Category Archives: Gene Medicine

AstraZeneca has announced a collaboration with UK biotech Silence Therapeutics to develop gene silencing drugs for cardiovascular, renal, metabolic and respiratory diseases.

The deal could see AZ adding small interfering RNA (siRNA) technology to its pipeline in one of its main areas of research.

Silence Therapeutics says its technology can selectively inhibit any gene in the genome, specifically silencing the production of disease-causing proteins.

The UK biotech says it has achieved an additional level of accuracy by delivering its therapeutic RNA molecules exclusively to target cells.

siRNA is a technology that showed much promise after biologists Andrew Fire and Craig Mello received the Nobel Prize in Physiology or Medicine for discovering the technology in 2006.

But in 2010 it became apparent that it was harder to convert into a working therapy because of the challenge of delivering therapeutic RNA molecules to target tissues and big pharma quickly lost interest.

But companies like Silence have managed to overcome this hurdle and its rival Alnylam made history in 2018 when its siRNA drug Onpattro was approved by the FDA to treat hereditary transthyretin (hATTR) amyloidosis, which causes the build-up of amyloid protein in nerves and organs.

AZ will pay $60 million up front and invest $20 million in Silence, and will pay up to $400 million in milestone payments plus tiered royalties.

The companies expect to work on five targets within the first three years of the collaboration, and AstraZeneca has the option to extend it to a further five targets.

Silence has technology that can inhibit liver-expressed gene targets and the companies will collaborate to find siRNA molecules to other tissues including the heart, kidney and lung.

The UK biotech will design SiRNA molecules against gene targets selected by AZ, and will manufacture material to support toxicology and phase 1 clinical studies.

AZ and Silence will collaborate during the discovery phase, while AZ will lead clinical trials and marketing.

Silence will have the option to negotiate for co-development of two drugs of their choice starting from phase 2.

AZ will pay an option fee of $10 million for each selected target when a drug candidate is nominated, and Silence could receive up to $140 million in development milestones, and up to $250 million in market milestones.

Link:
AZ adds gene silencing tech to pipeline with Silence Therapeutics deal - - pharmaphorum

Redpin Therapeutics closed on a $15.5 million Series A financing round. The round was led by 4BIO Capital and Arkin Bio Ventures. They were joined by new investor Takeda Venture Investments, as well as existing seed-round investors, New York Ventures and Alexandria Venture Investments.

Based in New York City, Redpin has a proprietary chemogenetics platform for targeted cell therapies. Its a mix of synthetic biology, gene therapy and traditional pharmacotherapy. The focus is built on an ultrapotent ion channel-based chemogenetics platform that allows targeted cell activation or inhibition controlled by low doses of the Pfizers anti-smoking drug varenicline (Chantix). The company has a worldwide exclusive license from the Howard Hughes Medical Institute for therapeutic use of the technology.

The funds will let Redpin continue to progress its platform to disorders with neural circuit dysfunction, including epilepsy, neuropathic pain and Parkinsons disease. Treatment for these usually uses systemic drugs that target local neuron dysfunction. This has the downside of adverse, off-target side effects. The lead programs are for epilepsy and chronic pain.

Redpins approach, the company believes, will be more targeted on the dysfunctional neurons while not affecting normal functioning cells. The company indicates its approach will only be activated in the presence of Chantix.

These new funds combined with the support and expertise of our new and existing investors will allow Redpin to swiftly progress to the next phase of its development in bringing highly targeted treatments to patients with neurological and psychiatric disorders, said Elma Hawkins, co-founder, president and chief executive officer of Redpin.

Chantix attaches to proteins called ion channels, which control neuron signaling. By controlling which neurons receive these proteins, researchers can modulate specific cells. In March 2019, Scott Sternson, group leader at the Howard Hughes Medical Institutes Janelia Research Campus, noted that chemogenetics often use molecules that would not be appropriate for human therapy. Its still many steps to the clinic, but were trying to shorten that route.

Sternson is one of the companys founders, along with Hawkins, Jeffrey M. Friedman at Howard Hughes, Michael Kaplitt, with Weill Cornell Medicine, Sarah Stanley at Icahn School of Medicine Mount Sinai, and Jonathan S. Dordick, Rensselaer Polytechnic Institute.

