Category Archives: Biotechnology

Remix Therapeutics Attains Near-Term Milestone from Roche Collaboration

PR Newswire

WATERTOWN, Mass., July 10, 2024

The collaborationleverages Remix'sREMasterplatform to discover novel drugs for a diverse range of target classes

Remix is eligible to receive up to $1 billion in milestone payments and royalties

WATERTOWN, Mass., July 10, 2024 /PRNewswire/ -- Remix Therapeutics (Remix), a clinical-stagebiotechnology company developing small molecule therapies to modulate RNA processing and address underlying drivers of disease, today announced that it has attained the near-term milestone as part of its collaboration and license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the discovery and development of small molecule therapeutics that modulate RNA processing using Remix's REMaster drug discovery platform.

"Completing this milestone further demonstrates the power and potential of the REMaster platform, purpose-built to identify novel, druggable RNA processing mechanisms," said Peter Smith, Ph.D., Co-Founder and Chief Executive Officer of Remix. "We are incredibly proud of the research team's accomplishments, which bring us closer towards potentially developing new, small molecule medicines for patients."

Under the terms of the agreement, which was announced in January 2024, Remix received an upfront payment of $30 million in addition to a near-term milestone payment and is eligible for preclinical, clinical, commercial and sales milestones of up to $1 billion and tiered royalties. In exchange,Roche will have exclusive rights to specific targets.Remix will conduct discovery and preclinical activities with Roche, and Roche will be responsible for development and commercialization of any resulting products.

About Remix Therapeutics

Remix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and treat disease. The REMaster technology platform facilitates RNA processing pattern identification, leveraging these learnings to modulate gene expression. Remix's innovative therapeutic approach has the potential to alter the way genes are read from the genome, to correct, enhance, or eliminate the gene message, thereby addressing disease drivers at their origin. For more information visit http://www.remixtx.com

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Remix Therapeutics Attains Near-Term Milestone from Roche Collaboration - Morningstar

My kids ran away with my iPhone the other day while chasing a virtual Pokmon on the other side of the park. I panicked.

I was afraid that they would accidentally drop and break it or have trouble sharing it. I was not worried, though, that they would inadvertently use the phone to disable critical infrastructure, derail a passenger train, or trigger a cyber Pearl Harbor.

When, in 2012, then U.S. Secretary of Defense Leon Panetta referenced the Pearl Harbor disaster in warning about the rise of cyber threats in a world increasingly dependent on internet-linked digital technologies, cybersecurity policies and practices in the public and private sectors were woefully inadequate. The revolution in powerful cyber technologies triggered urgency in building cybersecurity strategies and capabilities.

Cyber threats have not disappeared. The information technology landscape, though, has evolved to equip governments and companies to make remarkable strides in cybersecurity over the past decade, to the point that even children freely use powerful computers with 24/7 high-speed internet connections.

The emergence of improved cybersecurity across information technologies can point the way how to secure another technological domain undergoing revolutionary changebiotechnology. The tools of biotechnology continue to improve rapidly, and governments, including that of the United States, are rightfully prioritizing biotechnology in their economic and security strategies. Now is a good time to consider how lessons from securing digital tech can guide efforts to safeguard the potential of the bioeconomy.

The U.S. governmentexpects biotechnology to have "outsized importance over the coming decade" in the context of geopolitical competition. Across the Atlantic, the European Commissionhas declared that "advances in life sciences, supported by digitalization and artificial intelligence (AI), and the potential of solutions based on biology to solve societal issues, make biotechnology and biomanufacturing one of the most promising technological areas of this century."

Now is a good time to consider how lessons from securing digital tech can guide efforts to safeguard the potential of the bioeconomy

Likewise, China's fourteenth Five-Year Plan prominently featured biotech and aims to position China's bioeconomy at the forefront globally.Germany, Japan,Saudi Arabia, Singapore, and the United Kingdom are similarly looking to biotechnology to meet major environmental, economic, and security challenges. The new and intense prioritization of biotech is, according to U.S. National Security Advisor Jake Sullivan,motivated by advancements in the ability to "read, write, and edit genetic code, which has rendered biology programmable."

