CHICAGO, May 25, 2021 /PRNewswire/ -- In-depth analysis and data-driven insights on the impact of COVID-19 included in this global regenerative medicine market report.

The regenerative medicine market is expected to grow at a CAGR of over 30% during the period 20202026.

Key Highlights Offered in the Report:

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Regenerative Medicine Market Segmentation

Regenerative Medicine Market by Application

Regenerative Medicine Market by Product

Regenerative Medicine Market by End-Users

Regenerative Medicine Market Dynamics

Regenerative medicine is expected to evolve and impact the overall healthcare industry in a positive way in the coming years. Among the global pharmaceutical companies, nearly 1000 companies are working on gene therapy, cell therapy, and tissue-engineering therapeutic products. Many companies worldwide have been developing a wide array of scaffolds that can be used in different tissue engineering applications, which cater to patients who require tissue and organ substitutes. The advances in scaffolds are attributable to several innovations in tissue scaffolds, bone scaffolds, and dental scaffolds. Tissue scaffolds basically act by integrating local cells in the desired shape of the scaffold after implantation. The scaffolds are of different types, such as cellusponge scaffolds in which cells are distributed in sponge pores and start growing. Collagen scaffolds have a unique porous network that allows diffusion of nutrients for cell growth, while hydrogel scaffolds have water content similar to natural tissue. Nanofiber scaffolds are transparent and ease cell imaging and quantification of cells.

Key Drivers and Trends fueling Market Growth:

Regenerative Medicine Market Geography

In 2020, North America accounted for a share of over 62% in the global regenerative medicine market. The region is expected to grow at a significant rate during the forecast period due to the highest number of RM companies in the world. The region has nearly 534 of the 987 RM companies worldwide. The growth is primarily attributable to the increasing incidence rates of different types of cancers such as non-Hodgkin lymphoma, Hodgkin lymphoma, melanoma of the skin, leukemia, and rare disorders, including Spinal muscular atrophy and multiple sclerosis. Cancer is the leading cause of death in North America. In 2018, nearly 1.9 million new cancer cases were reported in the North American region, along with 693,000 deaths. In the North American region, the US shows the highest prevalence rate for cancers such as non-Hodgkin lymphoma and other life-threatening rare diseases.

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Regenerative Medicine Market by Geography

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Regenerative Medicine Market Size to Reach Revenues of over USD 27 Billion by 2026 - Arizton - PRNewswire

WASHINGTON, May 24, 2021 /PRNewswire/ --An article published in Experimental Biology and Medicine (Volume 246, Issue 10, May, 2021), describes a new treatment strategy for glioma.The study, led by Dr. Julun Yang, in the Department of Pathology at the 920th Hospital of the Joint Logistics Support Force of PLA in Kunming (China), reports that targeting RAS, a gene that promotes tumorigenesis in multiple cancers, enhances antitumor activity against glioma xenografts.

Gliomas are the most common type of central nervous system tumor in humans, with approximately 250,000 cases reported annually worldwide. Current treatment options include aggressive surgical resection, radiation therapy and chemotherapy.Nonetheless, the survival rate for patients is poor, and new treatments are needed.RAS genes play a critical role in regulating cell proliferation, differentiation, migration, apoptosis and senescence.Overexpression of and mutations in RAS genes promote tumorigenesis in numerous types of cancers, including gliomas.Nevertheless, there are no therapies targeting RAS approved for use in patients.

In the current study, Dr. Yang and colleagues used a gene therapy approach to deliver antibodies that inhibit RAS to glioma cells and tumors.Adenovirus delivery of a RAS antibody gene to glioma cells resulted in anti-RAS antibody expression, decreased growth and proliferation, and increased cell death.Intravenous delivery using cytokine-induced killer cells resulted in expression of high levels of RAS antibodies in tumors and low levels in normal organs.Furthermore, tumor volume was reduced and accompanied by decreased cell proliferation and expression of anti-apoptotic genes as well as increased cell death and expression of pro-apoptotic genes.Collectively, these results suggest that gene therapy targeting RAS may be a safe and effective treatment for glioma and other RAS-driven cancers.

Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology & Medicine, said, "Dr. Yang and colleagues have performed in vitro and in vivo studies that provide compelling evidence that the delivery of anti-p21Ras scFv by recombinant adenovirus and cytokine-induced killer cells may be an effective therapy against gliomas and, by extension, other Ras-driven cancers."

Experimental Biology and Medicine is a global journal dedicated to the publication of multidisciplinary and interdisciplinary research in the biomedical sciences. The journal was first established in 1903. Experimental Biology and Medicine is the journal of the Society of Experimental Biology and Medicine. To learn about the benefits of society membership, visit http://www.sebm.org. If you are interested in publishing in the journal, please visit http://ebm.sagepub.com.

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Using Gene-Therapy to Target RAS in Gliomas - PRNewswire

GenSight Biologics has reported the partial recovery of the vision of a retinitis pigmentosa patient treated with its mutation-independent approach to the restoration of visual function.

Spark Therapeutics, now part of Roche, won FDA approval for its retinitis pigmentosa gene therapy Luxturna in 2017. However, the approval only covered the use of Luxturna in patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. As retinitis pigmentosa is caused by changes to more than 70 genes, it is questionable whether mutation-specific approaches can cover all patients.

Recognizing that, GenSight is developing GS030, an optogenetic therapy. The drug component of the intervention consists of a viral vector designed to cause the expression of the light-sensitive opsin ChrimsonR in retinal ganglion cells.

In a phase 1/2 clinical trial, physicians administered the gene therapy via a single intravitreal injection to the worse-seeing eye of a 58-year-old man who was diagnosed with retinitis pigmentosa 40 years ago and was unable to detect objects visually. The patient was still unable to detect objects visually after the injection.

However, the patient could perceive, locate, count and touch different objects using his treated eye while wearing a pair of goggles. The goggles detect changes in light intensity and send pulses of light to the retina in real time, effectively amplifying the signal sent to the ChrimsonR-expressing cells.

Writing in Nature Medicine, the investigators said the patient is the first reported case of partial functional recovery in a neurodegenerative disease after optogenetic therapy. The claim is based on three visual tests, the last of which was performed five months after the others, that took place in an indoor laboratory, but there is also patient-reported evidence of real-world benefits.

The patient spontaneously reported identifying crosswalks and he could count the number of white stripes. Subsequently, the patient testified to a major improvement in daily visual activities, such as detecting a plate, mug or phone, finding a piece of furniture in a room or detecting a door in a corridor but only when using the goggles, the authors of the research paper wrote.

The paper only describes the experience of one patient, and the recovery in his vision was partial. GenSight recruited six patients across the first two cohorts of the dose-escalation trial before putting enrollment on hold in response to the pandemic. Enrollment in the third cohort is now complete, and GenSight expects to recruit an extension cohort that will get the optimal dose by the end of 2021.

