On October 4, 2022 miRecule, Inc., an innovator of next-generation RNA therapeutics, reported a strategic collaboration and exclusive license agreement with Sanofi to develop and commercialize a best-in-class antibody-RNA conjugate (ARC) for the treatment of facioscapulohumeral muscular dystrophy (FSHD) (Press release, miRecule, OCT 4, 2022, View Source [SID1234621663]). The collaboration marks miRecule’s first licensing transaction leveraging its proprietary DREAmiR platform.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

FSHD is the second most common type of muscular dystrophy – affecting more than one million individuals worldwide with no approved treatments. Patients with causative genetic mutations experience lifelong deterioration of muscle function and progressive disability. The collaboration will combine miRecule’s anti-DUX4 RNA therapy (discovered through its proprietary DREAmiR platform) with Sanofi’s proprietary muscle-targeted NANOBODY technology to join the two molecules into an ARC utilizing miRecule’s NAVIgGatorTM conjugation and formulation chemistry. The potential resulting best- in-class therapy may become a disease-modifying treatment that selectively targets and suppresses the underlying cause of FSHD in muscle tissue. This groundbreaking approach would enable FSHD patients to resume their normal course of aging free from the debilitating effects of this progressive disease.

Under the terms of the collaboration agreement, miRecule will grant Sanofi an exclusive worldwide license to intellectual property rights to the FSHD therapy. miRecule and Sanofi will collaborate on research activities through lead candidate selection. Following candidate selection, Sanofi will assume sole responsibility for IND enabling studies and subsequent development and commercialization activities globally. miRecule will receive an upfront payment and is eligible for near-term milestone payments, which combined could exceed $30 million. Additional development, regulatory, and commercial milestone payments could raise the total to nearly $400 million for advancement of the FSHD drug candidate, which will be exclusively developed and commercialized by Sanofi. miRecule is also eligible to receive tiered royalties on global net sales of the approved collaboration product.

Pablo Sardi
Global Head of Rare and Neurologic Diseases Research, Sanofi

"We look forward to working with miRecule to bring together our two groundbreaking technologies synergizing in a best-in-class therapy designed to suppress the underlying cause of FSHD. We hope that this will enable patients to live a life free from the debilitating symptoms of the disorder. We are excited to embark on this collaboration with miRecule as we work together to bring hope to the FSHD community."

Anthony Saleh
Founder and Chief Executive Officer, miRecule

"We are thrilled to enter our first major licensing transaction with a partner of Sanofi’s caliber and capabilities as a global leader in the development and commercialization of rare disease therapies. Our goal since initiating this program has been to develop a treatment to allow FSHD patients to live a normal life. The philosophy driving our DREAmiR discovery platform focuses on patient centered drug development and strong scientific decision making. We believe Sanofi shares these core values on creating life changing therapies, making this is an ideal partnership to bring forward our anti-DUX4 RNA therapy."

miRecule’s founder and CEO has a strong commitment to developing a therapy to treat FSHD as the disorder runs in his family. miRecule started its program in 2019 with strong support from the patient and academic community, receiving funding from the FSHD Society, the Friends of FSH Research, and National Institute of Neurological Disorders and Stroke (NINDS, Grant 5R44NS119147-02). Sanofi and miRecule look forward to continuing to work with these organizations to help bring forward a groundbreaking treatment for FSHD.

On October 4, 2022 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported that Brian M. Culley, Lineage’s Chief Executive Officer, will present at the Alliance for Regenerative Medicine 2022 Cell & Gene Meeting on the Mesa, on October 12th, 2022 at 2:15pm PT / 5:15pm ET at the Park Hyatt Aviara, Carlsbad, CA (Press release, Lineage Cell Therapeutics, OCT 4, 2022, View Source [SID1234621662]). Virtual meeting attendance is available and includes a livestream of Lineage’s presentation and the ability to view all conference sessions on-demand. Interested parties can visit the 2022 Cell & Gene Meeting on the Mesa website for full information on the conference, including registration.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Cell & Gene Meeting on the Mesa is the sector’s foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Tackling the commercialization hurdles facing the cell and gene therapy sector today, this meeting covers a wide range of topics from clinical trial design to alternative payment models to scale-up and supply chain platforms for advanced therapies. The program features expert-led panels, extensive partnering capabilities, exclusive networking opportunities, and dedicated presentations by the leading publicly traded and privately held companies in the space. This conference enables key partnerships through more than 3,000 one-on-one meetings while highlighting the significant clinical and commercial progress in the field.