Sullivan is right. Computers run on binary code. Cells also operate through digital code called DNA. Depending on its DNA sequence, a cell can be programmed to produce, for example, a therapeutic drug or inputs for food, agriculture, pharmaceutical, or chemical products. Analysts at McKinsey Global Institute have estimated that programmed cells can produce up to60% of the physical inputs to the global economy. In short, most things manufactured using traditional industrial processessuch as plastics, fuels, materials, and medicinescan soon be made through biomanufacturing and synthetic biology.

The growing interest in leveraging biotechnologies has stimulated an increasing focus on mitigating risks associated with intentional or accidental misuse of more powerful biotech tools. Conventional wisdom suggests that, as the tools of biotechnology become more powerful, mechanisms to mitigate risk could become increasingly limited. But it does not have to be that way. Technology can become both more powerful and more secure.

In fact, that is what happened in the information technology space. Changes in the way people used digital tech helped bring about improved approaches to handling cyber threats. That experience provides lessons for safeguarding biotechnology and the strategic importance and economic promise of the bioeconomy.

John Bumgarner, a cyber warfare expert who is chief technology officer of the U.S. Cyber Consequences Unit, works on his laptop computer, in Charlotte, North Carolina, on December 1, 2011. REUTERS/John Adkisson

In the early days, the information technology landscape was marked by isolated systems and diverse software applications that created a fragmented and often insecure environment. Over time, the desire for greater efficiency and more scalability led to increased centralization in the form of at-scale service providers. Simultaneously, cybersecurity also improved.

Consider cloud computing: Amazon Web Services, Microsoft Azure, and Google Cloud meet the need for government agencies and businesses to access scalable and flexible computing resources without the high costs and complexities of establishing and maintaining the workforce and physical infrastructure associated with on-site data storage. Cloud computing platforms offer on-demand services and allow organizations to pay for only what they use, significantly reducing overhead while enabling access to cutting-edge services. In many ways, cloud computing has transformed the digital age.

Similarly, app stores, such as Apple's App Store and Google Play Apps, revolutionized software distribution. Those marketplaces reach a global audience of users, reduce the barriers to entry in the software market, and provide a trustworthy, convenient experience for users.

Platforms helped establish a multilayered approach that raised the bar for cybersecurity across the entire digital ecosystem

Cloud computing and app store innovations also created opportunities to improve cybersecurity. For example, cloud computing companies not only streamline processes and services but also provide security through standardized protocols, regular updates, and controlled access. Cloud platforms deploy advanced security measures, including sophisticated encryption techniques and threat detection systems.

Centralization also facilitates the rapid deployment of security updates and patches, prompting operators and systems across cyberspace to address vulnerabilities quickly. Likewise, the app ecosystem allows for rigorous vetting of apps and authorization of developers under high standards of security and quality before consumerswhom app stores also often authorize and authenticateuse the apps.

The emergence of those centralized service providers enabled greater scaling of the digital economy and, at the same time, addressed cybersecurity challenges that a fragmented ecosystem could not handle. Platforms helped establish a multilayered approach that raised the bar for cybersecurity across the entire digital ecosystem.

The evolution of cybersecurity across the digital ecosystem offers a roadmap for the bioeconomy. Initially, decentralized systems opened doors to cybersecurity vulnerabilities. The shift toward centralization, partnering, and standardization in digital technologies and services proved instrumental in better mitigating those risks. Similar trends are evident in biotechnology.

The biotech field is transitioning from a fragmented landscape of research and development (R&D) toward more integrated and standardized platforms that support increased scaling of the bioeconomy. That ongoing shift can help establish a robust framework of biosecurity that parallels the improvement of cybersecurity in the digital realm.