A clearer picture of the efficacy of GS030 will emerge as GenSight shares data on other recipients of the treatment. Other companies are pursuing the same opportunity. Novartis, for example, bought optogenetic gene therapy startup Vedere Bio for $150 million upfront last year.

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GenSight's mutation-agnostic gene therapy triggers partial vision recovery - FierceBiotech

BOSTON, May 24, 2021 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced the appointment of leading experts in gene therapy, cochlear development and molecular genetics to its Scientific Advisory Board (SAB).

These additions to our SAB bring a deep understanding of the genetic roots of hearing loss and balance disorders, and we look forward to working closely with them as we progress our pipeline of gene therapies and regenerative medicines, said Joe Burns, Ph.D., Vice President, Discovery.

We value their insights and tremendous experience in the translational considerations for the development of gene therapies as Decibel works to address the vast unmet needs in hearing and balance disorders, said John Lee, Chief Development Officer.

Career summaries of the Scientific Advisory Board appointees:

Connie Cepko, Ph.D., is the Bullard Professor of Genetics and Neuroscience at Harvard Medical School and a Howard Hughes Medical Institute Investigator. She trained in virology with Dr. Phillip Sharp at MIT for a Ph.D., and later with Dr. Richard Mulligan at the MIT Whitehead Institute. She helped develop retroviral vectors for transduction into the central nervous system (CNS) for lineage analysis and for studies of gene function in vivo. Her laboratory has focused on the mechanisms of cell fate determination in the CNS with focus on retina through the analysis of progenitor and stem cells. More recently, she has been studying the mechanisms of photoreceptor death in diseases that cause blindness, such as retinitis pigmentosa and macular degeneration, and is developing gene therapies to avert photoreceptor death in order to prevent vision loss.

GuangpingGao, Ph.D.,is an internationally recognized researcher who played a key role in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes to advance the gene therapy field. He has published extensively in the field, with more than 300 papers, and holds more than 191 patents, with hundreds more pending. The Penelope Booth Rockwell Professor in Biomedical Research at the University of Massachusetts Medical School, Dr. Gao is an elected fellow of both the U.S. National Academy of Inventors and the American Academy of Microbiology. He is the Past President of the American Society of Gene and Cell Therapy. Dr. Gao co-founded Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics to develop AAV-based gene therapies for rare diseases.

Matthew Kelley, Ph.D., directs the Laboratory of Cochlear Development in the Intramural Program at the National Institute on Deafness and Other Communication Disorders, National Institutes of Health. A widely published and well-respected research scientist, he focuses on the cellular and molecular development of the mammalian cochlea. Dr. Kelley has long been an active member and is the past President of the Association for Research in Otolaryngology (ARO).

Glenn Pierce, M.D., Ph.D.,is Entrepreneur-in-Residence at Third Rock Ventures. He has 30 years of experience in drug discovery and developmentwith a particular focus on tissue regeneration, gene therapy and hematologyand has contributed to the development of six marketed products.As the former Chief Medical Officer, Hemophilia Therapeutic Area at Biogen, he led work culminating in multiple regulatory approvals for hemophilia therapeutics. Dr. Pierce has served in multiple leadership roles for the National Hemophilia Foundation as well as on advisory boards for the U.S. Food and Drug Administration and the U.S. Department of Health and Human Services. He has co-authored more than 150 scientific papers and has received more than 15 patents. Dr. Pierce serves on the Boards of Directors of Global Blood Therapeutics and Voyager Therapeutics.

Dinah Sah, Ph.D., is an accomplished drug developer and R&D leader with over 25 years of experience in research and drug development in the biotechnology industry. Most recently, she was Chief Scientific Officer at Voyager Therapeutics, joining soon after its start in 2014. Prior to Voyager, Dr. Sah was Vice President of Research at Alnylam, overseeing many of the research programs during her seven-year tenure. She has successfully led multiple research and preclinical programs toward and into clinical development across new modalities, including the CNS-focused AAV programs at Voyager and the groundbreaking novel class of RNAi therapeutics developed at Alnylam.

About Decibel TherapeuticsDecibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibels pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visitwww.decibeltx.comor follow us onTwitter.

Investor Contact:Julie SeidelStern Investor Relations, Inc.julie.seidel@sternir.com212-362-1200

Media Contact:Chris RaileyTen Bridge CommunicationsChris@tenbridgecommunications.com617-834-0936

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Decibel Therapeutics Expands World-Class Scientific Advisory BoardAdds leaders with expertise in hearing loss and balance disorders, combined with...

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the highly-regarded journal Nature Medicine has published the first case report of partial recovery of visual function in a blind patient with late stage retinitis pigmentosa (RP). The subject is a participant in the ongoing PIONEER Phase I/II clinical trial of GenSight Biologics GS030 optogenetic therapy. Published in the May issue under the title Partial recovery of visual function in a blind patient after optogenetic therapy, the paper* is the first peer-reviewed documentation of visual recovery after a blind patient was treated with optogenetic therapy.

These are truly groundbreaking findings that move the promise of optogenetics another step from therapeutic concept to clinical use, commented Bernard Gilly, Co-Founder and Chief Executive Officer of GenSight. These could not have occurred without the close collaboration we enjoyed with our partners at the Institut de la Vision, the Institute of Ophthalmology Basel and Streetlab. We are especially grateful to the patients who are participating in our trial, whose experiences and input will help us design the next stage of GS030s clinical development. We will now accelerate the GS030 program to make it our second product to reach the market after LUMEVOQ.

Optogenetic therapies combine cellular expression of light-sensitive opsins with light stimulation using a medical device. GS030 uses an optimized viral vector (GS030-DP) to express the light-sensitive opsin ChrimsonR in retinal ganglion cells and proprietary light-stimulating goggles (GS030-MD) to project the right wavelength and intensity of light onto the treated retina. GS030-DP is administered via an intravitreal injection.

It was breathtaking to witness the first recovery of some visual function in a blind patient, commented Dr. Botond Roska, MD, PhD, last and co-corresponding author and a pioneer in the field of optogenetic vision restoration. Dr. Roska is Founding Director of the Institute of Molecular and Clinical Ophthalmology Basel (IOB) in Switzerland and a Co-Founder of GenSight. We have worked on optogenetic therapy in the lab for 16 years and now seeing the proof of concept in a patient is a unique experience, he said. I am most grateful to have shared this long journey with Jos Sahel, a fellow founder of GenSight; the dedicated team at GenSight; and our other collaborators.

The subject in the case report, who had been diagnosed with RP 40 years prior to enrollment, had such low visual acuity that prior to receiving GS030, he could only perceive light. His gene therapy injection was followed four and a half months later by training on the use of the GS030-MD device. Seven months after the start of his training, he began to report signs of visual improvement. Visual function tests showed he acquired the ability to perceive, locate, count and touch objects when his treated eye was stimulated with the GS030-MD goggles. Without the goggles, he could not perform the tasks.

