Monthly Archives: June 2022

BOSTON--(BUSINESS WIRE)--Carbon Biosciences (Carbon), a Longwood Fund founded biotech company and emerging leader in the development of novel parvovirus-derived gene therapies, today announced a $38 million Series A financing led by Agent Capital. Agent is joined by Longwood Fund, Astellas Venture Management LLC, the Cystic Fibrosis Foundation, Solasta Ventures, University of Tokyo Innovation Platform (UTokyoIPC), and Camford Capital. Carbon is harnessing novel parvovirus vectors that can deliver larger gene therapy payloads with enhanced tissue specificity and with minimal neutralizing immunity. The company plans to use the Series A funding to advance the development of Carbons programs for genetic diseases, initially building on the groundbreaking research of scientific co-founders John F. Engelhardt, Ph.D., Director, Center for Gene Therapy at the University of Iowa, and Robert M. Kotin, Ph.D., Professor of Microbiology and Physiological Systems at the University of Massachusetts Chan Medical School.

Carbons platform has the potential to expand the role of gene therapy in treating some of the worlds most devastating and difficult to treat diseases, said Joel Schneider, Ph.D., President and CEO of Carbon Biosciences. Our vision is to enable a new generation of genetic medicines with differentiated vectors that have the potential to address the immunological, targeting and payload limitations inherent in current viral and non-viral delivery technologies. As the first jointly funded program launched under a collaborative agreement between the Cystic Fibrosis Foundation and Longwood Fund, we are motivated by the potential impact our research may have in significantly improving care for cystic fibrosis patients.

Identifying vectors that can effectively deliver therapeutics to target tissues, such as the lung, has been a major challenge in realizing the full potential of gene therapy, said Geeta Vemuri, Ph.D., Managing Partner and Founder of Agent Capital. We believe that Carbon's proprietary platform can address this challenge by leveraging novel vectors from the broader parvovirus family to deliver optimal payloads to specific tissues. Carbons technology will enable a diversified pipeline with potential applications across the wide range of tissues impacted in many unaddressed diseases.

Carbons novel platform addresses key challenges with AAV and non-viral based therapies. Our lead program is the first gene therapy program demonstrating tissue tropism to the lung with the capacity to deliver the full length CFTR gene and an appropriate promotor, said John F. Engelhardt, Ph.D., the Roy J. Carver Chair in Molecular Medicine, and Director, Center for Gene Therapy at the University of Iowa. Preliminary pre-clinical data as well as studies on human populations suggest wide applicability of our lead clinical candidate and the potential to re-dose patients.

Carbons scientific co-founders and scientific advisory board members are among the most experienced thought leaders in the gene therapy field. In addition to Kotin and Engelhardt, Carbons co-founders are David Steinberg, who served as founding CEO (Longwood Fund); Jianming Qiu, Ph.D., (University of Kansas); Ziying Yan, Ph.D., (University of Iowa); Sebastian Aguirre, Ph.D., (Carbon Biosciences); and Lucy Liu, Ph.D., (Longwood Fund). Carbons Scientific Advisory Board consists of John Engelhardt; Jay Chiorini Ph.D., Senior Investigator at NIH National Institute of Dental and Craniofacial Research; Beverly Davidson, Ph.D., Chief Scientific Strategy Officer at Childrens Hospital of Philadelphia; and Adrian Thrasher, Ph.D., MBBS, FRCP, MRCP, Professor at University College London (UCL), Great Ormond Street Institute of Child Health, Head of Infection and Inflammation and Director, Clinical Gene Therapy GMP Facility at UCL.

In conjunction with the financing, Joel Schneider, Ph.D, joins as President and CEO. Chen Schor, Adicet Bio President and CEO, joins as Board Chair. David Steinberg, Geeta Vemuri, Robert Kotin and Derek Yoon, President and CEO of Solasta Ventures, join the Board of Directors.