At that time, Sternson and his team modified the structure of two different ion channel proteins so the drug would be more likely to bind. One protein stimulates neurons to send messages when Chantix attaches. Another protein blocks neurons from sending those messages when Chantix is present. At that time, doses of Chantix much lower than required to quit smoking were found to have a large effect on neural activity.

Redpins technology uses adeno-associated virus (AAV) vectors to transport engineered ion channels to targeted cells. Once activated, they can control the function of the particular cell. Chantix was chosen because it is approved in 80 countries, has the necessary pharmacokinetic properties, and can penetrate the blood-brain barrier. The company has other small molecule-receptor pairs in its pipeline.

Chantix basically acts as a switch to turn the ion channel on and off.

Dmitry Kuzmin, managing partner at 4BIO, said, Our goal is to support and grow advanced therapy companies with the potential to cure chronic disease. Redpin has a highly compelling, validated chemogenetics approach that could have significant potential in the targeted treatment of neuropathic disorders. The strength of Redpins science alongside the world-class knowledge and expertise of the Companys founders and management team make us fully confident in the future success of the Company towards this goal.

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Redpin Launches with $15.5 Million Series A to Focus on Pain and Epilepsy - BioSpace

TAMPA, Fla. & DUBLIN--(BUSINESS WIRE)-- Vycellix, Inc., an immuno-discovery cell & gene therapy company, and Avectas Limited, a cell engineering technology business, today jointly announced that the companies have entered into a collaboration agreement to develop proprietary approaches for cell-based immunotherapeutic products.

The companies will collaborate on the delivery of Vycellix's novel RNA immunomodulator VY-M using Avectas' cell engineering platform, Solupore. The collaboration will address current limitations for cell-based therapies, in particular with respect to the need to accelerate the manufacturing process, reduce the cost of manufacture, and ultimately improve patient outcomes.

"We are delighted to partner with Vycellix and join forces in the development of novel cell-based products," stated Michael Maguire, Ph.D., CEO of Avectas. "We believe Solupore will play a critical role in the manufacture of cell-based therapies and will support a path towards effective patient outcomes."

According to Vycellixs President, Douglas Calder, Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our product candidate, VY-M, to T cells and natural killer (NK) cells. We expect to accelerate the expansion-time of T cells and NK cells by decreasing the non-dividing lag time, resulting in much shorter vein-to-vein delivery-time to patients. The studies will be conducted at Avectas Dublin-based facility and at Karolinska Institutet, Stockholm, Sweden.

Both Vycellix and Avectas are collaborative partners within NextGenNK, a newly established competence center for development of next-generation NK cell-based cancer immunotherapies based at Karolinska Institutet, Stockholm, Sweden. It is envisioned that Vycellix and Avectas will further expand their collaboration within the NextGenNK constellation.

We are excited to see the NextGenNK Competence Center catalyzing interactions among its industrial partners to advance NK cell-based immunotherapies, said Hans-Gustaf Ljunggren, M.D., Ph.D., Director of the NextGenNK Competence Center. The present collaboration may pave the way for similar collaborations among NextGenNK partners.

About Vycellix, Inc.: Vycellix is a private, immuno-discovery, life science company at the forefront of innovation in the development of cell & gene-based therapies targeting indications in, but not limited to, hematology/oncology, autoimmunity/chronic inflammatory diseases, and organ/tissue transplantation.

The Companys portfolio of transformational platform technologies encompass novel tools urgently sought after to enable broad global adoption of advanced therapies including: 1) the ability to generate Universal Cells (VY-UC), without the need to alter expression of any of the cellular components that control self-recognition (HLA Class I or II), obviating the need for immune-suppressive drugs and redefining the path towards off-the-shelf therapies; 2) the ability to amplify cell-potency through the upregulation of internal cytotoxic mechanisms (VY-X); 3) the ability to accelerate the expansion of cells for immunotherapy by near-elimination of non-dividing lag time to leap forward to shorter vein-to-vein time with expanded cells (VY-M); and, 4) the ability to markedly enhance gene transduction levels using viral vectors with implications for autologous and allogeneic CAR-T and CAR-NK cell development (VY-OZ).