Consider traditional approaches to developing biotechnologies. Like the digital information sector in its early stages, biotechnology work has generally taken place in a decentralized ecosystem, relying on expensive, laborious, slow, and uncertain experimentation across many labs and teams. Basically, scientists painstakingly manipulated biological materials at lab benches, akin to the era when digital programming and data processing required deep technical knowledge and bulky, expensive mainframes. The history of biotech development is marked by a relatively slow pace of discovery and high barriers to entry, just as early computing was limited to those with access to sophisticated equipment and the expertise to operate it.

Today, in a transformation that parallels the digital revolution, biotechnology is moving away from specialized, resource-intensive, and decentralized processes to service providers that embrace scale economics to offer more cost-effective and productive services, thus enabling a broader range of applications. That trend in biotech is multifaceted, but includes, for example, providers of R&D and DNA synthesis services.

An employee of BioNTtech works at the "Area 100 R&D" research laboratory for personalized mRNA-based cancer vaccines, in Mainz, Germany, on July 27, 2023. REUTERS/Wolfgang Rattay

Those shifts open opportunities for enhancing biosecurity. Consider the role of R&D platforms that provide services for companies and other actors across the bioeconomy. Those platforms function like cloud computing by offering faster, better, and cheaper results than traditional do-it-yourself approaches. In particular, in the context of building and training AI tools for biology, innovators are hungry for experimental data like never before, and external, purpose-built facilities are well suited to deliver that data on demand. Akin to cloud computing, those R&D platforms also stand to enhance safety and security within the biotech ecosystem.

For example, centralized R&D services are well positioned to adopt and implement security best practices and safety protocols. Biotech users can leverage reliable, standardized services while reducing the risks associated with conducting complex biological experiments independently. In a world where researchers can order biological data online and tap into trusted platforms to conduct experiments, innovators will benefit from improved convenience and efficiency as well as tested tools and validated methodologies that help ensure a higher degree of accuracy and safety in experimental outcomes.Accurate and safe outcomes are crucial in biotechnology, where errors or unintended consequences can have far-reaching implications.

Put simply, outsourcing to at-scale R&D service providers can accelerate biotech discovery and innovation and, at the same time, contribute to a foundation of trust and security that is crucial for the responsible advancement of the bioeconomy.

Similarly, companies that specialize in DNA synthesis services provide another way for at-scale service providers to enhance security within the bioeconomy. Such companies, equipped with advanced technologies and expertise, are better positioned to screen orders for DNA sequences and mitigate the risks of accidental or intentional synthesis of hazardous genetic material than researchers and organizations that only occasionally synthesize DNA.

DNA synthesis service providers are more likely to have the resources and specialized knowledge to thoroughly assess the implications of every DNA sequence they create. Such entities can implement stringent screening protocols that flag and prevent the production of potentially dangerous DNA, such as sequences associated with pathogens or toxins. DNA synthesis as a service not only streamlines an important production process, but also creates incentives to strengthen security. That approach reduces the likelihood that harmful genetic materials are inadvertently synthesized and offers a pathway toward a safer and more secure environment for genetic research and experimentation.

Achieving a secure bioeconomy is imperative for protecting strategic interests, bolstering economic growth, and advancing collective well-being

The trend toward partnership and outsourcing has already proven that it can deliver dramatically improved tools to respond to biological emergencies. Indeed, the response to the COVID-19 pandemic has shown that, when combined with public-sector leadership and support, the bioeconomy offers ready capacity to deliver advanced biosecurity tools and core capabilities of pandemic preparedness, including biosurveillance, environmental monitoring, and continuous development and large-scale production of diagnostics, therapeutics, and vaccines.

The shift in biotechnology toward prioritizing external services is under way and promises to increase innovation, efficiency, and reliability in the bioeconomy while improving biotech safety and security. Such a win-win outcome is not, however, inevitable.

The road to better cybersecurity has not been smooth, and continuing to make cyberspace more secure against evolving and new threats remains a constant challenge. Likewise, creating and sustaining an innovative and secure bioeconomy will not be easy. That task will confront challenges ranging from the machinations of geopolitics to the mysteries of biology.