About Carbon Biosciences

Carbon Biosciences is expanding the therapeutic potential of gene therapy through its proprietary platform which leverages novel parvoviruses that have been pressure tested by nature to target specific tissues and carry a larger cargo with minimal neutralizing immunity and the potential to re-dose. Founded by Longwood Fund and gene therapy pioneers, John F. Engelhardt, Ph.D., and Robert M. Kotin, Ph.D., Carbon is expanding the gene therapy toolbox for the treatment of the worlds most devastating and difficult to treat diseases. For more information, please visit our website http://www.carbonbio.com and follow us on LinkedIn.

About Longwood Fund

Longwood Fund is a venture capital firm dedicated to creating and investing in novel healthcare companies that develop important treatments to help patients while targeting significant value for investors. The Longwood team has a long history of successfully launching and building important life science companies while providing operational leadership and strategic guidance. Collectively, the Partners at Longwood Fund have co-founded 24 companies with over 20 launched or marketed drugs and therapies, as well as over two dozen clinical stage assets, all focused on helping patients in need. Companies founded by Longwood Fund, or its principals prior to the founding of the Firm, as lead investor and CEO/CBO include Vertex, Acceleron, Momenta, Alnylam, Sirtris, Vor, TScan, Pyxis Oncology, Immunitas, Be Biopharma, ImmuneID, Tome Bio, Photys, and Carbon Biosciences. For more information, visit http://www.longwoodfund.com.

About Agent Capital

Agent Capital is an international life sciences investment firm that supports disruptive healthcare companies focusing on novel, differentiated therapeutics and treatments that address unmet patient needs. Agent Capital aligns with scientists, entrepreneurs, and other investors to develop the next generation of healthcare innovations, leverages their industry expertise and successful track record to source premier deals, accelerate value, and drive successful exits. Their first fund invested in 15 portfolio companies, the majority of which have executed collaborations with major pharmaceutical companies and successfully raised additional capital in the private or public markets. For more information, please visit Agent Capital's website at http://www.agentcapital.com.

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Carbon Biosciences Launches with $38 Million Series A Financing to Advance Novel Gene Therapy Platform and Pipeline - Business Wire

We are all mirrors of our parents. We inherit their DNA, behaviour patterns, world view, dreams and even fears. However, over the years, several studies have corroborated that biologically speaking, fathers genes are more dominant and influential in a child, compared to the mother.

According to health experts, your fathers genetic endowment determines some of the most fundamental aspects of your being. On this Fathers day, find out more about your fathers contribution to who you are.

Dr Parag Tamhankar, Senior Consultant Medical Geneticist from MedGenome Labs says that the main thing thats inherited from the father is the Y chromosome which gives a child the maleness traits.

Apart from maleness, several other factors of a fathers genes determine how we shape up. Dr Gouri Pandit, Senior Genetic Counsellor, Indus Health Plus says that several physical traits of ours are inherited from the father.

Fathers are responsible for the gender of the babies, and its one of the physical traits that are 100% contributed by males as fathers transmit the Y chromosome defining the male gender. At least 700 genetic variations are responsible for determining height, coming from both mother and fathers genes. But there is evidence to suggest that each parents height gene functions differently to some extent. Dads genes play a significant role in promoting growth, adds Pandit.

Dr Anup Rawool, Consultant - Genetic Medicine,

Interestingly paternal obesity, malnutrition and consumption of a high-fat diet has been speculated to increased breast cancer development in offspring (daughters in particular) and same has been demonstrated in a mouse model study of paternal obesity. When considering genetic disease conditions due to the loss (or abnormality) of Y chromosome has been associated with increased risk of infertility secondary to spermatogenic failure, certain cancers, Alzheimer's disease, behavioural conditions like autism and cardiovascular diseases, he explained.

Mouse study using machine learning have shown that genes from each parent impacts their childs behavioural pattern differently, with mothers having more control over some of their sons decisions and fathers over some of their daughters. It is interesting to see further how such data could be applied to study aspects of human behaviour and relationship. Certainly, daughters are usually observed to share a strong bond with father than mother.