While the patient performed vision-oriented tasks, recordings were taken using extracranial multi-channel electroencephalography (EEG), a non-invasive technique that provides a readout of neuronal activity across the cortex. The EEG signals suggest that the act of carrying out the visual perception tests was accompanied by neurophysiological activity in the visual cortex.

In addition, the patient also reported significant improvements in his ability to conduct day-to-day activities such as navigating in outdoor and indoor environments and detecting household objects and furniture.

Watching a patient benefit for the first time from this trial using optogenetics to treat blindness has been a uniquely rewarding experience, commented Dr. Jos-Alain Sahel, MD, PhD, lead and co-corresponding author, Co-Founder of GenSight, and Founder of the Institut de la Vision (Sorbonne-Universit/Inserm/CNRS), Paris, France. Dr. Sahel is also Director of Institut Hospitalo-Universitaire FOReSIGHT, Paris, France, and Distinguished Professor and Chairman of the Department of Ophthalmology at the University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center), USA. He added, Being able to take part in bringing this new scientific approach to the clinic reflects the long-term collaboration with Botond Roska, the scientists of the Vision Institute, our clinicians, the Streetlab and psychophysics teams, and GenSight.

A video of the patient performing the tests, which was submitted as supplementary material to Nature Medicine, can be viewed at http://www.gensight-biologics.com.

Key Opinion Leader Webcast: June 4, 2021 at 2:00 PM CEST/8:00 AM EDT

Dr. Sahel and Dr. Roska will discuss the case report on a KOL webcast dedicated to Optogenetics and GS030 and hosted by GenSight Biologics.

Details will be announced at a later date.

Context

RP is the leading cause of inherited blindness and is caused by mutations in more than 71 different genes.a By using gene therapy to induce light sensitivity in unaffected retinal ganglion cells, GS030 overcomes the challenge among genetics-based treatments of exclusively addressing a specific underlying mutation and thus offers a treatment that is independent of the underlying pathogenic mutation.

PIONEER is the Phase I/II first-in-human, multi-center, open-label dose-escalation clinical trial to evaluate the safety and tolerability of GS030 in subjects with late-stage RP. A total of 12 to 18 subjects are planned to be enrolled. Three cohorts with three subjects each will be administered an increasing dose of GS030-DP via a single intravitreal injection in their worse-seeing eye. An extension cohort will receive the highest tolerated dose. A Data Safety Monitoring Board (DSMB) reviews the safety data of all treated subjects in each cohort and makes recommendations before the next cohort is enrolled. The primary outcome analysis will be the safety and tolerability at one year post-injection.

In line with the PIONEER protocol, the subject received the lowest dose (5.0E10 vector genomes) of GS030-DP in his worse-seeing eye. Four and a half months after injection, the patient began systematic training at Streetlab, a specialized visual rehabilitation facility, to learn how to use the light-stimulating goggles. The timing of the training was based on the estimated time it takes for the expression of light-sensitive opsin to stabilize in foveal ganglion cells.

Highlights of Visual Function Findings from Case Report

In the first visual test, the subject was asked to perceive, locate, and touch a single object placed in front of him on a white table. The subject had no success without the goggles. When the subjects treated eye was stimulated by the GS030-MD goggles, his ability to perceive, locate, and touch an object depended on the size of the object, with a significantly higher rate of successful trials with a large object (a notebook; 92%) than with the smaller object (a staple box; 36%). The success rate was similar for objects at different contrasts, suggesting that even objects at lower contrasts generated enough retinal activity for perception. Finally, the success rate was similar for the different tasks of perceiving, locating, and touching, suggesting that once the object was perceived, the patient could coordinate his motor system with the percept.

The second visual test required the subject to perceive, count, and locate two or three tumblers of different contrasts placed in front of him on a white table. As in the first test, the subject had no success without the goggles. When the subjects treated eye was stimulated by the GS030-MD goggles, the patient perceived, correctly counted, and located the objects in the majority (58-63%) of the trials. As in the first test, the success rate was similar for objects of different contrasts.

In the third visual test, the patient had to assess the presence or absence of a tumbler on a white table. The success rate with the goggles stimulating the treated eyes was statistically significantly higher than without the goggles (41% vs. 6%; p < 0.001).

Highlights of Safety Findings from Case Report

In-depth ocular examinations were performed regularly before and after injection, and potential intraocular inflammation was monitored according to international guidelines of the Standardization of Uveitis Nomenclature (SUN) Working Group.b Both eyes of the subject showed no intraocular inflammation and no changes in the anatomy of the retina; there were no ocular or systemic adverse events over the 84 weeks of assessment.

The subject tested the light-stimulating goggles three times before being injected with the gene therapy. On each of these occasions, he reported no change of vision or photophobia.

Detailed findings can be found at https://www.nature.com/articles/s41591-021-01351-4.

*About the paper:

Partial recovery of visual function in a blind patient after optogenetic therapy

Authors:

Jos-Alain Sahel1,2,3,4, Elise Boulanger-Scemama3,4, Chlo Pagot5, Angelo Arleo1, Francesco Galluppi6, Joseph N Martel2, Simona Degli Esposti7, Alexandre Delaux1, Jean-Baptiste de Saint Aubert1, Caroline de Montleau5, Emmanuel Gutman5, Isabelle Audo1,3, Jens Duebel1, Serge Picaud1, Deniz Dalkara1, Laure Blouin6, Magali Taiel6, Botond Roska8,9

Affiliations:

1 Sorbonne Universit, INSERM, CNRS, Institut de la Vision, Paris, France

2 Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, USA

3 INSERM-Centre d'Investigation Clinique 1423, Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Paris, France

4 Dpartement d'Ophtalmologie, Fondation Ophtalmologique Rothschild, Paris, France

5 Streetlab, Institut de la Vision, Paris, France

6 GenSight Biologics, Paris, France

7 NIHR Moorfields Biomedical Research Centre at Moorfields Eye Hospital and UCL Institute of Ophthalmology, London, United Kingdom

8 Institute of Molecular and Clinical Ophthalmology Basel, Basel, Switzerland

9 Department of Ophthalmology, University of Basel, Basel, Switzerland

References:

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About GS030

GS030 leverages GenSight Biololgics optogenetics technology platform, a novel approach to restore vision in blind patients using a combination of ocular gene therapy and tailored light-activation of treated retinal cells. The gene therapy, which is delivered via a single intravitreal injection, introduces a gene encoding for a light-sensitive protein (ChrimsonR-tdT) into retinal ganglion cells, making them responsive to light and bypassing photoreceptors killed off by diseases such as retinitis pigmentosa (RP). Because ChrimsonR-tdT is activated by high intensities of amber light, a wearable medical device is needed to stimulate the treated retina. The optronic lightstimulating goggles (GS030-MD) encode the visual scene in real-time and project a light beam with a specific wavelength and intensity onto the treated retina. Treatment with GS030 requires patients to wear the external wearable device in order to enable restoration of their visual function. With the support of the Institut de la Vision in Paris and the team of Dr. Botond Roska at the Friedrich Miescher Institute in Basel, GenSight is investigating GS030 as therapy to restore vision in patients suffering from late-stage RP. GenSights optogenetics approach is independent of the specific genetic mutations causing blindness and has potential applications in other diseases of the retina in which photoreceptors degenerate, like dry agerelated macular degeneration (dry-AMD). GS030 has been granted Orphan Drug Designation in the United States and Europe.

