Scientists back temporary global ban on gene-edited babies

Medical Xpress | March 13, 2019

An international group of scientists and ethicists on Wednesday called for a temporary global ban on making babies with edited genes. It's the latest reaction to last November's announcement that gene-edited twins had been born in China, which was widely criticized. Mainstream scientists generally oppose making babies with altered DNA now, citing safety and ethical issues that must be addressed first. Such genetic changes may be passed to future generations, unlike gene editing done in parts of the body not involved in reproduction. So news last year that Chinese scientist He Jiankui claimed to have edited DNA of embryos provoked widespread condemnation. Some scientists had called for a moratorium before the latest proposal, which carries no legal authority. It came from 18 researchers from seven countries who published a commentary in the journal Nature. They included prominent gene-editing experts Feng Zhang and David Liu of the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. They receive money from the Howard Hughes Medical Institute, which also supports The Associated Press Health & Science Department.

Spotlight

May.19 -- Bluebird Bio CEO Nick Leschly discusses gene therapy, Boston's biotech scene, and venture capital with Bloomberg's Caroline Hyde on "Bloomberg Technology" at the Museum of Science in Boston on May 9.

Spotlight

May.19 -- Bluebird Bio CEO Nick Leschly discusses gene therapy, Boston's biotech scene, and venture capital with Bloomberg's Caroline Hyde on "Bloomberg Technology" at the Museum of Science in Boston on May 9.

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MEDTECH

Entos and BioMarin Enter into Agreement for Product Candidates Incorporating Entos' Fusogenix Drug Delivery Platform

Entos Pharmaceuticals, Inc., BioMarin | November 16, 2021

Entos Pharmaceuticals, Inc. (Entos), a clinical-stage biotechnology company developing genetic medicines with its Fusogenix proteolipid vehicle (PLV) nucleic acid delivery platform, and BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that they have entered into an agreement that will see Entos apply its Fusogenix nucleic acid delivery technology to promising gene therapy candidates in the BioMarin pipeline. Under the agreement, Entos will create specially formulated product candidates for BioMarin, a world leader in developing and commercializing innovative biopharmaceuticals for genetic diseases driven by genetic causes. Entos' Fusogenix platform utilizes a PLV formulation with a novel mechanism of action to deliver molecular payloads, intact and unmodified, directly into target cells. By combining well-tolerated neutral lipids with proprietary fusion-associated small transmembrane (FAST) proteins, the Fusogenix platform efficiently delivers mRNA or DNA to target tissue with excellent tolerability. Fusogenix technology has been applied to a wide range of therapeutic approaches, including vaccines, gene therapy, and gene editing. "As a company committed to addressing the unmet therapeutic needs of patients living with genetic diseases, BioMarin values novel technologies that enable the development of transformative therapies," said Brinda Balakrishnan, M.D., Ph.D., Group Vice President, Corporate and Business Development at BioMarin. "We believe that Entos' Fusogenix platform offers potentially unique benefits for safe and effective tissue targeting compared with other lipid-based delivery systems. The Fusogenix PLV formulations generated under this agreement are a critical first step in determining how we may incorporate this promising platform into our drug development efforts." "With a 20-year track record of successfully discovering, developing, and commercializing first- or best-in-class therapies for diverse rare diseases, the BioMarin team has extensive expertise in harnessing the potential of breakthrough technologies. We believe this licensing agreement helps validate the potential of our Fusogenix platform. It also provides additional opportunities for incorporating our technology into cutting-edge treatments that may have significant clinical and commercial value." John Lewis, Ph.D., Founder and Chief Executive Officer of Entos Under the terms of the agreement, Entos will use its Fusogenix nucleic acid delivery system to specially formulate BioMarin products directed at multiple undisclosed genetic disease indications. BioMarin will conduct preclinical studies of the Fusogenix-formulated candidates to evaluate their potential as therapies to prevent or treat these conditions. Entos' most advanced clinical program is Covigenix VAX-001, a COVID-19 DNA vaccine encoding SARS-CoV-2 Spike protein and two genetic adjuvants engineered to stimulate host adaptive and innate immune systems. Formulated using the Fusogenix platform, Covigenix VAX-001 is the subject of an ongoing phase 2 clinical trial in South Africa, with additional trials planned in other regions. Licensees of the Fusogenix platform include Oisín Biotechnologies and OncoSenX, which are using the technology for age-related diseases and oncology applications, respectively. pH Partners, LLC served as financial advisor to Entos. About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and preclinical product candidates. About Entos Pharmaceuticals, Inc. Entos develops next generation nucleic acid-based therapies using its proprietary Fusogenix proteolipid vehicle (PLV) drug delivery system. Fusogenix is formulated with FAST proteins to deliver mRNA or DNA directly into the cytosol of target cells for translation into protective and therapeutic vaccines and medicines.

