GenEdit Announces Multiyear Collaboration and License Agreement with Genentech to Develop Novel Nanoparticles to Deliver Genetic Medicines for Autoimmune Disease

SOUTH SAN FRANCISCO, Calif., January 23, 2024 – GenEdit, Inc., a developer of genetic medicines that leverage its NanoGalaxy® platform for tissue-selective delivery, today announced a multiyear collaboration and license agreement with Genentech, a member of the Roche Group. The companies will employ GenEdit’s NanoGalaxy platform to discover and develop novel nanoparticles to deliver Genentech’s nucleic acid-based medicines for treatment of autoimmune disease.

“We are excited to work with Genentech to develop novel therapeutics for autoimmune disease,” said Kunwoo Lee, Ph.D., CEO of GenEdit. “Genentech is a leader in advancing breakthrough science to advance the development of novel therapeutic modalities. This collaboration underscores the promise of GenEdit’s NanoGalaxy platform to deliver nucleic acid-based medicines via our hydrophilic nanoparticle technology.”

“We look for external innovation to complement our internal science to help advance transformative medicines for people living with autoimmune diseases,” said James Sabry, global head of Roche Pharma Partnering. “Our collaboration with GenEdit will use their polymer nanoparticle library and platform with the potential to reshape treatment paradigms to help people with devastating and difficult-to-treat autoimmune disease.”

Under the terms of the agreement, GenEdit will collaborate with Genentech to discover and develop hydrophilic nanoparticles (HNPs) that can be used to develop nucleic acid-based medicines for autoimmune indications. Genentech will be responsible for preclinical, clinical, and regulatory development as well as commercialization of products resulting from the use of GenEdit’s nanoparticles.

GenEdit will receive an upfront payment of $15 million and is eligible to receive up to $629 million in near-term, preclinical and clinical development, commercial, and net sales milestone payments over the course of the collaboration as well as tiered royalties on global net sales for resulting products.

About GenEdit, Inc.
At GenEdit, our mission is to develop innovative genetic medicines with targeted in vivo delivery. Our NanoGalaxy® platform of non-viral, non-lipid hydrophilic nanoparticles (HNPs) offer a combination of advantages over other delivery methods, including tissue selectivity, payload flexibility, low immunogenicity, ability to re-dose, and ease of manufacturing. We are leveraging our platform to develop therapeutics for internal and partnered programs. For more information, visit www.genedit.com and follow us on LinkedIn .

Contacts
Corporate: Kunwoo Lee, Ph.D., GenEdit, Inc., info@genedit.com
Media: Jessica Yingling, Ph.D., Little Dog Communications Inc.jessica@litldog.com, +1.858.344.8091

GenEdit Announces Series A1 Financing

SOUTH SAN FRANCISCO, Calif., January 23, 2024 – GenEdit, Inc., a developer of genetic medicines that leverage its NanoGalaxy® platform for tissue-selective delivery, today announced that it has raised $24 million in a Series A1 financing. Existing investors Eli Lilly, Sequoia Capital, Korea Investment Partners, Woori Venture Partners, DAYLI Partners, KB Investment, IMM Investment and TIMEFOLIO Asset Management are joined by new investors KDB Silicon Valley, Mirae Asset Venture Investment, ACVC, Pathway Partners, LoftyRock Investment, Terra VC, K2 Investment, Dong-A ST, KIMCO and Huons. The funding will support continued development of the NanoGalaxy platform and a pipeline of preclinical therapeutic candidates.

The NanoGalaxy platform includes thousands of chemically distinct hydrophilic nanoparticles (HNPs), each with unique properties making them amenable to targeting different tissues and cell types and carrying diverse genetic medicine payloads. GenEdit has demonstrated in vivo delivery of siRNA, mRNA, and gene editors to the nervous, immune, and other extra-hepatic systems and is pursuing multiple therapeutic areas through internal and partnered programs.

“The bottleneck for developing new genetic medicines continues to be delivery,” said Kunwoo Lee, Ph.D., CEO of GenEdit. “The ability of our HNPs to carry diverse payloads to diverse tissues has the potential to be an enabling technology for genetic medicines.”