As true of digital tech, achieving a secure bioeconomy is imperative for protecting strategic interests, bolstering economic growth, and advancing collective well-being. In that context, current trends offer hope that, one day, everyone will have an opportunity to use biotech safely and productively the way they use digital tech today.

A biohazard warning sign is displayed at the Yemaachi Biotechnology cancer research laboratory, in Accra, Ghana, on May 19, 2022. REUTERS/Francis Kokoroko

Ryan Morhard is director of policy and partnerships at Ginkgo Bioworks, Inc. in Boston, Massachusetts; an affiliate of the Georgetown University Center for Global Health Science and Security; and a term member of the Council on Foreign Relations.

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New Biotech Platforms Offer Better Paths to Biosecurity - Think Global Health

Pictured: Man carrying a box of his office items after being laid off/iStock, megaflopp

Industry lobbying group Biotechnology Innovation Organization has kicked off a restructuring initiative that will see the termination of 30 employees, according to exclusive reporting by STAT News.

Citing four sources, STAT noted that the layoffs will affect senior leaders at Biotechnology Innovation Organization (BIO) including CSO Cartier Esham, Chief Policy Officer John Murphy and Chief Public Affairs and Marketing Officer Rich Masters, who confirmed to the publication that he would be leaving the group.

The layoffs were announced by BIO CEO John Crowley through an internal email, noting that the changes were meant to better align our operations with our mission and the strategic priorities. Crowley was named as BIOs new head in December 2023 and assumed the post in March 2024.

The newly minted CEO added in his email that BIO had been too absorbed trying to do everything, that the organization has been missing the opportunity to do many of the big thingsthose that are the most urgent, most important and that will have the greatest impact for our members, patients and persons worldwide, STAT reported.

BioSpacereached out to BIO for comment but the lobbying group was not immediately available.

BIOs restructuring come as growing tensions between the U.S. and China are potentially impacting the biopharma industry as the BIOSECURE Act advances in Congress. First introduced by Reps. Mike Gallagher (R-Wis.) and Raja Krishnamoorthi (D-Ill.) in January 2024, the bill is designed to prevent the flow of taxpayer dollars to biotech entities of concern.

The proposed legislation targets Chinese biotechs that are under the control of foreign adversaries including WuXi Apptec, Beijing Genomics Institute, Complete Genomics and MGI. According to Krishnamoorthi, these companies amass and analyze large amounts of genomic data, which adversarial governments can then use against the U.S.

In March 2024, BIO came under fire from Gallagher who asked U.S. Attorney General Merrick Garland to look into the lobbying group after it had allegedly undertaken a recent advocacy campaign against the BIOSECURE Act.

BIOs advocacy on behalf of WuXi Apptecintended to protect the companys access and commercial ties in the United States despite the risk to U.S. national securityis aligned with the interests of the CCP and the Peoples Republic of China government, Gallagher wrote.

Shortly after, BIO announced that it had parted ways with WuXi Apptec and reiterated its commitment to national security while voicing its support for the BIOSECURE Act.

Earlier this month, House lawmakers introduced a new draft of the BIOSECURE Act giving pharma companies until 2032 to sever ties with the Chinese biotech companies of concern. Crowley in a statement said the new version of the BIOSECURE Act bill highlights a key vulnerability in our global supply chain and importantly provides a reasonable timeframe for companies to decouple their reliance on China-based biomanufacturing.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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BIO Axes 30 Staff in Realignment as Congress Advances BIOSECURE Act - BioSpace