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INDUSTRIAL IMPACT

AMYRIS TO ACQUIRE MENOLABS FOR MENOPAUSE WELLNESS ACCELERATING GROWTH OF CONSUMER BUSINESS

Amyris, Inc. | January 25, 2022

Amyris, Inc. a leading synthetic biotechnology company accelerating the world's transition to sustainable consumption through its Lab-to-Market™ operating platform announces that it has agreed to exclusive terms to acquire the assets of MenoLabs, LLC, a women-founded company to treat menopause symptoms, drive research for women's health and improve society's understanding of menopause. Amyris previously communicated its intention of launching a new consumer brand to provide science-backed, wellness and personal care products for the high growth menopause market in 2022. The acquisition of MenoLabs will serve as a catalyst to accelerate growth and establish a leadership position in the fast-growing menopause market. MenoLabs, a growing women's wellness brand is focused on addressing perimenopause and menopause symptoms. MenoLabs is one of few companies offering research-backed all-natural treatments of menopause symptoms. Over the past two years, MenoLabs has developed and launched eight products, including its lead product, MenoFit, an all-natural menopause relief supplement. MenoLabs generates a subscription-based, recurring revenue stream via its website, in addition to the Subscribe & Save option offered via the Amazon channel. The company is further expanding its omnichannel by penetrating retail channels in 2022. Additionally, MenoLabs launched MenoLife, a highly rated perimenopause and menopause health tracker app which helps users track and analyze more than 40 menopause-related symptoms. MenoLife also has created an online forum where users can comment and react to posts. Menopause symptoms impact the lives of approximately 25 million women per year and 6,000 women in the U.S. enter perimenopause every day (2.2 million each year). By 2025, there will be over one billion people experiencing menopause in the world or around 12% of the entire world population. Stigma, lack of open discussion, and a small product offering at an affordable price are barriers to care for women. The menopause market was valued at around $15 billion in 2020 and is expected to reach approximately $23 billion by 2028. "We are very impressed with the MenoLabs offering which is synergistic with our science-based approach to real-world health and wellness issues. We are committed to empowering women and creating a platform to support them with our leading science and wellness technology platform. Women deserve products and solutions that work during this period in their lives. Our acquisition of the products and app that the MenoLabs team has built accelerates Amyris' entry into the menopause market. We expect the combination of MenoLabs and the previously announced menopause brand with Naomi Watts to have the potential to deliver an estimated $30 million in revenue in their first year and expect significant growth potential in the years ahead. We continue to execute on our growth strategy diversifying our consumer offering in Clean Beauty, Health and Wellness end-markets, continuing the strong growth momentum from 2021 into the current quarter." John Melo, President and Chief Executive Officer of Amyris About Amyris Amyris is a leading synthetic biotechnology company, transitioning the Clean Health & Beauty and Flavors & Fragrances markets to sustainable ingredients through fermentation and the company's proprietary Lab-to-Market™ operating platform. This Amyris platform leverages state-of-the-art machine learning, robotics and artificial intelligence, enabling the company to rapidly bring new innovation to market at commercial scale. Amyris ingredients are included in over 20,000 products from the world's top brands, reaching more than 300 million consumers. Amyris also owns and operates a family of consumer brands that is constantly evolving to meet the growing demand for sustainable, effective and accessible products. About MenoLabs MenoLabs was founded to fundamentally change how menopause is addressed. The brand's mission is to provide options to treat menopause symptoms, drive research for women's health and change how society approaches, views and educates around the topic of menopause. MenoLabs has developed a line of all natural, proprietary supplements focused on treating perimenopause and menopause symptoms, in addition to MenoLife, a leading menopause health tracker app that provides women a voice and community. MenoLife also provides symptom tracking to further fuel research and advancements in women's health.

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MEDTECH

Patritumab Deruxtecan Granted U.S. FDA Breakthrough Therapy Designation in Patients with Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