About GenEdit, Inc.
At GenEdit, our mission is to develop innovative genetic medicines with targeted in vivo delivery. Our NanoGalaxy® platform of non-viral, non-lipid hydrophilic nanoparticles (HNPs) offer a combination of advantages over other delivery methods, including tissue selectivity, payload flexibility, low immunogenicity, ability to re-dose, and ease of manufacturing. We are leveraging our platform to develop therapeutics for internal and partnered programs. For more information, visit www.genedit.com and follow us on LinkedIn.

Contacts
Corporate: Kunwoo Lee, Ph.D., GenEdit, Inc., info@genedit.com
Media: Jessica Yingling, Ph.D., Little Dog Communications Inc.jessica@litldog.com, +1.858.344.8091

GenEdit Is a Phase I Winner of the NIH TARGETED Challenge

SOUTH SAN FRANCISCO, Calif., December 13, 2023 – GenEdit, Inc., a developer of genetic medicines that leverage its NanoGalaxy® platform for tissue-selective delivery, today announced that it is a Phase I winner of the National Institutes of Health (NIH) TARGETED Challenge for programmable delivery systems for gene editing. GenEdit’s application highlighted delivery to the nervous, immune, and other extra-hepatic systems along with possible therapeutic applications.

“The NIH challenge shows the importance of programmable delivery systems for genetic medicines,” said Romu Corbau, Ph.D., Chief Scientific Officer of GenEdit. “The award is a recognition of the potential of the NanoGalaxy platform for a broad range of genetic medicines for multiple therapeutic areas.”

About the TARGETED Challenge
The Targeted Genome Editor Delivery (TARGETED) Challenge is a $6,000,000 challenge to improve the current state of in vivo delivery technologies for genome editors in two Target Areas: 1) Programmable Delivery System for Gene Editing and 2) Crossing the Blood-Brain Barrier. The Challenge is a three-phase competition. In Phase 1, participants were asked to submit a proposal describing their proposed solution and how it addresses the requirements for one of the Target Areas.

About GenEdit
At GenEdit, our mission is to develop innovative genetic medicines with targeted in vivo delivery. Our NanoGalaxy® platform of non-viral, non-lipid hydrophilic nanoparticles (HNPs) offer a combination of advantages over other delivery methods, including tissue selectivity, payload flexibility, low immunogenicity, ability to re-dose, and ease of manufacturing. We are leveraging our platform to develop therapeutics for internal and partnered programs. For more information, visit www.genedit.com and follow us on LinkedIn.

Contacts
Corporate: Kunwoo Lee, Ph.D., GenEdit, Inc., info@genedit.com
Media: Jessica Yingling, Ph.D., Little Dog Communications Inc.jessica@litldog.com, +1.858.344.8091

Sarepta Therapeutics and GenEdit Share Progress on Research Collaboration and Announce Agreement to Develop Gene Editing Therapeutics for Neuromuscular Diseases

  • Collaboration combines Sarepta’s proprietary gene editing technologies and GenEdit’s non-viral delivery platform to create new genetic medicines for patients with neuromuscular diseases
  • Initial results from the ongoing research collaboration have demonstrated delivery to muscle tissue after systemic administration
  • Sarepta and GenEdit will work together to identify development candidates for further testing

CAMBRIDGE, Mass., and SOUTH SAN FRANCISCO, Calif., Feb. 1, 2022 (GLOBE NEWSWIRE)– Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, and GenEdit, Inc., a developer of genetic medicines that leverage its NanoGalaxyTM polymer nanoparticle platform for tissue-selective delivery, today announced a research collaboration and option agreement under which the companies are employing GenEdit’s NanoGalaxy platform and Sarepta’s gene editing technology to develop gene editing therapeutics for the treatment of neuromuscular diseases. As part of the agreement, Sarepta obtains exclusive option rights to license polymer nanoparticles developed by GenEdit in the collaboration for up to four neuromuscular indications selected by Sarepta.

Initial in vivo results from the research collaboration between GenEdit and Sarepta have demonstrated the potential of GenEdit’s polymer nanoparticles to deliver therapeutic cargo to specific muscle tissue after systemic administration to allow for targeted, non-viral systemic delivery of genetic medicines. Theresearch collaboration and option agreement commenced in December 2020.

“We’ve been impressed with the diversity of GenEdit’s NanoGalaxy platform and its screening and selection process, which has generated a number of distinct polymers that deliver to muscle,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “Sarepta is committed to the development of therapies for rare neuromuscular diseases, and we look forward to continuing to work with the team at GenEdit to advance effective gene editing-based treatments for these patients.”

