<div>
  <ul>
    <li>
      <p>
        UK-based Orchard Therapeutics has received FDA approval for gene therapy &quot;Lenmeldy&quot; to treat children suffering from metachromatic leukodystrophy (MLD), a rare genetic disease. The therapy's price has not been disclosed.
      </p>
    </li>
    <li>
      <p>
        Lenmeldy is a one-time therapy for children experiencing certain stages of MLD and uses the patient's own hematopoietic stem cells. The FDA reported that Lenmeldy substantially reduced the risk of severe motor impairment or death compared to untreated children. In December 2020, 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 brand name &quot;Libmeldy.&quot;
      </p>
    </li>
  </ul>
  <p>
  </p>
</div>


<div>
  <p>
    <a href="https://www.reuters.com/business/healthcare-pharmaceuticals/us-approves-first-gene-therapy-rare-genetic-disease-2024-03-18/">
      Press release
    </a>
  </p>
  <p>
    <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-children-metachromatic-leukodystrophy">
      FDA&nbsp;
    </a>
  </p>
</div>


Cell & Gene Therapy

<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>
 
</div>

Orchard Therapeutics’ Lenmeldy receives FDA approval; becomes first-of-its kind treatment for children with genetic disorder

Using real-time data to create dynamic virtual representations of the real world

Digital Twin

ANALYST_QUICK_TAKE

<div>
 
 <p>Italian digital twin technology firm, EKORE, has garnered EUR 1.3 million (~ USD 1 million) in a seed investment round led by SOM S.p.A. and Habismart, marking the largest digital twin funding in Italy in 2024.</p>
 <p>These funds will be leveraged to enhance EKORE's platform and algorithms, extend its team, and accelerate commercial expansion.</p>
 <p>Providing a cloud-based service, EKORE equips buildings with a "digital brain," which amalgamates data and 3D Building Information Modeling (BIM) models in one centralized solution. The AI-powered algorithms of the platform contribute to reducing consumption, identifying anomalies, and cutting down carbon emissions in the property sector.</p>
 
</div> <p><b>Analyst QuickTake:<br>
 </b> The past year has seen several digital twin startups in the maintenance and monitoring segment raising funds, with notable investments including <a href="https:<br>
//www.finsmes.com/2024/09/samp-raises-e6m-in-funding.html"> <b>Samp </b> </a> (~ USD 6.3 million), <a href="https:<br>
//sp-edge.com/updates/35470"> <b>Neara </b> </a> (USD 45 million), and <a href="https:<br>
//sp-edge.com/updates/27592"> <b>Tomorrow Things </b> </a> (~ USD 1.6 million ). These startups are innovating across various sectors, from geospatial visualization and industrial asset management to construction and power grid solutions.</p>

EKORE raises EUR 1.3 million (~ USD 1 million) in seed funding to strengthen platform

Functional Nutrition

<div>
 
 <p>Culina Health, a digital nutrition platform, has secured USD 7.9 million in a Series A funding round. The initiative was led by Healthworx and saw participation from several other firms including Rethink Impact, Collab Capital, Collide Capital, Vamos Ventures, Tensility Venture Partners, Cake Ventures, and GW Ventures. The investment will primarily aid in the expansion of offerings to cater to dietitians and patients. It will also assist in the implementation of new AI platforms that can enhance care efficiency. Additionally, the funding will support bolstering the firm's leadership through new hires.</p>
 
</div>

Culina Health raises USD 7.9 million in Series A funding to expand offerings and expand team

<div>
 
 <p>ViGeneron has obtained US FDA approval for the Investigational New Drug (IND) application for VG801, a gene therapy for Stargardt disease and other related retinal dystrophies. This clearance enables the start of Phase I/II clinical trials to assess the therapy's safety and effectiveness.</p>
 <p>VG801 utilizes ViGeneron's patented reconstitution via mRNA trans-splicing (REVeRT) dual AAV vector technology to transport the whole ABCA4 gene. The treatment involves two distinct vectors, each containing half of the ABCA4 coding sequence. These vectors then merge to create a fully functional protein. This method aims to overcome the genetic primary cause of Stargardt disease. It is rumored to have shown significant and enduring performance in non-human primate retinas, indicating its potential effectiveness.</p>
 <p>ViGeneron designs gene therapies targeting eye diseases utilizing its three exclusive technology platforms–the vgAAV vector platform, designed to address the limited capability of existing adeno-associated virus (AAV) based gene therapies, REVeRT which efficiently delivers large genes, and AAV Transactivation that manages multiple genes in vivo. VG901, the company's prime candidate, is currently undergoing clinical trials for treating CNGA1-associated retinitis pigmentosa.</p>
 
</div>

ViGeneron receives IND clearance for VG801 gene therapy

Technology providers that enhance satellite operations

Next-gen Satellites

PROMINENT_CATEGORY

<div>
 <p>Reflex Aerospace, a German satellite technology firm, sent its first commercial satellite, SIGI, to Vandenberg Space Force Base. The satellite, which weighs 109 kg and was built for Media Broadcast Satellite (MBS), is set to launch on SpaceX's Transporter-12 Rideshare mission in January 2025.</p>
 <p>SIGI, which was completed within 14 months of receiving the order, is part of a versatile product line of dual-use satellite platforms suitable for payloads between 100 kg and 500 kg. This represents the fulfilment of the first of four commercial contracts the company has signed.</p>
 
</div> <p><b>Analyst QuickTake:<br>
 </b> Reflex Aerospace is actively pursuing the expansion of dual-use satellite platforms, which serve both the commercial and defense sectors. Recent developments, including a <a href="https:<br>
//sp-edge.com/updates/36478"> November 2024 </a> partnership with Sidus Space, indicate efforts to secure funding from diverse public and private sources.</p>