Daiichi Sankyo | December 24, 2021

Daiichi Sankyo Company, Limited announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to patritumab deruxtecan, a potential first-in-class HER3 directed antibody drug conjugate, for the treatment of patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer with disease progression on or after treatment with a third-generation tyrosine kinase inhibitor and platinum-based therapies. Lung cancer is the second most common cancer and the leading cause of cancer-related mortality worldwide, with 80% to 85% classified as NSCLC.1,2,3 While the efficacy of targeted therapy with EGFR TKIs is well-established in the treatment of advanced EGFR-mutated NSCLC, which comprises approximately 30% of patients, the development of a broad range of resistance mechanisms commonly leads to disease progression.4,5,6 After failure of an EGFR TKI, platinum-based chemotherapy has limited efficacy with progression-free survival (PFS) of approximately 4.4 to 6.4 months.7 Subsequent salvage therapies after EGFR TKI and platinum-based chemotherapy have PFS of 2.8 to 3.2 months.8 The U.S. FDA’s BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines. The FDA granted the BTD based on data from the dose escalation portion and two expansion cohorts of a three-cohort phase 1 study of patritumab deruxtecan. Extended follow-up data from the dose escalation portion and dose expansion cohort 1 of the study were recently presented at the 2021 American Society of Clinical Oncology annual meeting and published in Cancer Discovery. This is the first BTD for patritumab deruxtecan and the seventh BTD across Daiichi Sankyo’s oncology portfolio. “The Breakthrough Therapy Designation for patritumab deruxtecan acknowledges the need for new treatment approaches to overcome resistance and improve survival in patients with metastatic TKI-resistant, EGFR-mutated non-small cell lung cancer. We are proud that the FDA has once again recognized our innovative science and technology and we look forward to bringing this potential first-in-class HER3 directed antibody drug conjugate to patients with this specific type of lung cancer as quickly as possible.” Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo About Non-Small Cell Lung Cancer Lung cancer is the second most common cancer and the leading cause of cancer-related mortality worldwide, with 80% to 85% classified as NSCLC.1,2,3 There were an estimated 2.2 million new cases of lung cancer and 1.8 million deaths in 2020.9 NSCLC is diagnosed at an advanced stage in more than 50% of patients and often has a poor prognosis with worsening outcomes after each line of subsequent therapy.10,11,12 The introduction of targeted therapies and checkpoint inhibitors in the past decade has improved the treatment landscape for patients with advanced or metastatic NSCLC. For patients with advanced EGFR-mutated NSCLC, targeted therapy with EGFR TKIs offer higher response rates and PFS compared to chemotherapy.13 However, most patients eventually develop resistance to these therapies and subsequent therapy after EGFR TKI with platinum-based chemotherapy have limited efficacy with PFS of approximately 4.4 to 6.4 months.7,14 Subsequent salvage therapies after EGFR TKI and platinum-based chemotherapy have PFS of 2.8 to 3.2 months.8 New treatment approaches are needed to overcome resistance and improve survival in this subtype of NSCLC. About HER3 HER3 is a member of the EGFR family of receptor tyrosine kinases, which are associated with aberrant cell proliferation and survival.15 Approximately 25% to 30% of lung cancers have an EGFR-activating mutation, and it is estimated that about 83% of all NSCLC tumors express the HER3 protein, which can be associated with an increased incidence of metastases, reduced survival and resistance to standard of care treatment.16,17,18 Currently, no HER3 directed medicines are approved for the treatment of cancer. About the Phase 1 Non-Small Cell Lung Cancer Study The global, multicenter, open label, two-part phase 1 study is evaluating patritumab deruxtecan in previously treated patients with metastatic or unresectable NSCLC. The dose escalation part of the study evaluated patients with EGFR-mutated disease either with progression on osimertinib or T790M-negative after progression on erlotinib, gefitinib or afatinib. The primary objective of this part of the study was to assess the safety and tolerability of patritumab deruxtecan and determine the recommended dose for expansion. The dose expansion part of the study is evaluating patritumab deruxtecan at the RD in three cohorts. Cohort 1 includes patients with locally advanced or metastatic EGFR-mutated NSCLC who experienced disease progression after taking one or more EGFR TKIs and one or more platinum-based chemotherapy regimens. Cohort 2 includes patients with squamous or non-squamous NSCLC without EGFR-activating mutations following platinum-based chemotherapy and following an anti-PD-1 or anti-PD-L1 antibody regimen. Cohort 3 includes patients with NSCLC with EGFR-activating mutations including any histology other than combined small cell and non-small cell lung cancer; patients in Cohort 3 are randomized 1:1 to receive the 5.6 mg/kg RDE regimen or an escalating up-titration regimen of patritumab deruxtecan. The primary objective of the dose expansion part of the study is to assess efficacy of patritumab deruxtecan as measured by confirmed objective response rate assessed by blinded independent central review. Secondary study endpoints include investigator-assessed ORR, safety and pharmacokinetics. The study enrolled patients at multiple sites in Asia, Europe and North America. For more information, visit ClinicalTrials.gov. About Patritumab Deruxtecan Patritumab deruxtecan is one of three lead DXd ADCs in the oncology pipeline of Daiichi Sankyo. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, patritumab deruxtecan is comprised of a fully human anti-HER3 IgG1 monoclonal antibody attached to a topoisomerase I inhibitor payload via a stable tetrapeptide-based cleavable linker. Patritumab deruxtecan is currently being evaluated in a comprehensive development program across multiple cancers as both a monotherapy and in combination with other anticancer treatments. The development program includes HERTHENA-Lung01, a pivotal phase 2 study in patients with locally advanced or metastatic EGFR-mutated NSCLC previously treated with a TKI and platinum-based chemotherapy; a phase 1/2 study in HER3 expressing metastatic breast cancer; a phase 1 study in combination with osimertinib in locally advanced/metastatic EGFR-mutated NSCLC; and, a phase 1 study in previously treated patients with metastatic or unresectable NSCLC. Patritumab deruxtecan is an investigational medicine that has not been approved for any indication in any country. Safety and efficacy have not been established. About Daiichi Sankyo in Oncology The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon, our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025. About Daiichi Sankyo Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.”

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