If you had the chance to help diagnose sepsis, save stored human cells from dying in cryogenic tanks or measure brain data with just a pillowcase, which would you pick? These are the missions of the three local companies that vied Thursday night for a $20,000 prize and the chance to compete for $100,000 in the North Carolina Biotechnology Centers NC Biotech Venture Challenge Southeastern Venture Pitch Finals. Three Wilmington-based companies Predicate HPG, Boreas Monitoring Solutions and Nuream presented at the University of North Carolina Wilmingtons Lumina Theater on Thursday. Neuro-data startup Nuream took home first place. Winning this NC Biotech Venture Challenge is not about us, Nureams co-founder and CEO Rob Cooley told the Business Journal after winning the Southeastern title. It's about the ecosystem and everybody that contributes to it. We're just fortunate, on a stage of amazing entrepreneurs and innovators, to be recognized. Boreas Monitoring Solutions and Predicate HPG both went home with $10,000 for second and third place, respectively. Predicate HPG was also named the 2024 Coastal Entrepreneur of the Year on Wednesday. The group of three finalists participated in a two-month mentoring period leading up to the pitch final. Biotechnology and entrepreneurship professionals in the region served as mentors and judges throughout the process. During his pitch, Cooley said Nureams prototype was completed in recent months and works out of the box. The product is a pillow cover with sewn-in sensors that monitor brain activity. It proves the companys theory that they can collect users brainwaves while they sleep, he said. Cooley said eventually the company strives to create Fabric-as-a-Sensor (FAAS), a product in which the threads of fabric would be sensors instead of sensors sewn into the product. Cooley also said that the company filed for its first provisional patent on Wednesday. Nuream will advance to the NC Biotech Venture Challenges state-level finals on June 27 in Greensboro. Winners from all five N.C. Biotechnology Center office regions around the state will engage in additional mentoring until they compete for the state title. This years venture challenge brought an additional category into the mix. A pre-venture pitch challenge was added this year, allowing university researchers to compete for funding to move toward commercializing their work. UNCW researcher Ying Wang secured $20,000 as the first pre-venture pitch competition winner with his novel universal flu vaccine. Wangs research also received commercialization funding throughNC Innovation'spilot cyclelast week. The second and third-place researchers each won $10,000 to help commercialize their work. The Southeastern Venture Pitch Finals also featured an industry panel from biotech companies with a presence in Wilmington.Katie Schlipp,president of laboratory operations with pharmaceutical contract manufacturer Alcami, Michael Braddock, chief revenue officer of cold storage company Frontier Scientific Solutionsand Brett Lanier, president of pharmaceutical developer Isosceles Pharmaceuticals sat on the panel. In addition to discussing how their companies interact with the community and how the community can support their companies, some gave updates on what theyre working on launching soon. Braddock said Frontier Scientific Solutions is starting a non-stop, round-trip freight aircraft flight from Shannon, Ireland to Wilmington. Frontier officials are in the final stages of negotiations right now, he said. The company is also completing a case study that proves the new flight can reduce a 260-hour trip, transporting a product from Dublin, Ireland to its manufacturing site in Greenville, North Carolina to 27 hours, Braddock said. Frontier specializes in cold storage, which is often necessary in the life science industry when transporting pharmaceuticals that must be kept at a certain temperature throughout the supply chain. Lanier of Isosceles was the first winner of the NC Biotechs venture prize. His company, which specializes in non-opioid pain relief, is working on expanding into the immunology space, he said. We've got some pretty fantastic data that we're hoping to share in the near future around immunology, Lanier said. And so, we're looking at a pivot, so I may need to go back and retake the venture challenge and learn how to pivot into immunology, he joked.

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NC Biotech Southeast venture final names winner, highlights area life sciences - Greater Wilmington Business Journal

Philadelphia has rapidly emerged as a major hub for biotechnology, often referred to as Cellicon Valley due to its significant contributions to cell and gene therapy. This thriving ecosystem is driven by a combination of leading academic institutions, a robust network of research organizations, and a dynamic startup scene. Key institutions like the University of Pennsylvania and Childrens Hospital of Philadelphia have been instrumental in developing groundbreaking therapies, such as the first Food and Drug Administration (FDA)-approved gene therapy, Luxturna.Here are eight biotech companies based in Philadelphia to put on your radar.