New research will keep pouring in and add to the existing domain of knowledge. Physical traits are mostly inherited in polygenic and multifactorial way with underlying genetic component having a significant if not total impact. We are what we inherit - be it from the father or mother. This Fathers Day lets raise a toast to the legacy of Y chromosome since time immemorial.

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Happy Fathers Day: From biological sex, height to paternal obesity & cancers, here are things that your d - Economic Times

PITTSBURGH and CAMBRIDGE, Mass., May 12, 2022 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. ( NBSE) (NeuBase or the Company), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, today reported its financial results for the three-month period ended March 31, 2022, and other recent developments.

We are pleased with the progress being made across our development pipeline of therapeutic programs to treat DM1, HD, and KRAS-driven cancers. The new data weve announced to date this year have further validated the use of our PATrOL platform to design novel genetic medicines that target and rescue gene dysfunctions, with the potential for clinically impactful outcomes in both rare and common diseases, said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. We continue to execute the development strategy for our DM1 program, which includes a series of IND-enabling studies scheduled to report data throughout CY2022. Last quarter, we presented pharmacodynamic data that illustrated a single intravenous (IV) dose or multiple subcutaneous (SC) doses of our DM1 development candidate resolves the genetic defect and myotonia in skeletal muscle of the gold-standard mouse model of the disease. Building off these results, we plan on announcing at ASGCT additional pharmacokinetic (PK) data, which will illustrate the exposure levels of our development candidate when administered via systemic administration in skeletal muscles, heart, and brain, tissues that are affected in DM1. We expect these data to support further advancement of our lead candidate for DM1 and validate a differentiated whole-body solution for this disease. Considering this progress, we believe the submission of an IND application to the FDA is on track for the fourth quarter of CY2022.

Second Quarter of Fiscal Year 2022 and Recent Operating Highlights

Financial Results for the Second Fiscal Quarter Ended March 31, 2022

Financial Results for the Six-Month Period Ended March 31, 2022

About NeuBase TherapeuticsNeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines that Drug the Genome. The Companys therapies are built on a proprietary platform called PATrOL that encompasses a novel peptide-nucleic acid antisense oligonucleobase technology combined with a novel delivery shuttle that overcome many of the hurdles to selective mutation engagement, repeat dosing, and systemic delivery of genetic medicines. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular, and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions, who currently have limited to no treatment options. To learn more, visit http://www.neubasetherapeutics.com.

Use of Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the plan to provide updates on the Company's development pipeline, in particular the DM1 program, at the ASGCT 25th Annual Meeting, the potential and prospects of the Companys proprietary PATrOL platform and DM1 program, the Companys expectation that it will submit an IND application for the DM1 program to the U.S. Food and Drug Administration in the fourth quarter of CY2022, our expectations to initiate scale-up and toxicology activities for development of a systemically administered allele-selective NT-0100 program to treat HD in CY2022, the potential of our therapeutic program for HD and the potential for our PATrOL-enabled compounds to silence activating KRAS point mutations in vivo to inhibit protein production. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the risks that prior data will not be replicated in future studies; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

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NeuBase Therapeutics Reports Business Update and Financial Results for the Second Quarter of Fiscal Year 2022 - GuruFocus.com

$25 Million Gift to Establish the Armellino Center of Excellence for Williams Syndrome at Penn Medicine

With a vision to strategically bridge the gap between pediatric and adult care for individuals with Williams Syndrome, a $25 million gift fromMichael R. Armellino, W61, will establish the Armellino Center of Excellence for Williams Syndrome at Penn Medicine. The center will serve as a model for coordinated care across the lifespan and provide social support and pioneering research for individuals with the genetic condition.