Gene editing has the potential to revolutionize the treatment of diseases caused by genetic mutations by permanently modifying the genes that lead to disease. Sarepta is pursuing a variety of approaches to genetic medicine, including gene editing, as a potentially curative treatment for rare neuromuscular diseases. GenEdit has demonstrated in preclinical studies that its NanoGalaxy platform can selectively deliver to different tissues a variety of functional genetic medicine cargos, including CRISPR-Cas9 ribonucleoprotein, for targeted in vivo gene editing.

“GenEdit has demonstrated in this collaboration and in our own studies that the NanoGalaxy platform can overcome historic challenges in the field and achieve tissue-selective delivery of a broad range of genetic medicine cargos,” said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. “GenEdit is excited to continue to advance our collaboration with Sarepta and work together to identify and develop gene editing therapeutic candidates for neuromuscular diseases with the goal of having a tremendous impact on patients.”

In addition to research payments, under the terms of the collaboration and option agreement, GenEdit may receive up to $57 million in near-term payments and is also eligible for significant future development, regulatory and commercial milestones and tiered royalties ranging from upper-single to low-double digits on future product sales. Additional financial details were not disclosed.

About GenEdit, Inc.

At GenEdit, our mission is to discover and develop innovative genetic medicines with targeted in vivo delivery. Our NanoGalaxy™ platform of non-viral, non-lipid polymer nanoparticles will enable a universe of opportunities to launch the next generation of safe and effective genetic medicines. To bring these medicines to patients, we are developing our own internal pipeline and delivering a wide variety of therapeutic payloads for our partners’ programs. For more information, follow us on LinkedIn and Twitter.

About Sarepta Therapeutics, Inc.

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on TwitterLinkedInInstagram and Facebook.

Sarepta Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties’ obligations and responsibilities under the agreement; potential payments and fees; the potential for gene editing to revolutionize the treatment of diseases caused by genetic mutations by permanently modifying the genes that lead to disease; and the potential benefits of the collaboration between Sarepta and GenEdit, including the potential of GenEdit’s polymer nanoparticles to deliver therapeutic cargo to specific muscle tissue after systemic administration to allow for targeted, non-viral systemic delivery of genetic medicines, Sarepta’s option to license polymer nanoparticles developed by GenEdit in the collaboration for up to four neuromuscular indications selected by Sarepta, the potential to advance effective gene editing-based treatments for patients with rare neuromuscular diseases, and the identification of development candidates for further testing.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration and agreement may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; success in preclinical trials does not ensure that later clinical trials will be successful; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sarepta’s control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta’s product candidates and the COVID-19 pandemic; even if Sarepta’s programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2020, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.

Sarepta Contacts

Investors: Ian Estepan, iestepan@sarepta.com, +1 (617) 274-4052,

Media Contact: Tracy Sorrentino, tsorrentino@sarepta.com, +1 (617) 301-8566

GenEdit Contacts

Company Contact: Kunwoo Lee, Ph.D., GenEdit, Inc., info@genedit.com, +1 (510) 766-2575

Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com, +1 (858) 344-8091

 

GenEdit Announces Series A Financing and Provides Research Update at TIDES 2021

  • Funding will advance development of NanoGalaxyTM, the company’s proprietary platform of polymer nanoparticles optimized for tissue-selective delivery of genetic medicines, and selection of therapeutic candidates for clinical development by GenEdit
  • Data presented today show GenEdit’s polymer nanoparticles can be optimized for tissue-selective delivery of a range of genetic medicine modalities and dosed multiple times without diminished function of the genetic payload in animal models

SOUTH SAN FRANCISCO, Calif., September 23, 2021 – GenEdit, Inc., a biotechnology company with a mission to develop curative therapies through targeted in vivo delivery of genetic medicines, today announced that it has closed a $26 million Series A financing. New investors Eli Lilly, KTB Network, Ltd., Company K Partners, Korea Investment Partners, DAYLI Partners, KB Investment, IMM Investment, and TIMEFOLIO Asset Management, join existing investors DCVC Bio, SK Holdings, Bow Capital, and Sequoia Capital, participating in the financing. The funding will support the further development of GenEdit’s NanoGalaxyTM platform of non-viral, non-lipid polymer nanoparticles, and the selection for clinical development of therapeutic candidates targeting diseases of the nervous system.