AUM LifeTech, founded in 2013, is a Philadelphia-based preclinical biotech company. It focuses on developing RNA silencing technologies. Their proprietary FANA technology offers a next-generation approach to gene silencing and regulation. FANA antisense oligonucleotides (ASOs) are designed to target and silence specific RNA molecules, including mRNA, microRNA, and long non-coding RNA, without requiring delivery agents such as viral vectors or complex formulations. ASOs are short, synthetic strands of nucleic acids designed to specifically bind to the mRNA of a target gene. By binding to this mRNA, ASOs can modify the expression of the target gene. This self-delivering capability enhances the efficiency and reduces the toxicity of RNA silencing applications, making it particularly useful in therapeutic development and biomedical research.

AUM LifeTech has established key collaborations with institutions such as the Childrens Hospital of Philadelphia and the University of Pennsylvania. These partnerships aim to leverage FANA technology in cancer immunotherapy and other therapeutic areas.

AUM LifeTechs pipeline focuses on developing RNA silencing therapies for a variety of genetically defined diseases. The company also has a T-regulatory (Treg) candidate focused on lung cancer targeting the FOXP3 gene. AUM LifeTech also develops a project targeting Parkinsons disease.

BlueWhale Bio, a biotech company founded in 2023 and based in Philadelphia, emerged from the University of Pennsylvania. It focuses on cell and gene therapy manufacturing. BlueWhale Bio aims to overcome the bottlenecks in the production of cell and gene therapies to make these treatments more accessible and affordable for patients.

In September 2023, BlueWhale Bio secured $18 million in seed financing led by Danaher Ventures. This initial funding supports the development of BlueWhale Bios manufacturing platform and product portfolio.

While specific product details about the companys pipeline are still under wraps, the companys approach focuses on addressing the critical needs of cell and gene therapy production, including improving process efficiency, scalability, and cost-effectiveness.

The company is the first to emerge from the Danaher Ventures Pioneer Program, which supports the creation of companies with disruptive technologies in life sciences.

This is not the first time we have talked about Carisma Therapeutics as it was on our list of companies to follow last December. This biotech company based in Philadelphia is innovating in the field of immunotherapy with its development of chimeric antigen receptor macrophages (CAR-M) and CAR-monocytes. These engineered cells leverage the innate and adaptive immune responses to target and destroy cancer cells, particularly in solid tumors. Unlike traditional CAR-T therapies that face challenges in treating solid tumors, CAR-macrophages are designed to overcome these hurdles by infiltrating tumors, surviving in the hostile tumor environment, and activating a broader immune response.

Carisma raised substantial capital through multiple funding rounds, bringing the total to $151.9 million after completing a $30 million series C round in 2002.

Additionally, Carisma has established a collaboration with Merck to evaluate the efficacy of CAR-macrophages in combination with Mercks anti-PD-1 therapy, KEYTRUDA. This collaboration aims to explore the synergistic effects of combining CAR-M technology with existing immunotherapies to enhance treatment outcomes for HER2-positive cancers. The companies announced the dosing of the first patient in a phase 1 clinical trial in 2023.

In 2024, Carisma made strategic decisions to streamline its pipeline. While development of its CAR-Macrophage candidate CT-0508 has been halted, the company continues to advance CT-0525, a CAR-Monocyte that received FDA clearance in 2023. This clearance allows Carisma to begin evaluating CT-0525 in phase 1 clinical trials. Indeed, the company announced the dosing of the first patient in phase 1 clinical study, last week.

Century Therapeutics, founded in 2018 and based in Philadelphia, is a clinical-stage biotech company that leverages induced pluripotent stem cells (iPSCs) to develop allogeneic, or off-the-shelf, cell therapies. IPSCs are a type of stem cell generated directly from adult cells. They are created by reprogramming somatic cells, such as skin or blood cells, back into an embryonic-like pluripotent state. They offer a renewable source of various cell types, which can be used for therapeutic purposes, drug testing, and understanding disease mechanisms without the ethical concerns associated with embryonic stem cells.