Williams Syndrome, which iscaused by deletions on chromosome 7q11, affects one in every 7,500 people.Children with Williams Syndrome generally have unusual social abilities while also navigating intellectual disability, anxiety, executive function challenges, predisposition for cardiovascular and metabolic disease, and a variety of other medical conditions. Seeking to develop a comprehensive program that meets the needs of patients with Williams Syndrome and their families throughout all stages of life, Penn Medicine will collaborate closely with Childrens Hospital of Philadelphia (CHOP), which is home to one of the largest Williams Syndrome clinics in the country, to enhance clinical and social services and advance scientific discovery toencompass the social, psychological, genetic, metabolic, and clinical complexity of Williams Syndrome.

Mr. Armellino,a resident of New Jersey who is retired after a long career at Goldman Sachs, has three sons and seven grandchildren. His partner, Beverly Karch, has a granddaughter, Maelyn, who is living with Williams Syndrome, as well as a grandson.

Maelyn, along with her younger brother and my grandchildren, are the lights of my life, Mr. Armellino said. I am so impressed by Maelyns parents Jenna and Corey, and all the parents Ive met who are part of the Williams Syndrome community. This gift is my way to advance genetic research and to help create a bright future for individuals with Williams Syndrome.

The new Armellino Center aims to ensure robust and expanded clinical experiences for individuals with Williams Syndrome that exceed the current standard of care. This will include integrated lifelong clinical care, behavioral health and executive function support, and social services coordination. The center will also spearhead basic, translational and clinical research on Williams Syndrome.

The Armellino Center of Excellence for Williams Syndrome will be a hub and international model for clinical care and research but, most importantly, it will be a welcoming home forthose touched by this condition, saidJ. Larry Jameson,executive vice president of the University of Pennsylvania for the Health System and dean of the Perelman School of Medicine.Individuals with Williams Syndrome face physical and intellectual challenges, coupled with a complex and uneven clinical care landscape. Long into the future, this generous gift will pave the way for improved care and scientific breakthroughs that will help us better care for individuals with Williams Syndrome, creating the highest possible quality of life for this community.

Penn Medicine and CHOP are the ideal places to integrate clinical care and biomedical research for Williams Syndrome across the lifespan. The new center will be overseen byDaniel J. Rader,chair of the department of genetics and chief of the division of translational medicine and human genetics in the department of medicine at the Perelman School of Medicine. Dr. Rader also serves as chief of the division of human genetics in the department of pediatrics at CHOP, where the Williams syndrome clinic is directed bySanmati Cuddapah.

Ive dedicated my career to stimulating collaborations that bring together some of the best clinicians and scientists from various disciplines to focus on common goals, Dr. Rader said. Combining our passion for excellent care and discovery with the enthusiastic Williams Syndrome community will lead to more support, advocacy andhopefullymore answers than ever before.

Jocelyn Krebs, former president of the Board of Trustees of the Williams Syndrome Association and a researcher who studied a gene in the Williams Syndrome deletion before having a child of her own with Williams Syndrome, is helping to launch the Armellino Center. An international recruitment process for a founding director of the Armellino Center is actively underway.

CHOP and Penn Medicine have a long history of collaboration to provide a continuum of care for patients with rare conditions like Williams Syndrome, saidJoseph W. St. Geme,Physician-in-Chief, Chairman of the department of pediatrics, andthe Leonard and Madlyn Abramson Endowed Chair in Pediatricsat CHOP. Our multispecialty Williams Syndrome clinic has provided critical services for many pediatric patients, and this new center will allow us to meet all of their care needs as they transition from childhood through adulthood.

The Williams Syndrome deletion on chromosome 7q11 includes 27 genes, but the effect of the deletion of most of these genes is not understood. Scientific discovery through the center will deepen understanding of the roles of multiple genes underlying Williams Syndrome, will accelerate translational research in novel diagnostics and therapeutics for Williams Syndrome, and will permit the detailed study of individuals with Williams Syndrome throughout the lifespan. Rapid scientific advances present major opportunities to gather and analyze data in new ways, allowing researchers to better understand how deletion of specific genes leads to particular Williams Syndrome symptoms and point to new pathways for therapeutic development.

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$25 Million Gift to Establish the Armellino Center of Excellence for Williams Syndrome at Penn Medicine - UPenn Almanac