The Companys platforms were all discovered by scientists at the world-renowned Karolinska Institutet (KI) in Stockholm, Sweden. KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine. For more information, please visit the Companys website at: http://www.Vycellix.com and follow its Twitter feed at: @Vycellix.

About Avectas Limited: Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex-vivo manufacture of our partners' gene-modified cell therapy products, which will retain high in-vivo functionality. Our vision is to be a leading non-viral cell engineering technology provider, integrated into manufacturing processes for multiple autologous and allogeneic therapies, commercialized through development and license agreements. For more information, please visit the Company's website at http://www.avectas.com.

Forward Looking Statements: This press release contains forward-looking statements. All statements other than statements of historical facts are forward-looking statements, including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as plan, expect, anticipate, may, might, will, should, project, believe, estimate, predict, potential, intend, or continue and other words or terms of similar meaning. These statements include, without limitation, statements related to the pre-clinical, regulatory, clinical and/or commercial development and all anticipated uses of VY-OZ, VY-X, VY-M and VY-UC, and the Companys plans for seeking out-licensing opportunities for these assets. These forward-looking statements are based on current plans, objectives, estimates, expectations and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with immuno-discovery product development, including risks associated with advancing products to human clinical trials and/or ultimately regulatory and commercial success which is subject to the uncertainty of regulatory approval, market adoption and other risks and uncertainties affecting Vycellix and its development programs. Other risks and uncertainties of which Vycellix is not currently aware may also affect Vycellixs forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. Vycellix undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200325005026/en/

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Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace

It is well known that neurofibromin (NF1), a tumor suppressor produced by the NF1 gene, keeps cancer growth in check by repressing the activity of a cancer driver gene called Ras. It then follows that when NF1 is lost, Ras can drive cancer growth by promoting treatment resistance and metastasis. NF1, however, can do more than regulate Ras.

Drs. Eric C. Chang, Matthew Ellis and Zeyi Zheng at Baylor College of Medicine and their colleagues have discovered new insights into the function of neurofibromin that improve our understanding of breast cancer resistance and suggest novel therapeutic approaches to overcome it.

The team first studied the importance of neurofibromin loss in a study they published in 2018. In this study, they sequenced tumor DNA seeking for mutations that can promote resistance to tamoxifen, a drug commonly used to prevent relapses from estrogen receptor positive (ER+) breast cancer.

When we examined the mutational patterns in NF1, we observed that poor patient outcome only occurred when neurofibromin was lost, not through mutations that selectively affect Ras regulation. This suggested that neurofibromin may have more than one function, said Chang, co-corresponding author of this work and associate professor in the Department of Molecular and Cellular Biology and a member in the Dan L Duncan Comprehensive Cancer Centers Lester and Sue Smith Breast Center.

This thought triggered studies, spearheaded by Zheng in Changs lab, into the function of neurofibromin in ER+ breast cancer cells. One of his early experiments showed that when expression of NF1 is inhibited (to mimic neurofibromin loss in tumors), the resulting ER+ breast cancer cells were stimulated by tamoxifen instead of inhibited, as it usually happens. Furthermore, these neurofibromin-depleted cells became sensitive to a very low concentration of estradiol, a form of estrogen.

The clinical relevance of these findings was immediately apparent because it suggested that tamoxifen or aromatase inhibitors, which lower estrogen levels available to the cancer cells, would be the wrong choice for treatment when neurofibromin is lost by the tumor, said Ellis, co-corresponding author and professor and director of the Lester and Sue Smith Breast Center. Dr. Ellis also is a McNair Scholar at Baylor.

Follow-up gene expression studies all strongly suggested that neurofibromin behaves like a classic ER co-repressor.

A co-repressor must bind ER directly, but the group hesitated to conduct such an experiment without more evidence because it is not trivial to do so, Chang said.

A breakthrough came when Dr. Charles Foulds, a co-author on the paper and assistant professor at the Center for Precision Environmental Health at Baylor, searched the Epicome, a massive proteomic database created by Dr. Anna Malovannaya and Dr. Jun Qin, also at Baylor. This is a part of an effort by Dr. Bert OMalley, chancellor and professor of Baylors Department of Molecular and Cellular Biology to comprehensively document all the proteins associated with ER.