About Optogenetics

Optogenetics is a biological technique that involves the transfer of a gene encoding for a light sensitive protein to cause neuronal cells to respond to light stimulation. As a neuromodulation method, it can be used to modify or control the activities of individual neurons in living tissue and even in-vivo, with a very high spatial and temporal resolution. Optogenetics combines (1) the use of gene therapy methods to transfer a gene into target neurons with (2) the use of optics and electronics (optronics) to deliver the light to the transduced cells. Optogenetics holds clinical promise in the field of vision impairment or degenerative neurological disorders.

About Retinitis Pigmentosa

Retinitis pigmentosa (RP) is a family of orphan genetic diseases caused by multiple mutations in numerous genes involved in the visual cycle. Over 100 genetic defects have been implicated. RP patients generally begin experiencing vision loss in their young adult years, with progression to blindness by age 40. RP is the most widespread hereditary cause of blindness in developed nations, with a prevalence of about 1.5 million people throughout the world. In Europe and the United States, about 350,000 to 400,000 patients suffer from RP, and every year between 15,000 and 20,000 new patients with RP lose sight. There is currently no curative treatment for RP.

About the PIONEER Phase I/II trial

PIONEER is a first-in-man, multi-center, open label dose-escalation study to evaluate the safety and tolerability of GS030 in 12-18 subjects with late-stage retinitis pigmentosa. GS030 combines a gene therapy (GS030-DP) administered via a single intravitreal injection with a wearable optronic visual stimulation device (GS030-MD). Eligible patients in the first three cohorts are those affected by end-stage non-syndromic RP with no light perception (NLP) or light perception (LP) levels of visual acuity. The extension cohort will include patients with hand motion (HM) and counting fingers (CF) levels of visual acuity.

As per protocol, three cohorts with three subjects each will be administered an increasing dose of GS030-DP via a single intravitreal injection in their worse-seeing eye. An extension cohort will receive the highest tolerated dose. The DSMB will review the safety data of all treated subjects in each cohort and will make recommendations before a new cohort receives the next dose. The primary outcome analyses will be on the safety and tolerability at one year post-injection. PIONEER is being conducted in three centers in the United Kingdom, France and the United States.

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GenSight Biologics Announces Nature Medicine Case Report Showing Visual Recovery after GS030 Optogenetic Treatment - Business Wire

MIAMI--(BUSINESS WIRE)--Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR), a clinical-stage biopharmaceutical company dedicated to the development of regenerative therapies, today announced that the Company will be presenting at the annual meeting of the International Society of Cell and Gene Therapy (ISCT) taking place on May 26-28, 2021.

Organicells work, which was selected for an oral presentation, will be part of a session titled: "EVs for Infectious Diseases and Preparedness for Future Pandemics - ISCT-ISEV Joint Session and is scheduled to be broadcast live to program registrants on May 27th.

This presentation comes as a follow up to recently published case report studies demonstrating the investigation of Zofin in three severely ill COVID-19 patients. In these studies, results found the administration of Zofin to be associated with decreased levels of inflammatory biomarkers, such as CRP and IL6.

COVID-19 infection complications are, in part, the result of an excessive immune response with the over-production of pro-inflammatory cytokines such as IL6 and CRP. Therefore, the observation of reduced concentration of these biomarkers may indicate a positive trend towards recovery.

ISCT is the global steward fostering cell and gene therapy translation to the clinic. With a network of leading clinicians, regulators, researchers, technologists and industry partners, ISCT members have a shared vision to translate cell and gene therapies into safe and effective therapies to improve patients lives worldwide. For more information about the organization, please visit: isctglobal.org.

We are excited that our work was selected amongst other researchers investigating the therapeutic potential of extracellular vesicles for infectious disease of future pandemics, said Mari Mitrani, M.D., Ph.D., Chief Science Officer of Organicell.

About Zofin:

Zofin is an acellular biologic therapeutic derived from perinatal sources and is manufactured to retain naturally occurring microRNAs, without the addition or combination of any other substance or diluent. This product contains over 300 growth factors, cytokines, and chemokines as well as other extracellular vesicles/nanoparticles derived from perinatal tissues. Zofin is currently being tested in a phase I/II randomized, double blinded, placebo trial to evaluate the safety and potential efficacy of intravenous infusion of Zofin for the treatment of moderate to SARS related to COVID-19 infection vs placebo.

ABOUT ORGANICELL REGENERATIVE MEDICINE, INC.

Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR) is a clinical-stage biopharmaceutical company that harnesses the power of exosomes to develop innovative biological therapeutics for the treatment of degenerative diseases. The Companys proprietary products are derived from perinatal sources and manufactured to retain the naturally occurring exosomes, hyaluronic acid, and proteins without the addition or combination of any other substance or diluent. Based in South Florida, the company was founded in 2008 by Albert Mitrani, Chief Executive Officer and Dr. Mari Mitrani, Chief Scientific Officer. To learn more, please visit https://organicell.com/.

FORWARD-LOOKING STATEMENTS

Certain of the statements contained in this press release should be considered forward-looking statements within the meaning of the Securities Act of 1933, as amended (the Securities Act), the Securities Exchange Act of 1934, as amended (the Exchange Act), and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are often identified by the use of forward-looking terminology such as will, believes, expects, potential or similar expressions, involving known and unknown risks and uncertainties. Although the Company believes that the expectations reflected in these forward-looking statements are reasonable, they do involve assumptions, risks and uncertainties, and these expectations may prove to be incorrect. We remind you that actual results could vary dramatically as a result of known and unknown risks and uncertainties, including but not limited to: potential issues related to our financial condition, competition, the ability to retain key personnel, product safety, efficacy and acceptance, the commercial success of any new products or technologies, success of clinical programs, ability to retain key customers, our inability to expand sales and distribution channels, legislation or regulations affecting our operations including product pricing, reimbursement or access, the ability to protect our patents and other intellectual property both domestically and internationally and other known and unknown risks and uncertainties, including the risk factors discussed in the Companys periodic reports that are filed with the SEC and available on the SECs website (http://www.sec.gov). You are cautioned not to place undue reliance on these forward-looking statements All forward-looking statements attributable to the Company or persons acting on its behalf are expressly qualified in their entirety by these risk factors. Specific information included in this press release may change over time and may or may not be accurate after the date of the release. Organicell has no intention and specifically disclaims any duty to update the information in this press release.

