<div>
  <p>
  </p>
  <ul>
    <li>
      <p>
        Massachusetts-based biotech company KSQ Therapeutics has received Investigational New Drug (IND) clearance from the FDA to conduct a Phase I/II clinical trial for KSQ-001EX in collaboration with The University of Texas MD Anderson Cancer Center and the Cell Therapy Manufacturing Center for the treatment of solid tumors.
      </p>
    </li>
    <li>
      <p>
        KSQ-001EX is an engineered tumor-infiltrating lymphocyte (eTIL) program that involves the inactivation of the SOCS1 gene in TILs using CRISPR/Cas9 gene editing. The therapy reportedly has the potential to transform solid tumor treatment, having shown efficacy in preclinical studies against PD-1-resistant tumors and enhanced memory formation and persistence.
      </p>
    </li>
    <li>
      <p>
        KSQ Therapeutics uses its proprietary CRISPRomics discovery platform to systematically decode the genome and identify optimal gene targets for oncology and autoimmune diseases. KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies.
      </p>
    </li>
  </ul>
  <p>
  </p>
</div>


<div>
  <p>
    <a href="https://ksqtx.com/news-events/ksq-therapeutics-announces-fda-clearance-of-ind-application-for-ksq-001ex-a-crispr-cas9-engineered-tumor-infiltrating-lymphocyte-etil-therapy/">
      Press release
    </a>
  </p>
  <p>
  </p>
</div>


Human Gene Editing

<div>
    <p>Massachusetts-based biotech company KSQ Therapeutics has received Investigational New Drug (IND) clearance from the FDA for Phase I/II clinical trial of KSQ-001EX, a therapy for solid tumors. This trial is in collaboration with The University of Texas MD Anderson Cancer Center and the Cell Therapy Manufacturing Center.</p>
    <p>KSQ-001EX is an eTIL program involving the inactivation of the SOCS1 gene in TILs using CRISPR/Cas9 gene editing. It has shown efficacy against PD-1-resistant tumors in preclinical studies, with enhanced memory formation and persistence.</p>
    <p>KSQ Therapeutics utilizes its proprietary CRISPRomics discovery platform to identify gene targets for oncology and autoimmune diseases. They are developing a pipeline of drug candidates for the treatment of cancer, including targeted therapies, adoptive cell therapies, and immunotherapies.</p>
</div>

KSQ Therapeutics announces FDA clearance of IND for KSQ-001EX

<div>
 <ul>
  <li><p>CRISPR Therapeutics has laid off around 50 employees, about 10% of its workforce, due to challenging conditions in the biotech market. This comes after positive reception of its sickle-cell therapy and endorsement by UK regulators.</p></li>
  <li><p>The company is seeking partnership opportunities for its pipeline assets, focusing on in-vivo editing and immuno-oncology. It maintains a cash reserve of USD 1.74 billion and has reduced R&D expenses by 22% compared to last year.</p></li>
 </ul>
</div>

CRISPR Therapeutics lays off 10% of employees due to market challenges

<div>
<ul>
<li><p>Homology Medicines, a gene-editing biotech based in Massachusetts, has announced a merger agreement with drug discovery company Q32 Bio. The terms of the merger have not been disclosed.</p></li>
<li><p>After the merger, 75% of the combined company will be owned by Q32 Bio stakeholders and the remaining 25% will be owned by Homology's stakeholders. The merged company will operate as Q32 Bio and will be listed on the Nasdaq as "QTTB." Jodie Morrison, the current CEO of Q32 Bio, will be the head of the company.</p></li>
<li><p>The USD 42 million gained from a private placement agreement by Q32 Bio will be used for the clinical development of their two treatment candidates for autoimmune and inflammatory diseases. The company will have around USD 115 million in cash at closure and is expected to have enough funding until mid-2026.</p></li>
</ul>
</div>

Homology Medicines merges with Q32 Bio to advance clinical development

ANALYST_QUICK_TAKE

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

The gene editing therapy, exagamglogene autotemcel (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, has received authorization from the UK's Medicines and Healthcare Products Regulatory Agency for the treatment of sickle cell disease and beta-thalassemia in patients aged 12 and older. The drug will be initially available for up to 2,000 patients in the UK. The price of the treatment has not been disclosed. <p><b>Analyst QuickTake:</b> Initiated as part of a collaboration between Vertex and CRISPR in 2015, Casgevy marks the first CRISPR-based gene editing treatment to receive market authorization for two blood disorders. The drug is also nearing its market entry in the US, with the FDA’s decision expected on December 8, 2023 (exa-cel for sickle cell disease patients) and March 30, 2024 (for beta-thalassemia patients). Just <a href="https://sp-edge.com/updates/23331"> last month </a> , exa-cel received positive remarks from the FDA’s advisory team indicating a potentially positive decision from the FDA.</p>

Vertex-CRISPR’s gene editing drug Casgevy gets world-first approval in UK

<div>
  <ul>
    <li>
      <p>California-based gene editing biotech company Graphite Bio has merged with biopharmaceutical firm LENZ Therapeutics in an all-stock transaction.</p>
    </li>
    <li>
      <p>The newly combined entity will continue trading on Nasdaq under the ticker “LENZ,” and will focus on advancing treatments for presbyopia, a vision-related condition. The transaction and the acquisition cost has not been disclosed.</p>
    </li>
    <li>
      <p>As part of the merger, Graphite Bio has also signed an agreement to secure private investment in public equity (PIPE) funding worth USD 53.5 million led by LENZ's current and new investors. The merger is expected to close in the first quarter of 2024, and the combined company is projected to have cash or equivalents of approximately USD 225 million due to the additional funds expected from the merger and concurrent financing.</p>
    </li>
    <li>
      <p>The new firm will continue to expand LENZ's lead programmes, LNZ100 and LNZ101, for the treatment of presbyopia, following the completion of ongoing Phase III trials, NDA submission, and subsequent FDA approval. The company is also looking to enhance its product candidate and infrastructure, boosted by the merger and funding.</p>
    </li>
  </ul>
</div>