Foulds found neurofibromin in the database, which encouraged the team to ultimately investigate whether estrogen receptor and neurofibromin interacted directly. However, to seriously consider NF1 as an ER co-repressor, there was still another missing piece of the puzzle.

One day Charles casually asked me whether neurofibromin had a region rich in the amino acids leucine and isoleucine, because co-repressors use these regions or motifs to bind ER, and it dawned on me that neurofibromin indeed does, Chang said. In fact, neurofibromin has two such motifs that mediate ER binding in a cooperative manner. These motifs are frequently mutated in cancer, but are not required for Ras regulation.

Since tamoxifen or aromatase inhibitors were found to be ineffective for neurofibromin-deficient ER+ breast cancer tumors, the researchers worked with animal models to determine whether the ER-degrading drug fulvestrant was still effective. However, fulvestrant only temporarily inhibited tumor growth because secondary Ras-dependent fulvestrant resistance was induced by neurofibromin loss. This Ras-dependent growth phase could be inhibited with the addition of a MEK inhibitor, which shuts off a key signaling pathway downstream of Ras.

The team validated this combination treatment strategy using a patient-derived xenograft (PDX) mouse model. In this model, a section of a human tumor taken from a patient is directly transplanted into a mouse under conditions that maintain the genomics and drug response of the original human tumor from which it was derived (Cell Reports, 2013). In this case, this PDX was derived from a patient who failed several lines of endocrine therapy and had already developed fulvestrant resistance.

The results of the combination of fulvestrant to degrade ER and a MEK inhibitor (e.g., selumetinib or binimetinib) to inhibit Ras downstream signaling, were encouraging the tumor shrunk to almost undetectable levels, Chang said.

Our next goal is to test this combination therapy in clinical trials in order to determine its therapeutic potential in the clinic.

Neurofibromin is lost in at least 10 percent of metastatic ER+ tumors. As a result of these new data, we are now working on a clinical trial that combines a MEK inhibitor with fulvestrant, said Ellis, Susan G. Komen scholar and associate director of Precision Medicine at the Dan L Duncan Comprehensive Cancer Center at Baylor. Interestingly, MEK inhibitors are also being used to control peripheral nerve tumors in patients with neurofibromatosis, where a damaged NF1 gene is inherited. Our findings contribute to an understanding of why female neurofibromatosis patients also have a much higher incidence of breast cancer.

Other contributors to this work include Meenakshi Anurag, Jonathan T. Lei, Jin Cao, Purba Singh, Jianheng Peng, Hilda Kennedy, Nhu-Chau Nguyen, Yue Chen, Philip Lavere, Jing Li, Xin-Hui Du, Burcu Cakar, Wei Song, Beom-Jun Kim, Jiejun Shi, Sinem Seker, Doug W. Chan, Guo-Qiang Zhao, Xi Chen, Kimberly C. Banks, Richard B. Lanman, Maryam Nemati Shafaee, Xiang H.-F. Zhang, Suhas Vasaikar, Bing Zhang, Susan G. Hilsenbeck, Wei Li and Charles E. Foulds. The authors are affiliated with one or more of the following institutions: Baylor College of Medicine, Chongqing Medical University, Adrienne Helis Malvin Medical Research Foundation, Zhengzhou University and Guardant Health.

This work appears in Cancer Cell,

See the publication for a complete list of the sources of support for this work.

By Ana Mara Rodrguez, Ph.D.

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Meet a superhero that fights breast cancer, neurofibromin - Baylor College of Medicine News

California: In a breakthrough study, researchers have found a potential treatment for life-threatening cardiac diseases by using gene therapy.

Danon disease is a very rare, life-threatening condition where the fundamental biological process of removing and recycling proteins does not work.

This impairment results in dysfunction of the heart, skeletal muscle, neurologic system, eyes, and liver. Most patients die or require heart transplants by the third decade of life.

In the study, which was published in Science Translational Medicine, researchers have identified a novel way to treat Danon disease using gene therapy.

Heart transplant is not always available for patients and does not treat the other organs affected in Danon disease. We knew we needed to find therapies specifically designed to address the underlying cause, said the lead researcher Eric Adler.

Danon disease is a result of mutations in the gene LAMP2. For nearly a decade, Adler and a team of researchers at UC San Diego Health have been working to determine whether gene therapy could provide a new treatment approach.