In addition, GenEdit today disclosed new in vivo data demonstrating tissue-selective delivery after intravenous or intrathecal administration of its polymer nanoparticles. This selectivity was achieved through optimization of polymer structure utilizing the NanoGalaxy platform. Additional data show GenEdit’s polymer nanoparticles can be dosed multiple times with functional activity of the payload maintained in each subsequent dose. GenEdit’s CEO and Cofounder Kunwoo Lee, Ph.D. will present this data at 2:30 pm ET today at TIDES 2021 in Room 204AB in a talk entitled, “Delivery of Genetic Medicines with Non-Viral Polymer Nanoparticles.”

“With this financing, we are exploring the universe of opportunities within GenEdit’s NanoGalaxy platform to treat serious diseases without effective treatment options,” said Dr. Lee. “The data presented today indicates we can overcome the historic challenges in the field of gene therapy and establishes the feasibility of using GenEdit’s polymer nanoparticles to deliver genetic medicines to a variety of tissues, including the CNS, with the potential for delivering a therapeutic effect.”

GenEdit’s NanoGalaxy platform includes thousands of chemically distinct polymers, each with unique properties making them amenable to targeting different tissues and cell types and carrying diverse genetic medicine payloads. GenEdit’s polymer nanoparticles can deliver DNA, RNA, or CRISPR ribonucleoprotein, depending on the requirement to add, delete, edit, or silence a gene for therapeutic effect.

“With its NanoGalaxy platform, the GenEdit team has achieved impressive delivery to cells of the central nervous system and has a tunable platform to target genetic medicines to different tissue types,” said Andrew Adams, Vice President of New Therapeutic Modalities at Eli Lilly and GenEdit Board observer. “Solving the delivery problem is what will truly launch genetic medicines to being an essential therapeutic modality for a wide range of diseases.”

 

About GenEdit, Inc.

At GenEdit, our mission is to develop curative therapies through targeted in vivo delivery of genetic medicines. Our NanoGalaxy™ platform of non-viral, non-lipid polymer nanoparticles will enable a universe of opportunities to launch the next generation of safe and effective genetic medicines. To bring these medicines to patients, we are developing our own internal pipeline and delivering a wide variety of therapeutic payloads for our partners’ programs. For more info, visit www.genedit.com.

 

Contacts

Company Contact: Kunwoo Lee, Ph.D., GenEdit, Inc., info@genedit.com, +1 (510) 766-2575

Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com, +1 (858) 344-8091

 

GenEdit Expands Leadership Team to Advance Targeted Genetic Medicines into Clinical Trials

SOUTH SAN FRANCISCO, Calif., September 22, 2021 – GenEdit, Inc., a biotechnology company with a mission to develop curative therapies through targeted in vivo delivery of genetic medicines, today announced that it has added two accomplished industry executives to its leadership team. The company has appointed Romuald Corbau, Ph.D., as Chief Scientific Officer (CSO) and Aaron Mishel as Chief Financial Officer (CFO).

As part of his 20-plus years of drug discovery and development experience, Dr. Corbau led the translation of innovative genetic medicine therapies into clinical development at Spark Therapeutics and Freeline. Mr. Mishel brings to GenEdit more than twenty years of healthcare sector experience across various leadership positions in finance, operations, corporate development, and investment banking equity research.

“The expansion of the management team comes at a pivotal time for GenEdit as we further develop our NanoGalaxyTM platform to enable genetic medicine delivery and select therapeutic candidates for clinical development,” said Kunwoo Lee, Ph.D., GenEdit’s CEO and Co-founder. “Romu and Aaron bring a depth of experience and expertise that will be invaluable as we build our company, platform, and pipeline to advance new treatments internally and with partners for a broad range of diseases with unmet medical needs.”

Dr. Corbau joins GenEdit from Freeline, where he led the research organization and built differentiated systemic gene therapy capabilities. Before Freeline, he was the Translational Lead at Spark Therapeutics. At both companies, Dr. Corbau was focused on the discovery and development of AAV-based gene therapies. Prior to that, he was Director of Biology at the Center for Innovation and Stimulation of Drug Discovery. Dr. Corbau began his pharmaceutical career at Pfizer Global Research & Development.