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Organicell To Present Results Of Zofin Clinical Studies At The International Society Of Cell And Gene Therapy Annual Meeting - Business Wire

"Cancer is a disease of genetics, yet clinical practice has struggled to keep pace with rapid advancements in research, particularly with respect to the role of germline genetics. Testing guidelines and medical policy often codify barriers, further lengthening the path to adoption of widespread testing and in some cases restricting access to precision therapies and clinical treatment trials," said Ed Esplin, M.D., Ph.D., FACMG, FACP, clinical geneticist at Invitae. "Research presented at ASCO shows that cancer-linked genetic changes are common across cancer types and when patients do receive germline testing, over two thirds of those with positive results are eligible for changes to their treatment plans. It's clear that incorporating germline testing alongside tumor profiling can help oncologists better tailor treatment for each patient."

Data from 250 pancreatic cancer patients from the landmark INTERCEPT study conducted at the Mayo Clinic found that nearly one in six patients with pancreatic cancer (n=38) showed cancer-linked genetic changes and, importantly, receiving germline testing was associated with improved survival.

A separate study of prostate cancer patients confirmed similar findings in other cancer types that limiting testing deprives patients and clinicians of actionable information. In the first-ever presentation of the PROCLAIM study, which was conducted primarily in community urology clinics, of patients diagnosed with prostate cancer, a significant number of cancer-linked variants were missed if testing was done based on NCCN guidelines. Of the 532 patients with clinician-reported data, nearly half, 45% (n=239), did not meet NCCN criteria. Overall, 59 patients had a cancer-linked variant; one in 10 of them did not meet the criteria (9.6%, n=23), and 12.3% (n=36) of patients met the criteria. When a 12-gene panel was used, only 29 patients were found to have a cancer-linked variant and one third of these patients were missed by guidelines.

A third study showed simply changing medical policy is not enough to drive changes in clinician adoption. In a review of two independent datasets, including commercially insured and Medicare Advantage enrollees, only 3% (n=1,675) of the 55,595 colorectal cancer patients received germline genetic testing, despite medical policy recommending germline genetic testing for all colorectal cancer patients (consistent with the INTERCEPT colorectal cancer study). Of the patients who received testing, 18% (n=143) had a cancer-linked variant and two thirds, or 67% (n=96), of those patients were potentially eligible for precision therapy and/or clinical trials.

"The data have been available for years that show knowing what changes patients have in their genes is beneficial to treating their cancer. Yet the oncology community has been slower to adopt germline testing than tumor profiling, for reasons that are not entirely clear. These data presented at ASCO highlight the need for oncologists to embrace germline genetic testing as routine practice for all cancer patients," said Robert Nussbaum, M.D., chief medical officer at Invitae. "A positive germline genetic result may enable patients to enroll in clinical trials or gain access to new precision medicines. And equally important, the discovery of an inherited variant can alert relatives to seek out earlier cancer screening, helping avoid later-stage diagnoses and offering a treatment benefit if cancer develops."

Invitae aims to help overcome obstacles to the adoption of genetic testing by providing physicians with clinical consults to help interpret results and reducing cost as a barrier to genetic information. Invitae also provides patients direct access to genetic counselors, helping to integrate routine genetic testing into patient care with GIA, a HIPAA-compliant chatbot. Family members are also able to receive no-charge genetic testing if a positive result is found.

Details of the 2021 ASCO presentations:

Oral Abstract Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Discussion Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Gastrointestinal Cancer--GastroesophageaI, Pancreatic, and Hepatobiliary

About InvitaeInvitae Corporation(NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Safe Harbor StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the benefits of germline testing and genetic information; and that the data presented at ASCO highlight the need for increased germline testing in all cancer patients regardless of medical policy. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's filings with the Securities and Exchange Commission, including the risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Contact:Laura D'Angelo[emailprotected](628) 213-3283

SOURCE Invitae Corporation

http://www.invitae.com

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Germline genetic testing can benefit all cancer patients as a routine practice in cancer care - PRNewswire

Introduction

Laryngeal cancer is a relatively common malignant tumor in the head and neck, accounting for 15% of malignant tumors in the body. Squamous cell carcinomas account for 9698% of laryngeal cancers. Incidences of laryngeal cancer are increasing yearly as the smoking population expands, and the environment deteriorates. In 2015, there were 26,400 new cases of laryngeal cancer in China. Among these, 14,500 people died. The male to female ratio was 9:1.1 The incidence of laryngeal cancer is high in middle-aged and older males. In recent years, more incidences of laryngeal cancer are being found in the younger population, with significant differences among different races and regions.2 In China, the incidence of laryngeal cancer is highest in the Northeast and the North. In the United States, over the past 40 years, the percentage of patients with laryngeal cancer who survive for five years or more has decreased from 66% to 63%,3 suggesting the need for innovation in detection and treatment. Mitochondria are widely involved in cellular activities, including the regulation of cell cycle, oxidative stress, and apoptosis.4 Mitochondrial DNA (mtDNA) injury is closely correlated with tumors, aging, and neurodegenerative diseases.5,6 The D-loop region is the non-coding region in mtDNA. Most of the regulatory sequences related to the mtDNA replication, transcription, and translation are found in this region, which is prone to mutation. Although there have been achievements in the field concerning the relationship between tumorigenesis and mitochondrial mutation, and some related research at home and abroad have clarified some related mechanisms, the research on the relationship between tumorigenesis and mtDNA remains unclear. Moreover, no direct relationship between the abnormal changes of mtDNA and tumorigenesis has been found so far. Although many studies have reported the relationship between mitochondrial gene mutations and various tumors, the study on the mechanism of laryngeal cancer has not been reported. The purpose of the present study was to investigate the relationship between the D-loop gene mutation in mtDNA and the clinical and pathological parameters of laryngeal cancer by gene sequencing.

From June 2013 to June 2019, 60 patients with squamous cell laryngeal carcinoma were selected from the First Affiliated Hospital of Henan University of Science and Technology. No patients received radiotherapy or chemotherapy before the operation. The age ranged between 4080, with an average age of 59.12 10.35. There were 55 males and five females. There were 15 cases in stage I, 26 in stage II, ten in stage III, and nine in stage IV (according to the 2017 UICC staging criteria). The specimens from all cases were diagnosed and screened by the pathological experts of the First Affiliated Hospital of Henan University of Science and Technology. This study was approved by the medical ethics committee of the Henan University of Science and Technology. Written informed consent was obtained from all participants. This study complied with the Declaration of Helsinki.

DNA Marker DL2000 (CB15727772 TAKARA, Japan); 10Xbuffer (Mg2+plus) (DR001AM TAKARA, Japan); dNTPs (DR001AM TAKARA, Japan); Taq DNA polymerase (DR001AM TAKARA, Japan); agarose (D1200 Beijing Solabo Technology Co., Ltd); Primers (JN0060-02 Shanghai Bioengineering Technology Co., Ltd).

Thermo high-speed centrifuge (USA); PCR instrument (ABI, USA); Gel imaging system (USA); 80C refrigerator (Japan).

