Huntington Disease

NEW YORK, March 19, 2024 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL) ("Seelos"), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today provided an update on top-line data of the Phase 2/3 HEALEY ALS Platform trial. This study was performed in collaboration with The Sean M. Healey and AMG Center, which is viewed as an influential force in ALS research and in caring for the ALS community. Their unique and innovative approach continues to be a benefit to the ALS community and its contributions have helped bring the last two FDA approved therapies for ALS to market. We plan to request a meeting with the FDA to discuss potential next steps for the program The study was designed to evaluate SLS-005 (IV trehalose), a low molecular weight disaccharide that stabilizes misfolded proteins and activates autophagy, in decreasing the slope of the ALS Functional Rating Scale (ALSFRS-R) and separation from placebo in Function and Mortality in an all-comers population of Persons with ALS (PALS). While the study did not meet statistical significance in the primary and secondary endpoint in the Full Analysis Set (FAS)2, by showing a 13% improvement in Function and Mortality with an 88% success probability (versus the pre-specified 98%), it showed a potential signal of efficacy in a pre-specified subgroup (ERF)1. In the pre-specified subgroup of PALS treated with SLS-005, without RELYVRIO®, the top-line data favored SLS-005 versus placebo in efficacy measures in the Efficacy RELYVRIO® Free (ERF)1 data set (n=130), including: a 22% improvement in slope of change in ALSFRS-R assessment adjusted for mortality, with an 89% success probability, at 24 weeks the rate of decline in ALSFRS-R slope (points per month) also favored the SLS-005 treatment group versus placebo over 6 months (-0.80 and -1.07 points per month, respectively) a 25% slowing of Slow Vital Capacity (SVC) decline versus placebo (-11.5% for SLS-005 and -15.4% for placebo) at 24 weeks Additional analysis and information from these data accessible here. "The HEALEY platform is designed to detect signals of efficacy and we believe the observed signal and success probability is competitive to other recently FDA-approved therapies for ALS which also failed to achieve statistical significance when measured for function and mortality on similar primary and efficacy endpoints," said Raj Mehra Ph.D., Chairman and Chief Executive Officer of Seelos. "We plan to request a meeting with the FDA to discuss potential next steps for the program and will continue our potential partner discussions." Seelos has not yet received the full dataset and upon receiving it in the near future, Seelos plans to run additional analyses, including biomarkers of neurodegeneration, neurofilament light chain (NfL), exploratory efficacy results, subgroups and post-hoc analyses. SLS-005 was generally well-tolerated and comparable to placebo in safety. There was an imbalance of deaths/death equivalents observed in the study, with more events seen in the SLS-005 group compared to placebo, all were considered unrelated to the study drug. Populations – ERF1, FAS2 and ERO3: Seelos received top-line results for Regimen E (RGE). This included a Full Analysis Set (FAS), an Efficacy Regimen Only (ERO) population and an Efficacy RELYVRIO® Free (ERF) population. FAS includes shared placebo from all regimens, including RGE (n=204) and the RGE SLS-005 participants (n=120). The Efficacy Regimen Only (ERO) population includes 120 RGE SLS-005 and 41 RGE placebo participants. 31 participants in the ERO population received RELYVRIO® during the SLS-005 RGE study, thus confounding results from this population. The ERF analyses exclude participants who initiated RELYVRIO® during the study. ERF represents the participants who followed the protocol as envisioned (n=130, i.e., the ERF population comprises 81% of ERO/RGE participants). About SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) SLS-005 is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier and is thought to stabilize proteins and activate autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. The activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material. In animal models of several diseases associated with abnormal cellular protein aggregation or storage of pathologic material, SLS-005 has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. SLS-005 has previously received Orphan Drug Designation for the treatment of ALS from the U.S. Food and Drug Administration and from the European Medicines Agency in the EU. SLS-005 is an investigational treatment and is not currently approved by any health authority for medicinal use. About Amyotrophic Lateral Sclerosis (ALS) According to the National Institute of Neurological Disorders and Stroke, amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die and stop sending messages to the muscles. Unable to function, the muscles gradually weaken, start to twitch (called fasciculations), and waste away (called atrophy). Eventually, the brain loses its ability to initiate and control voluntary movements. The disease is progressive, meaning the symptoms get worse over time. The majority of ALS cases (90 percent or more) are considered sporadic. This means the disease seems to occur at random with no clearly associated risk factors and no family history of the disease. Although family members of people with sporadic ALS are at an increased risk for the disease, the overall risk is very low, and most will not develop ALS. Most people with ALS eventually die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. However, about 10 percent of people with ALS survive for 10 or more years. Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. A 1-point change in the ALSFRS-R score can indicate a meaningful difference in a person's ability to function independently with activities of daily living (ADL's), including eating, bathing, dressing or walking.4 4. FDA Center for Drug Evaluation and Research, Application Number 209176Orig1s000, Office Director Memo, Ellis F. Unger, MD, May 4, 2017. Paganoni S, et al. New Eng J Med. 2020. About Seelos Therapeutics Seelos Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system (CNS) disorders and other rare diseases. The Company's robust portfolio includes several late-stage clinical assets targeting indications including Acute Suicidal Ideation and Behavior (ASIB) in Major Depressive Disorder (MDD), amyotrophic lateral sclerosis (ALS) and spinocerebellar ataxia (SCA), as well as early-stage programs in Huntington's disease, Alzheimer's disease, and Parkinson's disease. For more information, please visit our website: , the content of which is not incorporated herein by reference. Forward Looking Statements Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, among others, those regarding the Phase 2/3 study of SLS-005, statements regarding the competitiveness of the results to recently approved FDA therapies for ALS, statements regarding any planned meetings and discussions with the FDA, statements regarding The Sean M. Healey and AMG Center's contributions to the ALS community, statements regarding the timing of Seelos's receipt of the full dataset for the trial, statements regarding additional analyses to be run by Seelos and statements regarding SLS-005's prospects and any future analyses that may be conducted. These statements are based on Seelos' current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Risks associated with Seelos' business and plans described herein include, but are not limited to, the risk of not successfully executing its preclinical and clinical studies, not being able to move forward with the development of SLS-005, and not gaining marketing approvals for SLS-005 and/or its other product candidates; the risk that prior clinical results may not be replicated in future studies and trials (including the risk that the results from the prior studies of SLS-005 may not be replicated or may be materially different from the results of the top-line data from the SLS-005 study); the risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval; the risks associated with the implementation of Seelos' business strategy; the risks related to raising capital to fund its development plans and ongoing operations; the risks related to Seelos' current stock price; as well as other factors expressed in Seelos' periodic filings with the U.S. Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we do not know whether our expectations will prove correct. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, even if subsequently made available by us on our website or otherwise. We do not undertake any obligation to update, amend or clarify these forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Contact Information: Anthony Marciano Chief Communications Officer Seelos Therapeutics, Inc. (Nasdaq: SEEL) 300 Park Avenue, 2nd Floor New York, NY 10022 (646) 293-2136 anthony.marciano@seelostx.com Mike Moyer Managing Director LifeSci Advisors, LLC 250 West 55th St., Suite 3401 New York, NY 10019 (617) 308-4306 mmoyer@lifesciadvisors.com Company Codes: NASDAQ-NMS:SEEL

Novel AAV capsid engineered by Sangamo showed robust penetration of blood-brain barrier (BBB) and widespread transgene expression throughout brain in non-human primates (NHPs) following intravenous administration. Demonstrated industry-leading brain tropism and enrichment in NHPs, resulting in 700-fold higher transgene expression than benchmark capsid AAV9. Capsid-enabled delivery of zinc finger payloads targeting prion disease and tauopathies resulted in robust and widespread repression of target genes. STAC-BBB capsid could potentially unlock multiple neurology epigenetic regulation programs paused by Sangamo pending identification of suitable capsid and could be advanced either internally or with a collaborator. Sangamo expects to to three neurology Investigational New Drug (IND) submissions and/or Clinical Trial Applications (CTA) by end of 2025. Sangamo to discuss results in conference call scheduled for Wednesday, March 13 at 4:30pm Eastern Time. RICHMOND, Calif.--(BUSINESS WIRE)-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced preclinical data from its proprietary adeno-associated virus (AAV) capsid variant, STAC-BBB, that demonstrated robust penetration of the blood-brain barrier (BBB) and strong transgene expression throughout the central nervous system (CNS) of NHPs when administered intravenously at clinically-relevant doses, outperforming results obtained by Sangamo for other known neurotropic capsid variants evaluated in the study. Importantly, potent repression of target genes was observed in brain cells expressing the zinc finger cargo, indicating that STAC-BBB could enable development of genomic medicines to potentially treat a wide range of neurological diseases. “The advancement of neurological medicines has long been limited by the inability to achieve widespread CNS delivery, an essential attribute for an effective treatment for devastating neurological