“GenEdit has a truly convincing approach to the delivery of genetic medicines that offers advantages over other vector systems,” added Dr. Corbau. “The team has demonstrated that NanoGalaxy polymer nanoparticles can achieve the delivery of a wide range of payloads to target tissues and can be successfully redosed. I look forward to working with the team to explore all the possibilities within NanoGalaxy to develop innovative therapies that will benefit as many patients as possible.”

Prior to joining GenEdit, Mr. Mishel served as the Chief Financial Officer of Magnetic Insight. Previously, he was the Head of Finance for Modis Therapeutics, where he helped lead the company’s sale to Zogenix. Prior to Modis, he was Director of Business & Corporate Development at BioMarin Pharmaceutical. Mr. Mishel also worked in Equity Research at Deutsche Bank and Thomas Weisel Partners, where he covered the specialty pharmaceuticals industry.

Mr. Mishel concluded, “I am thrilled to be joining GenEdit at this important stage as the company transitions its focus from demonstrating proof-of-concept for the platform to selecting initial therapeutic programs for clinical development. I look forward to working with the GenEdit team to ensure that the company’s platform technology and therapeutic programs realize their full potential.”

 

About GenEdit, Inc.

At GenEdit, our mission is to develop curative therapies through targeted in vivo delivery of genetic medicines. Our NanoGalaxy™ platform of non-viral, non-lipid polymer nanoparticles will enable a universe of opportunities to launch the next generation of safe and effective genetic medicines. To bring these medicines to patients, we are developing our own internal pipeline and delivering a wide variety of therapeutic payloads for our partners’ programs. For more info, visit www.genedit.com.

 

Contacts

Company Contact: Kunwoo Lee, Ph.D., GenEdit, Inc., info@genedit.com, +1 (510) 766-2575

Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com, +1 (858) 344-8091

GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery

BERKELEY, Calif., December 11, 2019 – GenEdit, Inc., a developer of a novel polymer nanoparticle technology platform for non-viral- and non-lipid-based delivery of gene therapies, today announced that it has entered into a worldwide, exclusive license and collaboration agreement with Editas Medicine, Inc., a leading genome editing company. GenEdit has developed a comprehensive delivery system for CRISPR-based therapeutics, including gene knockout and gene repair therapies, to enable safer delivery options with improved efficiency.

“This license and collaboration agreement further validates the strength of our intellectual property portfolio and the potential of GenEdit’s technology,” said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. “We are pleased to establish our relationship with Editas Medicine as they leverage our technology to develop potential genomic medicines.”

Under the terms of the agreement, GenEdit has granted Editas Medicine an exclusive worldwide license, with rights to sublicense, to GenEdit’s Cpf1-based technologies. In return for these rights, GenEdit will receive undisclosed upfront and development milestone payments, including royalties on net sales of products incorporating the licensed intellectual property. In addition, GenEdit and Editas Medicine will collaborate on evaluating delivery of Cpf1-based technologies with GenEdit’s nanoparticle platform. Editas Medicine will provide research funding and have an option to continue development after the initial collaboration period.

GenEdit’s nanoparticle platform consists of a proprietary non-viral, non-lipid library of polymers that efficiently encapsulate and deliver cargo [RNA, DNA, protein and/or ribonucleic acid-protein complexes (RNP)] to specific tissues. The company screens the library to identify initial hits and then uses computational analysis and medicinal chemistry for iterative lead optimization. The company has used this platform to identify multiple candidate polymers for efficient and specific delivery of gene editing to a range of tissues.

“Compared to viral vectors and lipid-based nanoparticles, our approach has the potential for better targeting, more cargo, and lower manufacturing cost,” said Timothy Fong, Ph.D., chief scientific officer of GenEdit. “In particular, our approach has the potential to enable in vivo gene editing of multiple tissues with CRISPR and expand the potential of gene therapies to treat more diverse sets of diseases.”

About GenEdit, Inc.
GenEdit was founded to transform the delivery of gene and gene editing therapies. We have synthesized the NanoGalaxy™ library of polymers that can encapsulate RNA, DNA, protein and/or RNP. Through advanced screening methods, computational analysis and iterative medicinal chemistry, we have demonstrated efficient delivery of gene editing cargo to specific tissues. We seek development partnerships for specific tissues and/or gene targets while advancing our internal pipeline of gene editing therapies.

For more information, visit www.GenEdit.com.

Contacts
Kunwoo Lee, Ph.D.
GenEdit, Inc
info@genedit.com