Reflex Aerospace ships first commercial satellite SIGI

Technology to explore opportunities beyond Earth

Space Travel and Exploration Tech

INCUMBENTS_PRESENT

<div>
 <p>California-based developer, Vast Space, has teamed up with SpaceX for two Crew Dragon private astronaut missions (PAMs) to the International Space Station (ISS). This is dependent on approval from NASA.</p>
 <p>This collaboration aims to grow Vast’s role in human spaceflight operations and potentially place it as a potential successor to the ISS. The plan includes the launch of Haven-1 module and at least one Crew Dragon mission, and potential astronaut flight missions with foreign space agencies including the Czech Republic.</p>
 <p>Previously, NASA has given Axiom Space exclusive rights to four PAMs, with little competition from other providers. Axiom has already completed three missions and is gearing up for the fourth, <a href="https://sp-edge.com/updates/37059"> Ax-4 </a>, all facilitated through SpaceX’s Crew Dragon spacecraft. Vast might have to compete with Axiom for future PAM opportunities.</p>
 
</div>

Vast partners with SpaceX for two private astronaut missions to ISS

Reinforcing recycling, rerouting refuse, and reducing wasteflows

Waste Recovery & Management Tech

Carbios appoints Philippe Pouletty as interim CEO amid plant delay

Quantum information processing is slowly becoming a reality, ushering in a new technological era.

Quantum Computing

<div>
 <p>BlueQubit, a quantum computing platform, has successfully raised 10 million USD in seed funding in a round led by Nyca Partners. Other participants in this funding round included Restive, Chaac Ventures, NKM Capital, Presto Tech Horizons, BigStory, Untapped Ventures, Formula VC, and Granatus.</p>
 <p>This funding will be utilized to expand BlueQubit's software stack and to strengthen their partnerships with businesses that are interested in exploring quantum solutions.</p>
 <p>The company's platform, dubbed QSaaS, leverages both classical GPUs and quantum processors. This enables quantum process emulation that is up to 100 times faster than other available options. BlueQubit's platform targets industries such as finance, pharmaceuticals, and material science, aiming to solve problems that traditional computers struggle to handle.</p>
 
</div>

BlueQubit raises USD 10 million in seed funding to develop quantum platform

Reinventing gene therapies with next-generation technologies

Human Gene Editing

<div>
 <p>The Food and Drug Administration (FDA) has approved an Investigational New Drug (IND) application from Arbor Biotechnologies for ABO-101. This is a gene editing therapy designed to treat primary hyperoxaluria type one (PH1), a condition that leads to kidney stones and end-stage kidney disease. Arbor will now begin the redePHine Phase I/II study, which will test the safety, tolerability, and preliminary efficacy of ABO-101 in adults and children with PH1.</p>
 <p>ABO-101 is a liver dirigible therapeutic. It uses a lipid nanoparticle to transport a Type V CRISPR Cas12i2 nuclease-expressing messenger RNA. The therapy's objective is to decrease HAO1 gene expression in the liver, thus reducing oxalate production, which complicates PH1.</p>
 <p>Preclinical data demonstrates that ABO-101 results in specific, robust in vivo editing of the HAO1 gene and decreases urinary oxalate, as per PH1 disease models. Arbor's initiation of clinical trials for this venture marks a significant step for the company.</p>
 </div>

Arbor Biotechnologies receives FDA clearance for ABO-101 IND application

Leveraging personal data for custom-fit diagnosis and treatment

Precision Medicine

<div>
  <p>Personalis, a company that provides cancer testing solutions, has secured a USD 50 million investment from Merck. With this deal, Merck will obtain around 14 million shares of Personalis' common stock at a rate of USD 3.56 per share, giving them a 16.5% ownership stake.</p>
  <p>Furthermore, Personalis has expanded its partnership with Moderna for cancer therapy development using the ImmunoID NeXT Platform. This extension permits Moderna to continue leveraging Personalis’ ImmunoID NeXT Platform and technology for V940/mRNA-4157, a new individualized neoantigen therapy that Merck and Moderna are collaboratively developing.</p>
</div>

Personalis partners with Merck and Moderna for cancer therapy development and investment

<div>
 <p>Oncology data and analytics provider, COTA, has teamed up with precision oncology company, Guardant Health. Their combined healthcare data resources will be utilised for cancer therapy development.</p>
 <p>By integrating COTA's electronic health record data from academic and community care centers with Guardant's clinicogenomic testing data, the partnership enables researchers to study correlations between molecular biomarkers, treatment decisions, and clinical outcomes. This way, they can optimize drug development across different patient demographics. The partnership will place a specific focus on expanding data coverage in solid tumors, such as lung, breast, and colorectal cancers.</p>
 
</div>

All Updates

Orchard Therapeutics’ Lenmeldy receives FDA approval; becomes first-of-its kind treatment for children with genetic disorder

EKORE raises EUR 1.3 million (~ USD 1 million) in seed funding to strengthen platform

Culina Health raises USD 7.9 million in Series A funding to expand offerings and expand team

ViGeneron receives IND clearance for VG801 gene therapy

Reflex Aerospace ships first commercial satellite SIGI

Vast partners with SpaceX for two private astronaut missions to ISS

Carbios appoints Philippe Pouletty as interim CEO amid plant delay

BlueQubit raises USD 10 million in seed funding to develop quantum platform

Arbor Biotechnologies receives FDA clearance for ABO-101 IND application

Personalis partners with Merck and Moderna for cancer therapy development and investment

COTA partners with Guardant Health to develop clinicogenomic data solutions for cancer research

Cell & Gene Therapy

Orchard Therapeutics’ Lenmeldy receives FDA approval; becomes first-of-its kind treatment for children with genetic disorder

Contact us