The specimens resected during laryngeal cancer surgery were removed, and the central part of the specimen with cancer focus was taken as the cancer tissue. Approximately 20 mg of the cancer tissue was cut and placed in a centrifuge tube. The tissue was broken into cell suspension by a high-speed tissue homogenizer. The following procedures were operated according to the kit instructions. After a series of extraction and washing, the final purified DNA solution was placed in a 20C refrigerator for storage and further detection.

The sequence in the D-loop region in mtDNA was the target sequence with a full length of 1122 bp. The primers and probes were synthesized by the Shanghai Bioengineering Technology Service Co., Ltd. The sequences of primers were as follows: the upper primer 5-CCATTAGCACCCAAAGCTAAG-3, the lower primer 5-TGCTTTGAGGTAAGCTACA-3. The reaction system was 50 L, with 5 L of the 10 buffer (Mg2+ plus) solution, 4 L of the mixture of dNTP, 0.25 L of the DNA polymerase, and 1 L of the upper primer and 1 L of the lower primer, 1 L of the template DNA, and 37.75 L of distilled water. The reaction conditions were as follows: 94C 30s, 55C 30s, 72C 90s, with a total of 30 cycles. The electrophoresis of the PCR products was under the condition of 120V for 30 minutes. After the electrophoresis, the gel imaging system was used to take photos to confirm that the amplified fragment was the desired target fragment.

The purification of the PCR products and the determination of gene sequences were completed by the Beijing Sequencing Department of Shanghai Yingjun Biotechnology Co., Ltd. The quality of the peak map was evaluated by Chromas 2.31 software, and the samples with low quality were re-sequenced. After landing on the website (https://www.Ncbi.Nlm.Nih.gov), the Align two (or more) sequences using BLAST software was used to compare the gene sequence of laryngeal cancer tissue with the Cambridge standard sequence. Once the mutation site is identified, the Chromas software should be adopted to observe the sequencing peak and compare it with the databases reported mutation site. If there is no report, it is considered a novel mutation. At the same time, these mutation sites were compared with the sub-database of the polymorphic sites of the mtDB database. If the mutation frequency was more than 1% in all populations tested in the database, it was regarded as the single nucleotide polymorphism (SNP). The gene sequencing data was provided in the Supplementary Document.

The Prism GraphPad 5.0 software was used for statistical analysis of the results. The Pearsons or the Spearman correlation test was adopted to test the correlation between the two variables, and 2 test was used to compare the number of mutations between genders. P < 0.05 was considered statistically significant.

The length of the target gene was 1122bp after the PCR amplification of the full length of the D-loop region (Figure 1). A single bright band was demonstrated by the gel imaging instrument, and the molecular weight was determined by the standard of DL2000DNAMark.

Figure 1 Results of the mtDNA PCR product.

Among the 60 samples, 38 mutation sites had been detected in 51 samples (85%), with a total of 174 mutations. In three HV in the D-loop, 160 mutations had been detected, accounting for 91.9% of the total mutations (Table 1). Microsatellite instability existed in the D310 regions in 15 cases (25%) (Figure 2), mainly manifested as the increased insertion of poly-C. Among them, nine cases showed insertion of one base C, six cases showed insertion of two bases, and the base pair exchanges within a microsatellite were found in two cases (Figure 3).

Table 1 Sequencing Results and Analysis

Figure 2 Microsatellite instability in D310 region (insertion of base pair C).

Figure 3 Microsatellite instability in D310 region (T, C exchange).

As shown in Table 2, there was no correlation between the age, gender, tumor diameter, TNM stage, and the number of the D-loop mutations in mtDNA in the 51 specimens with mutations (P > 0.05).

Table 2 The Correlation Between the D-Loop Region Mutations in mtDNA and the Clinical and Pathological Parameters in Laryngeal Cancer (Number of Cases)

Mitochondria are the only organelles with DNA outside the nucleus in the eukaryotes, which are the main sites for oxidative phosphorylation to produce active oxygen and provide necessary energy and oxygen radicals for cell activities. When mtDNA mutation occurs, the cellular energy supply is dysfunctional, and a large number of ROS are produced. This leads to changes in cell function and even cell necrosis, thus showing a variety of clinical symptoms. Mitochondrial dysfunction may play an important role in tumor development, early diagnosis, drug resistance, prevention of recurrence, and prognosis.79 MtDNA is a closed-loop double-stranded DNA molecule composed of 16,569 base pairs. As the main non-coding region, the D-loop region of npl6024-np576 is responsible for regulating mtDNA replication and transcription.10 Sanchez-Cespedes et al11 found that 41% of head and neck squamous cell carcinoma had mutations in the D-loop region, including the deletion, insertion, and point mutation. Ha et al12 revealed that patients with head and neck tumors had the change of poly-C in the D-loop region of mtDNA. Our experiment confirmed this. In recent years, the D-loop region is agreed to be the HV of mtDNA mutations. In the present study, 91.9% of the mutation sites were concentrated in the HV region, consistent with the above view. Microsatellites are the short tandem repeats in the human genome. Microsatellite instability refers to the changes of microsatellite in length and the emergence of new microsatellite alleles in tumors due to the insertion or deletion of repeat units compared with normal tissues. Habano et al13 first proposed the mitochondrial microsatellite instability in a study on rectal cancer. In the present study, microsatellite instabilities were found in 15 cases of laryngeal cancer, mainly concentrated in the D310 region and manifested as the increase of poly-C insertion. Among these cases, nine showed insertion of one base C, six showed insertion of two bases, and base exchanges within a microsatellite were found in two cases. This was consistent with the study by Sanchez-Cespedes et al that 41% of head and neck squamous cell carcinoma had mutations in the D-loop region area.11 The HV of the D310 region may be related to its location in the intron, coder, and promoter of the gene and the repeated repair caused by slip error during replication.14 Therefore, the analysis of mtDNA mutations, especially the detection of changes in the D310 region, may play an important role in the cytological diagnosis, especially for cases with no obvious morphological changes or rare tumor cells. In conclusion, PCR amplification and direct sequencing were used in the present study to detect the D-loop gene changes in mtDNA in samples of laryngeal cancer tissue. It was found that there were a large number of mutations and microsatellite instability in the D-loop region in mtDNA of patients with laryngeal cancer, indicating that the D-loop gene mutations in mtDNA may play an important role in the development of laryngeal cancer.

We would like to acknowledge the hard and dedicated work of Dr. Kai Xi and Dr. Hao Chang that helped and contributed to us in terms of technology and software of the study.

The authors report no conflicts of interest in this work.

