CELL AND GENE THERAPY
Graphite Bio | November 26, 2021
Graphite Bio, Inc. a clinical-stage, next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, announced that the first patient has been enrolled in the company’s Phase 1/2 clinical trial of GPH101, an investigational gene-edited autologous hematopoietic stem cell therapy designed to directly correct the genetic mutation that causes sickle cell disease. The company expects to treat the first patient with GPH101 in the first half of 2022, with initial proof-of-concept data anticipated by the end of 2022.
“GPH101 is the first investigational therapy to enter clinical development that uses our next-generation gene editing platform technology to directly correct the mutation in the beta-globin gene that causes sickle cell disease. Using our gene correction approach, we have demonstrated in preclinical studies an ability to decrease the production of harmful sickle hemoglobin and restore the expression of normal adult hemoglobin. This approach has the potential to restore normal physiology and is viewed as the gold standard for curing sickle cell disease. We are thrilled that our first patient is now enrolled in our CEDAR clinical trial, and we look forward to evaluating GPH101’s potential as we continue to advance its development with urgency in hopes of delivering a curative therapy to the sickle cell community.”
Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio
The CEDAR trial is an open-label, multi-center Phase 1/2 clinical trial of GPH101 designed to evaluate the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics of GPH101 in patients with severe SCD. The trial will enroll approximately 15 adult and adolescent participants at up to five clinical trial sites in the United States.
Graphite Bio will present information about the CEDAR trial at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, being held virtually and in Atlanta December 11-14. The company’s poster presentation will take place on Saturday, Dec. 11, at 5:30-7:30 p.m. ET.
About Sickle Cell Disease (SCD)
SCD is a serious, life-threatening inherited blood disorder that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide. SCD is caused by a single mutation in the beta-globin gene that leads red blood cells to become misshapen, resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life span of approximately 20-30 years. Despite advancements in treatment and care, progressive organ damage continues to cause early mortality and severe morbidity, highlighting the need for curative therapies.
GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore normal adult hemoglobin (HbA) expression, thereby potentially curing SCD.
Graphite Bio is evaluating GPH101 in the CEDAR trial, an open-label, multi-center Phase 1/2 clinical trial designed to assess the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing company harnessing high efficiency targeted gene integration to develop a new class of therapies to potentially cure a wide range of serious and life-threatening diseases. Graphite Bio is pioneering a precision gene editing approach that could enable a variety of applications to transform human health through its potential to achieve one of medicine’s most elusive goals: to precisely “find & replace” any gene in the genome. Graphite Bio’s platform allows it to precisely correct mutations, replace entire disease-causing genes with normal genes or insert new genes into predetermined, safe locations. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.
Ori Biotech | October 15, 2020
Ori Biotech Ltd (Ori), a leading innovator in cell and gene therapy (CGT) manufacturing, today announced the successful close of a $30 million Series A financing round, bringing the company's total funding to date to $41 million. The new funding will be used to help bring Ori's innovative manufacturing platform to the market. The Ori platform delivers scalable solutions to flexibly address the critical clinical and commercial manufacturing needs of CGT developers. The Series A investor syndicate was led by the experienced life sciences investment team at Northpond Ventures, a leading global science, medical, and technology-driven venture fund, alongside Octopus Ventures, a leading European venture fund. Northpond and Octopus invested alongside significant support from Ori's existing institutional investors, Amadeus Capital Partners, Delin Ventures, and Kindred Capital. "Closing a significant Series A round, during these uncertain times, further validates Ori's disruptive approach to fully automating cell and gene therapy manufacturing to increase throughput, improve quality, and decrease costs," said Jason C. Foster, CEO of Ori Biotech. "We are excited to work with our top tier investors and development partners to bring our platform to market as fast as possible to achieve our mission of enabling patient access to life-saving cell and gene therapies." "As early investors in disruptive life sciences platform technology companies, we have seen how cutting-edge technologies like Ori's can bring significant value to the industry and lead to breakthroughs for patients," said Michael Rubin, M.D., Ph.D., Founder and CEO of Northpond Ventures. "We believe that the Ori team has developed a truly innovative technology that can enable millions of patients to gain access to these important treatments." Ori's bespoke platform was designed specifically to address the unique requirements of a new generation of personalized cell and gene therapies. By fully automating and standardizing CGT manufacturing in a closed platform, Ori offers therapeutics developers the opportunity to seamlessly scale from pre-clinical process development to commercial-scale manufacturing. Its flexible platform enables increased throughput, improved quality, and decreased costs.
Cedilla Therapeutics | October 25, 2021
Cedilla Therapeutics a biotechnology company bringing a new dimension to precision oncology, today announced that it has raised an additional $25 million in an expansion of its Series B financing, bringing the total amount raised to $82.6 million. The oversubscribed Series B expansion includes new investors RA Capital Management, Janus Henderson Investors, Woodline Partners LP and Logos Capital, along with participation from existing investor Third Rock Ventures.“We are grateful for the support from our new and existing investors, which reflects the promise of our novel approach to conditionally modulating proteins as well as the unique potential of our lead programs, inhibitors against TEAD and CDK2, two critical but historically undruggable targets,” said Alexandra Glucksmann, Ph.D., President and Chief Executive Officer of Cedilla Therapeutics. “With this financing, we plan to accelerate and expand our development efforts by progressing our most advanced programs toward the clinic while continuing to invest in ongoing discovery efforts against additional high value cancer targets.”
Proceeds from the financing will support Cedilla’s continued growth and development of its two lead programs, an inhibitor of TEAD for the treatment of solid tumors, such as mesothelioma and certain squamous cell carcinomas, and a highly selective inhibitor of CDK2/Cyclin E for the treatment of multiple tumor types, including CDK4/6-resistant breast cancer, ovarian, uterine, stomach and esophageal cancers. The company plans to initiate investigational new drug application-enabling studies for the TEAD program in the first half of 2022 and for the CDK2 program in the second half of 2022.
In conjunction with the financing, Jake Simson, Ph.D., Partner at RA Capital Management, joins Cedilla’s Board of Directors.
Dr. Glucksmann continued, “We are pleased to welcome Jake to our Board of Directors. He brings a wealth of experience advising companies from the earliest stages of company formation through their maturation into fully integrated biopharmaceutical organizations. We look forward to his many contributions as we continue to grow Cedilla into a clinical-stage organization with a robust and growing portfolio of product candidates.”
“Cedilla brings a new dimension to precision oncology by conditionally modulating proteins in their functional state Preclinical data generated to-date suggest that Cedilla’s lead programs may challenge historical perceptions of TEAD and CDK2 as undruggable targets and could offer patients new options that would be well-tolerated and extremely effective. In addition, Cedilla’s approach, based on a deep understanding of how the cellular context modulates protein activity, could deliver superior inhibitors against a range of other key cancer drivers. I look forward to partnering with the team with the aim to deliver a broad portfolio of small molecule medicines, each with potential profound benefit to patients.”
Jake Simson, Ph.D., Partner at RA Capital Management
About Cedilla Therapeutics
Cedilla Therapeutics is bringing a new dimension to precision oncology. We have discovered new ways to selectively inhibit oncogenic drivers with small molecules that conditionally modulate proteins in their functional state. Our conditional inhibitors are unlocking critical and elusive cancer targets including TEAD and CDK2. We are a driven and patient-focused team. x