Gene therapy involves either replacing or repairing a gene that causes a medical problem or adding genes to help the body treat disease. In this case, Adler and the team focused on adding a specially designed gene that restores the LAMP2 function, resulting in improved cardiac and liver function.

We utilised mice that were a model for Danon disease and missing this specific LAMP gene. We applied gene therapy to a group of these mice and compared to mice that did not receive treatment, said Adler.

The mice that received gene therapy expressed positive results in heart, liver and muscle function. The hearts overall function of ejecting blood and relaxing improved, as did the bodys ability to degrade proteins and metabolism.

Danon disease is more common in males, and symptoms begin in early childhood or adolescence.

In many cases, the condition is inherited by a parent, typically the mother. We believe Danon disease is actually more common than we think, but it is often misdiagnosed, said Adler.

By utilising gene therapy, we were able to identify a possible new treatment approach other than a heart transplant. This study is a significant step for patients with Danon disease, Adler added.

Prior studies in Adlers lab have focused on using a patients skin cells to create stem cells. These stem cells were used to create a heart model, allowing researchers to study Danon disease at the cellular level.

The approach has provided new insight into the diseases pathology and led to the idea of using gene therapy. Our work is also proof that using stem cells to model diseases has great potential for helping develop new medicines, said Adler.

The next step, said Adler, is testing in patients with Danon disease. A Phase I clinical trial for safety and efficacy has begun.

This is the first trial using gene therapy to treat a genetic cardiac disorder and three patients are currently being treated, which means were that much closer to finding a cure for this terrible disease, and may be able to use similar methods to treat other diseases, said Adler.

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Study reveals gene therapy may help in treating cardiac disease - The Siasat Daily

The BioHealth Capital Region (BHCR) and its life science ecosystem have a rich and deep history of pioneering scientific innovation, research, development, and commercialization. The regions history has been written by life science anchor companies, scientific research universities, government research organizations, rich startup culture, and serial entrepreneurs, all of whom have played critical roles in transforming the BHCR into one of the most innovative and productive biocluster in the world.

Contributions to the BHCRs legacy of life science achievement have emerged from all staffing levels, various labs, countless executive teams, numerous entrepreneurs and biohub support organizations. Contributions have arisen from an intricate tapestry of backgrounds and cultures.

Women, in particular, have had a strong hand in shaping the history of the BHCR. In celebration of Womens History Month, were taking a closer look at the achievements of female life science leaders that have laid the groundwork for the next generation of women trailblazers in the BHCR and made the region what it is today.

Dr. Fraser is one of the most influential figures in BHCR history. In 1995, she was the first to map the complete genetic code of a free-living organism while at the Institute for Genomic Research (TIGR) in Rockville, Maryland. It was there that the automation of the DNA sequencing process made the idea of large-scale sequencing efforts tangible. As President and Director of TIGR, Fraser and her team gained worldwide public notoriety for its involvement in the Human Genome Project, which was completed in 2000 with the presentation of a working draft of the fully sequenced human genome.

As a leader, Fraser provided her researchers with the infrastructure to collaborate and apply multi-disciplinary team science and empowered them to think big. She is also most importantly known for how she challenged her team to ask the right questions, which is the root of scientific progress and success.

Her work at TIGR and as part of the Human Genome Project are foundational events in the regions history, as it marked the BHCR as the epicenter of genomic research and helped spark the regions biotech boom. In fact, it was a controversial partnership with TIGR that gave Human Genome Sciences(HGSi) the first opportunity to utilize any sequences emerging from TIGR labs. The mass of genetic information and sequences, especially that associated with diseases, that HGSi acquired catapulted them into biotech history and an important anchor company within the region.

Dr. Fraser is widely viewed as a pioneer and global leader in genomic medicine; she has published approximately 320 scientific publications and edited three books; she is also one of the most widely cited microbiology experts in the world. She founded the Institute for Genome Sciences at the University of Maryland in 1997. The institute currently holds 25 percent of the funding thats been awarded by the Human Microbiome Project and has been referred to as The Big House in genetics.