Graphite Bio merges with LENZ Therapeutics to advance treatment of eye condition

DISRUPTOR_PRESENT

<div>
 
 <ul>
  <li><p>Massachusetts-based gene-editing biotech company Intellia Therapeutics has received the European Commission's (EC) orphan drug designation for its in-vivo CRISPR treatment, NTLA-2002, for the treatment of hereditary angioedema (HAE). The drug also received Investigational New Drug (IND) clearance from the <a href="https://sp-edge.com/updates/22894"> FDA </a> in October 2023.</p></li>
  <li><p>Since it has been granted “orphan drug” status in the European Union, the treatment receives regulatory and financial benefits, along with marketing exclusivity aimed to support the development of the treatment.</p></li>
 </ul>
 
</div> 
Massachusetts-based gene-editing biotech company, Intellia Therapeutics, has received orphan drug designation from the European Commission for its in-vivo CRISPR treatment, NTLA-2002, for hereditary angioedema (HAE). The drug has also received Investigational New Drug clearance from the FDA. The orphan drug status in the European Union grants regulatory and financial benefits and marketing exclusivity to support treatment development.

Intellia Therapeutics receives Orphan Drug Designation for NTLA-2002

<div>
    <ul>
        <li>
            <p>Nucleic acid delivery company Genevant Sciences and Novo Nordisk have entered a collaboration and non-exclusive license agreement to develop a gene-editing treatment for hemophilia A, utilizing Genevant's lipid nanoparticle (LNP) technology and mRNA-based megaTAL technology.</p>
        </li>
        <li>
            <p>The partnership will build on a previously shared R&amp;D effort in hemophilia A by Novo Nordisk and 2seventy bio, previously signed between Genevant and 2seventy bio, which was later transferred to Novo Nordisk in January 2022. The Genevant-Nordisk partnership agreement follows the same terms.</p>
        </li>
        <li>
            <p>Genevant Sciences is a company that leverages its extensive experience and expertise in nucleic acid drug delivery and development to create new medicines. The company possesses an extensive LNP patent portfolio and has decades of experience in nucleic acid drug delivery and development. This platform enables a wide range of RNA-based applications, including vaccines, therapeutic protein production, and gene editing.</p>
        </li>
    </ul>
</div>

Genevant Sciences and Novo Nordisk partners to develop treatment for hemophilia A

<div>
 <ul>
  <li><p>Gene editing biotech company Cellectis and AstraZeneca have partnered to accelerate the development of gene editing therapeutics, with a focus on oncology, immunology, and rare diseases.</p></li>
  <li><p>Cellectis will receive an initial payment of USD 105 million, with a USD 25 million upfront fee and USD 80 million in equity investment. AstraZeneca is expected to make an additional USD 140 million equity investment in early 2024.</p></li>
  <li><p>The partnership will target 25 exclusive genetic targets, potentially leading to 10 novel treatments. These treatments may be eligible for option fees, milestone payments, and product development payments ranging from USD 70 million to USD 220 million.</p></li>
  <li><p>The partnership also aims to expand AstraZeneca's cell therapy offerings for cancer and advance genomic medicines for rare diseases.</p></li>
 </ul>
</div>

Cellectis signs investment agreement with AstraZeneca worth up to USD 245 million to develop next-gen therapeutics

<div>
 <ul>
  <li><p>CRISPR gene editing company Verve Therapeutics is collaborating with Eli Lilly to develop gene editing therapies for cardiovascular diseases. Eli Lilly acquired rights to Verve's products from Beam Therapeutics, including programs targeting PCSK9, ANGPTL3, and an undisclosed cardiovascular target. Verve will retain control of development and commercialization outside the US, while joint development and commercialization will occur in the US. The agreement also includes an option for Lilly to co-fund and share profits for the undisclosed disease target. As part of the acquisition, Beam will receive a USD 200 million upfront payment and USD 50 million equity investment from Lilly.</p></li>
 </ul>
</div>

Verve expands collaboration with Eli Lilly following Lilly’s acquisition of Verve’s product rights from Beam Therapeutics

<div>
 <ul>
  <li><p>Vertex Pharmaceuticals and CRISPR Therapeutics's gene editing therapy, exagamglogene autotemcel (exa-cel), for the treatment of sickle cell disease in patients aged 12 and older, has successfully passed through the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee, reportedly the last step before the FDA's final decision. The FDA is expected to make a decision on the therapy by December 8.</p></li>
  <li><p>The therapy, which aims to restore normal red blood cell function and alleviate symptoms, has proved to be safe and effective in preventing painful episodes associated with the disease. However, there are concerns about potential long-term safety issues.</p></li>
  <li><p>Exa-cel is the first therapy developed by the duo, which entered into a partnership in October 2015. If approved by the FDA, it will be the first gene editing therapy available for SCD patients.</p></li>
 </ul> 
</div>
Summary: Vertex Pharmaceuticals and CRISPR Therapeutics have developed exagamglogene autotemcel (exa-cel), a gene editing therapy for sickle cell disease. It has passed the FDA's advisory committee and a decision is expected by December 8. The therapy aims to restore normal red blood cell function and alleviate symptoms, effectively preventing painful episodes. However, concerns about its long-term safety remain. If approved, it will be the first gene editing therapy for SCD patients.

All Updates