These therapies aim to treat cancers and autoimmune and inflammatory diseases. Centurys approach utilizes the self-renewing capacity of iPSCs combined with advanced genetic engineering and manufacturing technologies.

In April 2024, the company closed a $60 million private placement being utilized to expand the clinical development of their lead program, CNTY-101, among other initiatives. Additionally, Century Therapeutics has a collaboration with Bristol-Myers Squibb.

CNTY-101, the companys lead program is an iPSC-derived NK (natural killer) cell therapy targeting CD19-positive hematologic cancers. It is currently in clinical development and has shown promising preliminary phase 1 results in inducing cytolysis of B-cells. The company plans to expand CNTY-101s application into additional autoimmune disease indications.

Century is also advancing other iPSC-derived NK and T cell therapies targeting various cancers and autoimmune diseases, leveraging their platform to develop multiple product candidates with the potential to provide significant therapeutic benefits.

In 2024, Century Therapeutics announced the acquisition of Clade Therapeutics, which enhances its capabilities in developing scalable, consistent stem cell-based medicines.

Context Therapeutics, headquartered in Philadelphia, is a clinical-stage biotech company focused on developing treatments for solid tumors. The companys primary focus is on hormone-driven cancers, including breast, ovarian, and endometrial cancers. Contexts lead product candidate, CTIM-76, is a selective Claudin 6 (CLDN6) x CD3 bispecific antibody designed to target CLDN6-positive tumors. CLDN6 is a membrane protein found in multiple solid tumors but is minimally expressed in healthy adult tissues, making it an attractive target for cancer therapy.

In May 2024, Context Therapeutics announced the completion of a $100 million private placement. This funding is expected to support the companys operations into 2028, providing substantial resources for the development and clinical testing of CTIM-76 and other pipeline candidates. The company has also received FDA clearance for its IND application for CTIM-76, allowing it to proceed with a phase 1 clinical trial.

iECURE, founded in 2020 and based just outside of Philadelphia, is a clinical-stage gene editing company dedicated to developing mutation-agnostic in vivo gene insertion therapies for treating liver disorders. These therapies aim to knock in healthy copies of disease-causing genes, meaning, introducing a functional copy of a gene directly into the genome of a patients cells. The companys approach targets a wide range of genetic mutations, making it a versatile solution for many monogenic liver diseases.

iECURE has raised significant funding with a notable $50 million series A round, followed by an additional $65 million in series A-1 financing. The company has also received FDA Fast Track designation and orphan drug designation for its lead product candidate, ECUR-506, allowing the initiation of the OTC-HOPE clinical trial. Additionally, the company received approval from the U.K. Medicines & Healthcare Products Regulatory Agency (MHRA) to expand this study.

ECUR-506, the companys lead candidate targets ornithine transcarbamylase (OTC) deficiency, a severe inherited metabolic disorder. ECUR-506 utilizes two adeno-associated virus (AAV) capsids: one carrying an ARCUS nuclease to target gene editing in the PCSK9 gene, and another carrying the functional OTC gene. This innovative approach aims to provide permanent expression of the healthy OTC gene. The OTC-HOPE study, a phase 1/2 clinical trial, is currently enrolling newborn males with severe neonatal onset OTC deficiency to evaluate the safety, tolerability, and efficacy of ECUR-506.

Passage Bio, founded in 2019 is developing transformative therapies for patients suffering from central nervous system (CNS) disorders. The companys primary focus is on creating one-time gene therapies designed to address the underlying pathology of neurodegenerative diseases. Passage Bios lead product candidate, PBFT02, aims to treat frontotemporal dementia (FTD) in patients with mutations in the GRN gene, by elevating progranulin levels to restore lysosomal function and slow disease progression.

PBFT02 uses a harmless virus (AAV1) to carry a healthy copy of the GRN gene directly to the brain. The virus is injected into a fluid-filled space at the base of the brain, the cisterna magna. This method aims to deliver the gene precisely where its needed to help restore normal brain function and slow down the diseases progression.