Dr. Judy Britz is yet another female life science pioneer that put the BHCR on the map. While working as a research scientist at Electro-Nucleonics Inc., Dr. Britz developed one of the first licensed blood screening tests for HIV, and launching a storied career that has spanned approximately 25 years. She is also a serial entrepreneur that has successfully raised $50M in capital and served as the top executive for two highly successful Maryland-located companies.

Dr. Britz was the first woman to lead the states biotech initiative as the first announced Executive Director of the Maryland Biotech Center. The center was launched under the Maryland Department of Commerce to deploy a strategic life science economic development plan under Governor Martin OMalleys $1.3B, 2020 Vision and to be a one-stop-shop and information center to promote and support biotechnology innovation and entrepreneurship in Maryland.

Judy was the first woman to lead Marylands life sciences initiative, bringing industry experience and perspective to the states economic development activities, a focus still maintained under Governor Hogans leadership today, shared Judy Costello, Managing Director, Economic Development BioHealth Innovation, Inc., who served as Deputy Director under Dr. Britz.

Much of the work done by Dr. Britz and her team laid the foundation and seeded the commercialization efforts that have blossomed into the thriving #4 Biotech Hub that we have today.

GeneDx was founded by Dr. Bale and Dr. John Compton in 2000. The company recently celebrated its 20th anniversary. Since its founding, GeneDx has become a global leader in genomics and patient testing. Under her leadership, the Gaithersburg, Maryland company has played an important role in the history of genetic sequencing and the rise of the BHCR as a global biohealth cluster.

GeneDx was the very first company to commercially offer NGS (Next Generation Sequencing) testing in a CLIA (Clinical Laboratory Improvement Amendments) lab and has been at the leading edge of genetic sequencing and testing for two decades. The companys whole exome sequencing program and comprehensive testing capabilities are world-renowned.

Prior to launching GeneDx, Dr. Bale spent 16 years at NIH, the last nine as Head of the Genetic Studies Section in the Laboratory of Skin Biology. She has been a pioneer during her storied career, publishing over 140 papers, chapters and books in the field. Her 35-year career includes deep experience in clinical, cytogenetic, and molecular genetics research.

Prior to being named CEO and Chair of the Board of Sequella in 1999, Dr. Nacy was the Chief Science Officer and an Executive VP at EntreMed, Inc. EntreMed was one of the most influential BHCR companies in the 1990s. EntreMed, MedImmune, Human Genome Sciences and Celera Genomics all played critical roles in creating the globally recognized, top biocluster that the BHCR has become.

After earning her Ph.D. in biology/microbiology from Catholic University, Nacy did her postdoc work at the Walter Reed Army Institute of Research in the Department of Rickettsial Diseases; her postdoc performance earned a full-time position at Walter Reed that started a 17-year career at the institute. After a highly successful run, Nacy left Walter Reed to join EntreMed.

Today, Dr. Nacy leads Rockville, Marylands Sequella, a clinical-stage pharmaceutical company focused on developing better antibiotics to fight drug-resistant bacterial, fungal and parasitic infections. Sequellas pipeline of small molecule infectious disease treatments have the potential to improve the treatment and outcomes for the over 3 billion people worldwide that are impacted by increasingly drug-resistant infectious diseases.

Emmes Corporation is the largest woman-led organization in the BHCR and is headed by Dr. Lindblad, who started her career at Emmes in 1982 as a biostatistician. She has been with Emmes for nearly 40 years, ascending to become VP in 1992, Executive VP in 2006 and ultimately the companys CEO in late summer of 2013.

Dr. Lindblad has published more than 100 publications and presentations has served as a reviewer of grant and contract applications for the National Institutes of Health (NIH) and has chaired or served on Safety and Data Monitoring Committees across multiple disease areas. Emmes is a life science anchor company for the BHCR, employing more than 600 staff globally with its headquarters in Rockville, Maryland.

Under Kings leadership, GlycoMimetics (GMI), an oncology-focused biotech, went public, secured an exclusive global licensing agreement with Pfizer and was instrumental in raising significant amounts of capital for the company. She was also the first woman Chair of Biotechnology Innovation Associations (BIO, 2013-14), where she still plays an active role on BIOs Executive Committee.

