy is a one-time infusion for relapsed and refractory multiple myeloma (RRMM) patients who have had at least one previous treatment.</p>
 <p>Cilta-cel is a chimeric antigen receptor T-cell (CAR T) therapy that specifically targets B-cell maturation antigen (BCMA), a protein found frequently on myeloma cells. It is approved for use in eligible patients as soon as the first relapse occurs. According to the company, this therapy can reduce the chances of RRMM progression or death by as much as 74%.</p>
 <p>Since May 2022, the drug has been approved in the EU for patients who have undergone a minimum of three previous treatments. Earlier this month, the FDA also approved it for RRMM patients who have received at least one prior therapy.</p>
 
</div>

Janssen’s cancer drug 'CARVYKTI' expands indication in the EU

<div>
 
 <p>Gene therapy company GeneVentiv Therapeutics has been awarded a USD 2.5 million grant from the National Heart Lung Blood Institute (NHLBI), part of the National Institutes of Health (NIH). The funds were granted through the Direct to Phase II Small Business Innovation Research (SBIR) program. Information regarding the company's valuation and total investments has not been disclosed.</p>
 <p>The grant will be utilized for the research and development of gene therapy for hemophilia A or B. This will include large animal studies, assay development, biodistribution, and toxicology studies.</p>
 <p>GeneVentiv Therapeutics is primarily involved in the development of unique gene therapies for the treatment of hemophilia. Their lead candidate is GENV-HEM, a gene therapy which uses an adeno-associated virus (AAV). The therapy is designed to treat all forms of hemophilia, regardless of whether inhibitors are present. The goal of the treatment is to provide a potential lifetime solution for patients, achievable through a single infusion.</p>
 
</div>

GeneVentiv Therapeutics secures USD 2.5 million SBIR grant to advance gene therapies

<div>
 <p>Obsidian Therapeutics, a gene therapy company, has successfully raised USD 160.5 million in a heavily subscribed Series C funding round. The round was led by new investor Wellington Management and saw participation from other new investors like Foresite Capital, Janus Henderson Investors, Novo Holdings A/S, and Woodline Partners LP. Existing investors such as Atlas Venture, Blue Owl Healthcare Opportunities, Bristol Myers Squibb, Deep Track Capital, among others, also participated in the round.</p>
 <p>The collected funds will be channelled towards the clinical advancement of Obsidian’s primary investigational program, OBX-115. This program is designed to treat patients suffering from metastatic melanoma or non-small-cell lung cancer.&nbsp;</p>
 
</div>

Obsidian Therapeutics raises USD 160.5 million in oversubscribed Series C funding for clinical development of lead candidate 'OBX-115'

<div>
 <p>Modulus Therapeutics, a firm that uses artificial intelligence in cell therapy, has sold its cell therapy platform assets to a cell programming and biosecurity company, Ginkgo Bioworks. The specific financial details of the deal were not disclosed.</p>
 <p>Ginkgo plans to integrate Modulus' CAR and switch receptor designs and libraries into its own services. This incorporation will support Ginkgo's customers in developing and improving the performance of T-cell- and NK-cell-based CAR therapies.</p>
</div>

Modulus Therapeutics sells cell therapy platform to Ginkgo Bioworks for undisclosed amount

Nkarta to raise USD 240 million in underwritten offering to strengthen R&D efforts

<div>
 
 <p>Capstan Therapeutics, a cell therapy company, has secured USD 175 million in an oversubscribed Series B funding round. The round was led by RA Capital Management, with participation from new investors Forbion and Johnson &amp; Johnson Innovation, as well as existing investors Bristol-Myers Squibb, Eli Lilly and Company, and Pfizer Ventures.</p>
 <p>This fresh injection of funds will be used to progress the clinical proof-of-concept of Capstan's leading in vivo CAR T candidate, CPTX2309, for the treatment of autoimmune disorders. Moreover, the newly secured funds will facilitate further development of Capstan’s tLNP pipeline.</p>
 
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Capstan Therapeutics raises USD 175 million in Series B funding

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 <p>Cell therapy firm Fate Therapeutics has revealed its pricing for an underwritten offering of 14.5 million common shares at USD 5.50 each. New and existing institutional investors such as Adage Capital Partners LP. and Boxer Capital are involved. The firm has also disclosed a concurrent private placement of pre-funded warrants to institutional and other accredited investors under Redmile Group, LLC. These fundraising efforts are anticipated to yield approximately USD 100 million in overall gross proceeds. </p>

<p>The proceeds will be channelled into funding clinical trials and non-clinical studies of the company’s product candidates. They'll also cover manufacturing expenses related to product development, support preclinical research and development, and cater to other working capital and corporate needs. </p>

<p>Fate Therapeutics focuses on developing programmed cellular immunotherapies for cancer and immune disorders derived from induced pluripotent stem cells (iPSC). As of March 2024, the company had seven programs underway, with five in Phase I clinical trials.</p>

</div>

Fate Therapeutics raises USD 100 million in underwritten offering and private placement funding

<div>
 <p>Orchard Therapeutics, a UK-based company, has gained FDA approval for its gene therapy, "Lenmeldy", designed to treat children afflicted with metachromatic leukodystrophy (MLD)—a rare genetic disease. The cost of the therapy has not yet been revealed.</p>
 <p>"Lenmeldy" is a single-use therapy suitable for children at certain levels of MLD. It uses the patient's hematopoietic stem cells. According to FDA, Lenmeldy notably decreases the risk of severe motor impairment or death as compared to children who remain untreated. The therapy was approved and marketed in the <a href="https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-receives-ec-approval-libmeldytm-treatment"> European Union </a> under the name "Libmeldy" in December of 2020.</p>
 
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Orchard Therapeutics’ Lenmeldy receives FDA approval; becomes first-of-its kind treatment for children with genetic disorder

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 <p>Bluebird Bio, a gene therapy company, has obtained a five-year loan of USD 175 million from Hercules Capital. The loan comprises an immediate disbursement of USD 75 million at the closing of the deal, two available tranches of USD 25 million each based on commercial milestones, and a possible fourth tranche of USD 50 million at Hercules' decision.</p>
 <p>The company plans to use the loan to fund the commercial launches of their three FDA-approved gene therapies: LYFGENIA, ZYNTEGLO, and SKYSONA. This financing is foreseen to extend the company's cash runway until Q1 2026.&nbsp;</p>
 
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Bluebird Bio secures USD 175 million in debt financing to fuel commercialization

ANALYST_QUICK_TAKE

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 <p>Bristol-Myers Squibb's CAR T therapy, Breyanzi, has been approved by the FDA to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) that have relapsed or become refractory. These are types of non-Hodgkin lymphomas.</p>
 <p>Breyanzi is a one-time treatment that involves an infusion of CAR-positive viable T-cells, which are personalized for each patient. The therapy is designed for patients who have tried at least two prior treatment regimens. Key features of Breyanzi include its ability to create deep and long-lasting responses, its effectiveness in patients who haven't responded to standard care, and its established safety profile.</p>
 
</div> <p><b>Analyst QuickTake:<br>
</b> Breyanzi is not a new name in the market and has been available since the FDA approved it in February 2021 for treating relapsed or refractory large B-cell lymphoma in adults, after two or more lines of systemic therapy. In June 2022, the FDA promoted Breyanzi enabling it to be used after one line of therapy. The drug has a current price of USD 487,477, rising from the original price tag of USD 410,300. The latest approval is a testament to the capabilities and potential of Breyanzi in treating various disease indications.&nbsp;</p>

All Updates