1. Chen W, Zheng R, Baade PD, et al. Cancer statistics in China, 2015. CA Cancer J Clin. 2016;66(2):115132. doi:10.3322/caac.21338

2. Shin JY, Truong MT. Racial disparities in laryngeal cancer treatment and outcome: a population-based analysis of 24,069 patients. Laryngoscope. 2015;125(7):16671674. doi:10.1002/lary.25212

3. Siegel RL, Miller KD, Jemal A. Cancer statistics, 2016. CA Cancer J Clin. 2016;66(1):730. doi:10.3322/caac.21332

4. Correia-Melo C, Passos JF. Mitochondria: are they causal players in cellular senescence? Biochim Biophys Acta. 2015;1847(11):13731379. doi:10.1016/j.bbabio.2015.05.017

5. Bose A, Beal MF. Mitochondrial dysfunction in Parkinsons disease. J Neurochem. 2016;139(Suppl 1):216231. doi:10.1111/jnc.13731

6. Lee J, Chang JY, Kang YE, et al. Mitochondrial energy metabolism and thyroid cancers. Endocrinol Metab. 2015;30(2):117123. doi:10.3803/EnM.2015.30.2.117

7. Chen N, Wen S, Sun X, et al. Elevated mitochondrial DNA copy number in peripheral blood and tissue predict the opposite outcome of cancer: a meta-analysis. Sci Rep. 2016;18(6):37404. doi:10.1038/srep37404

8. Huang X, Zhou X, Hu Q, et al. Advances in esophageal cancer: a new perspective on pathogenesis associated with long non-coding RNAs. Cancer Lett. 2018;28(413):94101. doi:10.1016/j.canlet.2017.10.046

9. St John JC. Mitochondrial DNA copy number and replication in reprogramming and differentiation. Semin Cell Dev Biol. 2016;52:93101. doi:10.1016/j.semcdb.2016.01.028

10. Morandi L, Asioli S, Cavazza A, Pession A, Damiani S. Genetic relationship among atypical adenomatous hyperplasia, bronchioloalveolar carcinoma and adenocarcinoma of the lung. Lung Cancer. 2007;56(1):3542. doi:10.1016/j.lungcan.2006.11.022

11. Sanchez-Cespedes M, Parrella P, Nomoto S, et al. Identification of a mononucleotide repeat as a major target for mitochondrial DNA alterations in human tumors. Cancer Res. 2001;61(19):70157019.

12. Ha PK, Tong BC, Westra WH, et al. Mitochondrial C-tract alteration in premalignant lesions of the head and neck: a marker for progression and clonal proliferation. Clin Cancer Res. 2002;8(7):22602265.

13. Habano W, Sugai T, Nakamura SI, Uesugi N, Yoshida T, Sasou S. Microsatellite instability and mutation of mitochondrial and nuclear DNA in gastric carcinoma. Gastroenterology. 2000;118(5):835841. doi:10.1016/S0016-5085(00)70169-7

14. Lin CS, Lee HT, Lee MH, et al. Role of mitochondrial DNA copy number alteration in human renal cell carcinoma. Int J Mol Sci. 2016;17(6):814. doi:10.3390/ijms17060814

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Clinical Significance of the D-Loop Gene Mutation in Mitochondrial DNA | OTT - Dove Medical Press

Welcome to this week's Chutes & Ladders, our roundup of hirings, firings and retirings throughout the industry. Please send the good wordor the badfrom your shop to Fraiser Kansteiner, and we will feature it here at the end of each week.

TevardBiosciencesGopi Shanker, Ph.D., lands at the startup as chief scientific officer.

Shanker's departure marks something of a trend for Novartis. In late April,Jeff Engelman, M.D., Ph.D.,left the Novartis Institutes for Biomedical Research (NIBR) to stand up the new oncology biotechTreelineBiosciences. Just a few days later, fellow Novartis cancer R&D execPeter Hammerman, M.D., Ph.D., leftto start as chief scientific officer of molecular machines biotech MOMA Therapeutics.Shanker, for his part, will take up the post ofchief scientific officer at gene therapy startup TevardBiosciences. After heading upneuroscience atNIBR, Shanker will nowlead the preclinical scientific programs at the transfer RNA-based gene therapystartup.A few months back, Tevard forged a pact withZogenix to seek out and work on gene therapies for Dravet syndrome, a rare form of epilepsy, and other genetic epilepsies.Fierce Biotech

Blueprint MedicinesPercy Carter, Ph.D., takes up the helm of chief scientific officer.

Carter is in good company at Blueprint, where he joins former Bristol Myers Squibb compatriotFouad Namouni, M.D., who became president of R&D last fall. Carter, who joinedBristol Myers Squibb in 2001and eventually rose through the ranks tosenior vice president and head of discovery, willspearhead research and preclinical development as the new chief scientific officer at Blueprint. More recently, Carter held posts atJohnson & Johnson, where he was global head of discovery sciences in the healthcare giant's Janssen unit, andFibroGen, where he was in the CSO seat for less than a year. He joins Blueprint a little over a year after the company's first approval. In January 2020,Ayvakit,kinase inhibitor, became the first targeted treatmentfor patients with a type of stomach cancer called gastrointestinal stromal tumor who carry a (PDGFR) exon 18 mutation.Fierce Biotech

IovanceBiotherapeuticsMariaFardis, Ph.D.,is hitting the exit.

Fardis, who's also Iovance's president and a director, alertedthe company on Tuesday that she wasresigning topursue other opportunities," Iovance quietly revealed in a Wednesday securities filing. Her exit coincides withadditional data requests on potency assays for Iovance'slead candidate lifileucelfrom the FDA.Iovancewill continue validating the potency assays and expects to submit further data and meet with the FDA in the second half of 2021.The setback pushesIovancesbiologics license application submission into the first half of 2022,the company said. The "optics" of Fardis departure are "challenging" and raise "questions about whether something else is behind the change or whether the Board chose to make the change, Jefferies analysts wrote to clients this week. Long term,we think the drug works [] and it will eventually get to market," the Jefferies team added.IovancesBoard will appoint general counsel Frederick Vogt, Ph.D., as interim president and CEO. The company will immediately kick off the huntfor a successor.Fierce Biotech

>Passage Bio has bidadieu to chief medical officer Gary Romano, M.D., Ph.D. The gene therapy startup and Romanomutually agreed they should part waysTuesday, and Romano hit the exit that same day.Chief Financial Officer Richard Morris is also leaving the company on May 30, but Passage Bio described the situation differently: It said Morris' departure was not the result of any disagreement regarding any matter relating to the Companys operations, financial statements, internal controls, auditors, policies, or practices. Passage didn't say whether Romano's departure was the resultof a disagreement.Fierce Biotech

>Merck KGaA has slottedChris Round into the role ofpresident at its North America healthcare unit, replacing Andrew Patterson, who'll step into the newly minted role of chief marketing officer.EMD Serono is hoping to cash in with three promising drugsits Pfizer-partnered immuno-oncology medBavencio, multiple sclerosis treatment Mavenclad and Tepmetko for non-small cell lung cancer.Round will be based in Rockland, Massachusetts. He served for 20 years at Merck & Co. before arriving at Merck KGaA in 2017 to spearheadcommercial operations in Asia, the Middle East and Africa.Fierce Pharma

>Clinical Ink, a global clinical trial technology firm, has summoned Janette Morgan as EVP, general manager, Europe, the Middle East and Africa. Morgan joins after a 13-year run at Medidata, where she most recently served as vice president, global partner initiatives & partner business management. In that role, she was in charge ofglobal leadership and management of Medidatas Partner Study-by-Study business.Release