A graduate of Dartmouth College and Harvard Business School, King has had a celebrated career in both biopharma and finance. Prior to becoming CEO of GMI, King served as an Executive in Residence for New Enterprise Associates (NEA), one of the leading venture capital firms in the U.S. She has also held the position of Senior Vice President of Novartis-Corporation. King joined Novartis after a remarkable ten year run with Genetic Therapy, Inc. where she was named CEO after helping Genetic Therapy navigate the organization through various growth stages, including the companys sale to Novartis. King was named the Maryland Tech Councils Executive of the Year in 2013, the Top 10 Women in Biotech by FierceBio and has served on multiple boards across her career.

Dr. Connolly has had a pioneering career in the life sciences. She was the very first woman to graduate from Johns Hopkins Universitys Biomedical Engineering Doctoral Program in 1980. She was also a member of the first female undergraduate class entering Stevens Institute of Technology in 1971.

For decades, Dr. Connolly tirelessly worked to build up what is now known as the BHCR. In 1997, shortly before the region gained wider recognition as a biotech hub, she was the first person to be designated the state of Marylands biotechnology representative. Dr. Connollys career has spanned academia, government, and industry, including co-founding a startup and working as the Business Development Director for EntreMed, Inc., an original BHCR anchor company. She is the former Director of Maryland Industrial Partnerships Program (MIPS) and was inducted into the College of Fellows by the American Institute for Medical and Biological Engineering (AIMBE) in 2013.

Dr. Kirschstein played an enormous role in shaping the BHCR as NIH Deputy Director from 1993 to 1999 during the regions early formative years. She also served as Acting Director of NIH in 1993 and from 2000 to 2002. A pathologist by training, she received her medical degree from Tulane University in 1951 and went on to a long, successful career at the Division of Biologics Standards that lasted from 1957 to 1972.

While at the Division of Biologics Standards, Dr. Kirschstein played an important role in testing the safety of viral vaccines and helped select the Sabin polio vaccine for public use. She eventually ascended to Deputy Director of the group in 1972 and was later appointed the Deputy Associate Commissioner for Science at the FDA. In 1974 she became the Director of the National Institute of Medical Sciences at NIH and served in that role for 19 years.

Her awards and accolades are too numerous to list, but one notable honor came in 2000 when she received the Albert B. Sabin Heroes of Science Award from the Americans for Medical Progress Education Foundation.

Lastly, we want to recognize four additional women for their contributions to launching an organization that has impacted thousands of women by promoting careers, leadership, and entrepreneurship for women in the life sciences Women In Bio.

Women In Bio (WIB), one of the most important and influential support organizations for women in the life sciences, was founded in 2002 to help women entrepreneurs and executives in the Baltimore-Washington-Northern Virginia area build successful bioscience-related businesses. WIB started as a BHCR organization but has expanded its footprint to 13 chapters across the U.S. with 225 volunteer leaders and 2,600 members. The non-profit group has created a forum for female life science entrepreneurs and executives based on its core philosophy of women helping women.

WIB founders are Anne Mathias, a local venture capitalist and current Senior Strategist with Vanguard;

Elizabeth Gray, co-founder of Gabriel Pharma and current Partner at Willkie Farr & Gallagher LLP;

Robbie Melton, former Director of Entrepreneurial Innovation at TEDCO and current Director of Kauai County, Hawaiis Office of Economic Development;

and Cynthia W. Hu, COO, and General Counsel at CASI Pharmaceuticals.

In conclusion, we can not fairly capture the true history of life science and the BioHealth Capital Region without giving special recognition to Henrietta Lacks. In 1951 a Johns Hopkins researcher created the first immortal human cell line from cervical cancer cells taken from Lacks. That cell line, known as HeLa, is the oldest and most commonly used human cell line which was essential in developing the polio vaccine and has been used in scientific landmarks such as cloning, gene mapping and in vitro fertilization.

Though she was a black tobacco farmer from southern Virginia, her impact on science and medicine is unquestionable. She never knew that the Doctor took a piece of her tumor that would be used by scientists who had been trying to grow tissues in culture for decades without success. For some reason, that is still unknown, but her cells never died and the first immortal human cell line was born.

Thank you to all of the women who have been so influential in shaping the field of science, the industry of biotechnology and the BioHealth Capital Region.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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Twelve Women Who Have Shaped The History of the BioHealth Capital Region - BioBuzz