In 2024, Passage Bio reported positive interim results from the upliFT-D clinical trial for PBFT02. The data showed that the therapy was generally well-tolerated with no serious adverse events in patients receiving an enhanced steroid regimen. Additionally, significant biomarker improvements were observed. Passage Bio plans to expand the clinical development of PBFT02 into additional neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and Alzheimers disease.

Passage Bio is also developing gene therapies for GM1 gangliosidosis, Krabbe disease, metachromatic leukodystrophy (MLD), and other neurodegenerative conditions.

Verismo Therapeutics, founded in 2022 and based in Philadelphia, is a clinical-stage biopharmaceutical company working on the development of next-generation CAR T-cell therapies using its novel KIR-CAR platform. This platform incorporates killer immunoglobulin-like receptors (KIRs) derived from NK cells, aiming to improve T-cell persistence and efficacy, especially in challenging solid tumor environments.

KIRs are a family of receptors found on the surface of NK cells, which are part of the innate immune system. These receptors help NK cells recognize and respond to infected or cancerous cells by binding to specific ligands on the target cells. KIRs regulate the activity of NK cells by either activating or inhibiting their functions. When KIRs bind to their specific ligands, they can enhance the cytotoxic activity of NK cells against tumor cells, making them a critical component in targeting cancer cells.

Verismos approach seeks to address the limitations of traditional single-chain CAR T-cell therapies, such as T-cell exhaustion and limited efficacy in solid tumors.

Verismo Therapeutics has secured notable funding, including a $17 million pre-series A financing round. The company collaborates closely with the University of Pennsylvania, where the KIR-CAR technology was originally developed. This partnership includes access to research and clinical trial support.

Verismos pipeline features several promising candidates:

The citys strategic location along the U.S. Northeast corridor offers easy access to major markets, investors, and collaborators, making it an attractive base for biotech companies. Philadelphias biotech landscape is characterized by its collaborative environment where academic institutions, research centers, and biotech companies work closely together. This synergy is evident in initiatives like the Keystone LifeSci Collaborative, which focuses on building a coordinated strategy for industry growth, talent development, and regional competitiveness.

Philadelphias biotech sector is supported by substantial investments and state-of-the-art infrastructure. The University City Science Center and the Navy Yard, home to 150 companies, are examples of dedicated biotech hubs within the city, providing space and resources for innovation and development.

Partnering 2030: The Biotech Perspective 2023

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Eight Philadelphia biotech companies spearheading the industry - Labiotech.eu

SEATTLE, May 23, 2024 (GLOBE NEWSWIRE) -- Nautilus Biotechnology, Inc. (NASDAQ: NAUT; or Nautilus), a company pioneering a single-molecule protein analysis platform, today announced the company will be participating in the Goldman Sachs 45th Annual Healthcare Conference.

Nautilus management is scheduled to participate in a fireside chat on Monday, June 10, 2024, at 8:40 a.m. Eastern Time. Interested parties may access a live and archived webcast of the presentation on the Investors section of the company website at: http://www.nautilus.bio.

About Nautilus Biotechnology, Inc. With its corporate headquarters in Seattle, Washington and its research and development headquarters in San Carlos, California, Nautilus is a development stage life sciences company working to create a platform technology for quantifying and unlocking the complexity of the proteome. Nautilus mission is to transform the field of proteomics by democratizing access to the proteome and enabling fundamental advancements across human health and medicine. To learn more about Nautilus, visit http://www.nautilus.bio.

Media Contact press@nautilus.bio

Investor Contact investorrelations@nautilus.bio

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Nautilus Biotechnology to Participate in the Goldman Sachs 45th Annual Global Healthcare Conference - GlobeNewswire

SPMVV receives 47.9 lakh grant from the governments Department of Biotechnology  The Hindu

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SPMVV receives 47.9 lakh grant from the governments Department of Biotechnology - The Hindu

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