>Xenter, hot on the heels of last week's $12 million series A, has enlistedScott Heuler as senior vice president of global sales and marketing, whileGary Baldwin signs onas chief technology officer in Xenter'shospital technologies business unit.Baldwin previously led R&D, engineering and product teams for multiple organizations. Heuler, for his part, has held seniorsales and marketing leadership roles with NeuroOne Medical Technologies, Guidant Corporation and U.S. Surgical Corporation, Xenter said.Release

> Clinical-stage immuno-oncology companyMedicenna has tapped Mann Muhsin, M.D., as chief medical officer. With more than 20 years experience in medical practice and drug development, Muhsin signs on with "an outstanding track record of innovation in oncology and immuno-oncology trial design," Medicenna said in a release. Muhsin kicked off his clinical research career atPICR phase I unit, where he conducted more than 17 clinical trials for international sponsors likeAstraZeneca,Hoffmann La Roche, Merck, Novartis, Eli Lilly, Johnson & Johnsonand Bayer. He then went on to lead early clinical development programs at Johnson & Johnson's Janssen unit.Release

>CDMO and clinical supply services company Experic is putting David Wood in charge as chairman and chief executive officer. Over a 30-year run in the industry, Wood has lead organizations and teams atCaptek Softgel International, Catalent Pharma Solutions, Cardinal Health PTS and Becton Dickinson. He's taking the reins from Jeffrey McMullen, one of Experic's founders and its former chairman and CEO. McMullen took on aninterim CEOrole in 2019 after the untimely passing of the executive previously managing the company.Release

>NeuBase Therapeutics, looking to push its first drug candidate into the clinic next year, will welcomeSandra Rojas-Caro, M.D., as chief medical officer onMay 24. At NeuBase, she'll be in charge of thepreclinical and clinical development, medical, and regulatory strategy of NeuBases pipeline. She most recently worked at Gemini Therapeutics, where she held the same post. Before that, she was CMO atAeglea BioTherapeutics.Release

>Rain Therapeutics, working on precision oncology therapeutics, has lifted co-founderRobert Doebele, M.D., Ph.D., into the role ofpresident and chief scientific officer. Doebele co-founded Rain with chairman and CEOAvanish Vellanki in 2017. Before he joined the company full-time in October 2020, Doebele served asassociate professor of medicine in the division of medical oncology at the University of Colorado School of Medicine, director of the Thoracic Oncology Research Initiative at the University of Colorado Cancer Center and as principal investigator for the University of Colorado Lung Cancer Specialized Program of Research Excellence.Release

> Precision oncology firm Fore Biotherapeutics is building out its leadership team with the addition ofStacie Peacock Shepherd, M.D., Ph.D., as chief medical officer and Nora Brennan as chief financial officer. Shepherd will oversee clinical strategy and operational advancement of Fore's lead precision oncology program, FORE8394, and she will also helpexpand the company's clinical pipeline in the coming months. Brennan, for her part, previously served as CFO atTELA Bio, and held the same role at Xeris Pharmaceuticals before that.Release

>Cybrexa Therapeutics has summonedStephen Basso as its chief financial officer. Before joining the Cybrexa team, Basso served as senior vice president of finance atInozyme Pharma. He also served as vice president, North America commercial finance, global G&A at Alexion Pharmaceuticals, and as director, financial planning and analysis at Pfizer.Release

>Celularity, developing off-the-shelf cell therapiesderived from the postpartum human placenta, has signed onBradley Glover, Ph.D., as EVP and chief technology officer. At Celularity, Glover will oversee all aspects of the company's technical operations, includingprocess development, quality, manufacturing, supply chain, IT, facilities and engineering, and more. He joins the team from Kite Pharma, where he wore a number of executive hats in corporate development and technical operations.He rose through the ranks at Genentech and Roche before that.Release

> Early-stage allogeneic cell therapy company Appia Bio has enlisted Qi Wei, Ph.D. as senior vice president and head of technical operations. Wei is bringingsome serious cell and gene therapy know-how to the fold. He's had stints atNovartis, Gilead's Kite, Eli Lilly, TCR2 Therapeutics and Geneception.He had a hand in the development of several CAR-Ts, including Novartis' Kymriah and Kite and Gilead's Tecartus.Release

>Aerovate Therapeutics, working on meds for patients with rare cardiopulmonary disease, has handed the CEO torch toTimothy Noyes. Noyes was also elected to the company's board of directors in April. He previously served as president and chief executive ofProteon Therapeutics. He previously served aschief operating officer of Trine Pharmaceuticals and held the position of president of Genzymes renal division following its acquisition of GelTex Pharmaceuticals. Noyes started his industry run at Merck & Co.Release

> Biopharma product and portfolio strategy planner Prescient has appointed John Crowley as director of the company's intelligence and insight business, whileSugandh Sharma enters the role of senior director in the same business unit. Before joining the fold at Prescient, Crowleyled analyst teams at Decision Resources Group, covering rare diseases in neurology, immunology and hematology, as well as infectious diseases.Release

>Cognito Therapeutics, developing a new class ofdisease-modifying digital therapeutics to treat neurodegenerative disorders likeAlzheimers disease,tapped Everett Crosland as its chief commercial officer. He recently held the same role at AppliedVR, where he was in charge ofnational launch sales, marketingand payer strategy on the first breakthrough designated virtual reality therapeutic. Prior to that, he was VP of market access & reimbursement atPear Therapeutics.Release

>Umoja Biopharma, developing an integrated, in vivo immunotherapy platform, has signed onIrena Melnikova, Ph.D., as chief financial officer. She comes over from SVB Leerink, where she was a managing director in investment banking. Before that, shewas a managing director at Burrill & Company and, prior to that, director of strategy and external innovation at Sanofi.Release

>ONI has enlistedTyler Ralston, Ph.D., as its chief technology officer. In his role, Ralston willprovide leadership, strategic vision and oversee the research and development of ONI's technology. He signs on from the 4Catalyzer incubator, where he most recently served as CTO for Tesseract. Release

> Quanterix, working ondigital protein biomarker technology to enableprecision health, tappedMasoud Toloueas president of Quanterix and Diagnostics. He'll enter the role on June 9. He joins the team fromPerkinElmer, where he most recently served as senior vice president, diagnostics. He alsofounded and led Bioo Scientifics next generation sequencing business, which was acquired by PerkinElmer in 2016.Release

> Gene therapy firm AavantiBio has named Jessie Hanrahan, Ph.D., as Chief Regulatory Officer. She's the fifth senior executive to joins AavantiBio's leadership team in recent months and will be in charge ofglobal regulatory affairs for AavantiBios diversified pipeline of gene therapy programs targeting rare diseases with significant unmet medical need.Most recently, she was vice president of regulatory science at bluebird bio.Release

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Chutes & LaddersAnother Novartis exec answers the biotech call, this time for gene therapy startup Tevard - FierceBiotech