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-engineered therapies. In its 13-year history, ARM has become the global voice of the sector, representing the interests of 450+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups.

On October 4, 2022 Aurobac Therapeutics SAS (Aurobac), reported the appointment of Florence Séjourné as Chief Executive Officer (Press release, Evotec, OCT 4, 2022, View Source [SID1234621661]). Aurobac is a joint venture recently created by Boehringer Ingelheim, Evotec SE and bioMérieux, focusing on the development of a new precision medicine approach, from diagnosis to cure to fight Antimicrobial Resistance (AMR).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Florence’s is an experienced biotech leader with outstanding strategic and operational acumen. The Board has great confidence in her to successfully set-up and lead Aurobac," said Frank Kalkbrenner, Ph.D., Co-Founder and Member of the Board of Aurobac. "We are excited to welcome Florence to Aurobac and look forward to her building a strong team under her leadership to put Aurobac on track for growth and success."

Mrs. Séjourné brings 25 years of biotech experience, most recently in leadership roles in the field of Antimicrobial Resistance (AMR). She is founder and board member of the BEAM Alliance, an association representing European biotechs developing new solutions to fight against AMR. Further she has been actively involved in advocating for biopharmaceutical small and medium-sized enterprise (SME) compatible policies with worldwide stakeholders to stimulate much needed innovation in AMR for the last 8 years.

She joins Aurobac from Da Volterra, where she served as CEO and brought the company from bench scientific concepts to late-clinical stage, developing products to protect the intestinal microbiota from the deleterious effects of antibiotics, including prevention of infections and antibacterial resistance. Prior to this role, Mrs Séjourné co-founded and worked as CBO/COO of Genfit (GNFT), a biotech company focused on metabolic and liver related diseases. She holds engineering and pharmaceutical sciences degrees from MINES ParisTech and the University of Illinois at Chicago.

"Aurobac has the remarkable ambition to become a worldwide leader in AMR innovation with a unique combination of the best capabilities of the three founding companies towards developing a new precision medicine approach, from diagnosis to cure" said Mrs. Séjourné. "I am thrilled to join the initiative next to the founding partners and transform this fantastic vision into new drugs for patients."

On October 4, 2022 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported that Rick Fair, President and CEO, will present at the annual Cell & Gene Meeting on the Mesa to be held October 11-13 in Carlsbad, CA and livestreamed globally (Press release, Bellicum Pharmaceuticals, OCT 4, 2022, View Source [SID1234621659]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Details regarding Bellicum’s presentation at the conference are as follows:

Event: 2022 Cell & Gene Meeting on the Mesa
Date: October 11, 2022
Time: 4:30 p.m. PT
Location: Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

Virtual attendance is available which includes a livestream of Bellicum’s presentation and the ability to view all conference sessions on-demand. Please visit View Source for full information including registration.

Complimentary attendance at this event is available for credentialed investors and members of the media only. Investors should contact Laura Stringham at [email protected] and interested media should contact Stephen Majors at [email protected].

About the Cell & Gene Meeting on the Mesa

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 90 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

On October 4, 2022 AIVITA Biomedical, Inc., a biotech company specializing in innovative cell applications, reported that the Journal of Oncology Research and Therapy has published data from its completed Phase 2 glioblastoma (GBM) study which demonstrates enhanced progression-free survival (PFS) (Press release, AIVITA Biomedical, OCT 4, 2022, View Source [SID1234621657]). The publication, titled "Enhanced Progression-Free Survival in a Phase 2 Trial of Personal Dendritic Cell Vaccines in Patients with Newly Diagnosed Glioblastoma" can be viewed online at: View Source

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The strengths of our approach include its lack of toxicity and the ability to pair it with other treatment modalities such as chemotherapy, inhibitors of angiogenesis and immune checkpoints," said Robert O. Dillman, chief medical officer at AIVITA.

AIVITA completed a multi-center Phase 2 trial of its immunotherapy candidate, AV-GBM-1, which had demonstrated an encouraging 50% improvement in PFS over standard of care in patients with a recent diagnosis of GBM. This publication focuses on PFS resulting from AV-GBM-1 administration and compares the results to those reported in GBM trials of other therapies. The data suggest that continuous treatment could further improve progression-free survival and overall survival.

"AIVITA has developed an inexpensive, pain-free personal immunotherapy that meaningfully increases survival," said Hans S. Keirstead, chairman and chief executive officer of AIVITA. "These data are a significant milestone in our quest to make GBM a non-lethal and manageable disease."