disorders. We are extremely encouraged that STAC-BBB, a potentially game-changing capsid variant engineered using our SIFTER capsid platform, demonstrated results that outperformed other known neurotropic capsid variants, achieving widespread brain delivery and transgene expression, desired de-targeting of liver and other peripheral tissues and a favorable safety profile,” said Sandy Macrae, Chief Executive Officer of Sangamo. “Furthermore, these data demonstrated that delivery of our zinc finger epigenetic regulators using STAC-BBB could result in a meaningful repression of disease-relevant target genes throughout the brain, which we believe may result in profound improvements in disease pathology and progression. These data further support our transformation into a neurology-focused genomic medicine company focused on combining potent epigenetic regulation capabilities with innovative capsid delivery technology to develop best-in-class neurology medicines.” Sangamo intends to use the novel STAC-BBB capsid in its wholly owned prion disease and tauopathy programs. In addition, STAC-BBB could potentially unlock multiple neurology epigenetic regulation programs that were paused by Sangamo pending the identification of a suitable capsid, including programs previously in development under Sangamo’s former collaboration agreements with Biogen and Novartis. Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators. Summary of STAC-BBB Preclinical Data In NHP studies when administered intravenously at clinically relevant doses, STAC-BBB demonstrated its potential to be a leading neurotropic capsid. Highlights include: Broad brain coverage. Robust penetration of the BBB and widespread transgene expression throughout the brain, including key regions integral to human neurological diseases such as Alzheimer’s disease, Parkinson’s disease, Amyotrophic Lateral Sclerosis (ALS), Huntington’s disease and prion disease. Industry-leading brain tropism. Exhibited 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the study. Widespread neuronal transduction across all animals. STAC-BBB mediated robust expression of zinc finger cargo in neurons, the key cell type to target for treatment of neurological diseases. Moreover, results were dose-dependent and consistent across all NHPs in the study. Potent and widespread repression of target genes. Capsid-enabled delivery of zinc finger payloads resulted in the repression of prion and tau genes across key brain regions, demonstrating the potential for modification of disease progression in prion disease and various tauopathies. Visualization of gene expression in individual brain cells by RNAscope revealed highly potent repression of tau in neurons expressing the zinc finger cargo across multiple brain regions. Desired de-targeting of the liver and other peripheral organs. Capsid biodistribution was shown to be enriched in the CNS and de-targeted from the liver, dorsal root ganglia (DRG) and other peripheral organs. This biodistribution pro by STAC-BBB is optimal for an AAV-based treatment of neurological diseases. Favorable safety profile. STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues. Manufacturable using standard processes and at scale. We believe STAC-BBB is manufacturable at commercial scale using standard cell culture and purification processes, is soluble using known excipients, and can be characterized using available analytics. Sangamo expects to IND submission for its Nav1.7 program addressing chronic neuropathic pain, which leverages an intrathecally administered capsid, in the fourth quarter of 2024, and a CTA submission for its prion disease program, which is expected to leverage the STAC-BBB capsid, in the fourth quarter of 2025, each subject to additional funding. Sangamo also intends to resume development of its tau program leveraging the STAC-BBB capsid, with an IND submission expected as early as the fourth quarter of 2025. The Sangamo management team will discuss these results on a conference call on Wednesday, March 13 at 4:30pm Eastern Time. Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly. An updated corporate presentation is available in the Investors and Media section under Presentations. The link to access the live webcast can also be found on the Sangamo website in the Investors and Media section under Events. A replay will be available following the conference call, accessible at the same link. About Sangamo Therapeutics Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo’s zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and Sangamo’s capsid discovery platform is expanding delivery beyond currently available intrathecal delivery capsids, including in the central nervous system. Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit and connect with us on LinkedIn and Twitter/X. Forward-Looking Statements This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: expectations regarding the therapeutic potential of Sangamo’s technologies, including the delivery of zinc finger epigenetic regulators using STAC-BBB to result in the repression of disease-relevant target genes in the brain, to result in profound improvements in disease pathology and progression, to modify disease progression in prion disease and various tauopathies, to be manufacturable at commercial scale using standard cell culture and purification processes, to be soluble using known excipients, and to be characterized using available analytics; Sangamo’s intentions to use STAC-BBB in two of its epigenetic regulation product candidates under development internally; expectations that preclinical data from STAC-BBB will support Sangamo’s transformation into a neurology-focused genomic medicine company; expectations regarding STAC-BBB’s potential to unlock multiple neurology epigenetic regulation programs that have been paused by Sangamo pending the identification of a suitable capsid, and plans to either resume their development internally, with receipt of additional funding, or with a new potential collaborator; Sangamo’s plans to submissions and/or CTAs and the expected timing of such regulatory submissions; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the uncertain and costly research and development process, including the risk that preclinical results may not be indicative of results in any future clinical trials; Sangamo’s lack of capital resources to fully develop, obtain regulatory approval for and commercialize its product candidates, including Sangamo’s ability to secure a partnership or the funding required to initiate planned IND and/or CTA submissions in a timely manner or at all; Sangamo’s need for substantial additional funding to execute its operating plan and to continue to operate as a going concern, including the risk that Sangamo will be unable to obtain the funding necessary to advance Sangamo’s Nav1.7, prion disease and tau programs and to otherwise continue to operate as a going concern, in which case, Sangamo may be required to cease operations entirely, liquidate all or a portion of its assets, and/or or seek protection under applicable bankruptcy laws; the effects of macroeconomic factors or financial challenges, including as a result of the ongoing overseas conflict, current or potential future bank failures, inflation and elevated interest rates, on the global business environment, healthcare systems and business and operations of Sangamo and our collaborators; the research and development process; the potential for technological developments that obviate technologies used by Sangamo; Sangamo’s reliance on collaborators and its potential inability to secure additional collaborations, and Sangamo’s ability to achieve expected future operating results. There can be no assurance that we and our current or potential future collaborators will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators. These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in its Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

Could you give us an overview of your work? ​ I am the chief innovation officer at Maravai Life Sciences, the parent company to several life science brands, including TriLink BioTechnologies, Alphazyme, and Cygnus Technologies. I direct the research and development focus for the company and lead where we should be going in the future. Altogether, we specialize in nucleic acid products and services, including enzyme development and biologics safety testing. Our work is focused on building holistic partnerships with therapeutic developers, helping them overcome manufacturing challenges to streamline and scale research through clinical trials to commercialization. We mostly support nucleic acid therapeutic developers focused on DNA or RNA but are now expanding into cell and gene therapy applications. The COVID-19 pandemic was a massive inflection point for our work because of the immeasurable impact of the mRNA vaccines. How can we utilize mRNA outside of COVID-19? How should we be extending our reach in different areas? What other RNA molecules should we be prioritizing and incorporating into the therapeutics of the future? These are the types of questions we’re trying to answer through our work – It's an incredibly exciting and rewarding job to do. When did you realize you were interested in science - as a young child/teen/older? ​ As a young child, I was very interested in the medical and science fields which made my educational path from secondary school to graduate school very simple. From the beginning, I was completely fascinated by how science was applied within a medical setting. How could theoretical learning be employed to improve people's lives? What started in my childhood as a pure fascination continued on into my career, where I've been so lucky to be able to push science forward and hopefully make an impact on patient lives. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Could you describe your personal journey bringing us to where you are now? ​ Springing from my passion for science and medicine at a very early age, I decided to study genetics at the University of Glasgow. After finishing my Bachelor's degree, I jumped at the opportunity to complete my PhD in molecular genetics, where I worked with animal models to act as surrogates for a human disease model. Though it was an incredible experience, I felt a pull toward more translational medicine so I could connect with actual patients. Chasing that instinct, I enrolled in a post-doctorate program at the University of California – San Diego, applying my work on animal disease models to human beings. Moving across the world was an overwhelming, but incredibly rewarding experience. What began with a three-year student visa has turned into a 20-year tenure in San Diego. As I neared the end of my postdoc program, I felt I still wasn’t close enough to the clinical applications of my work. When I finished my post-doc program, I began a position as an entry-level bench scientist working on clinical trials for DNA-based systems and medicines at Inovio Pharmaceuticals. I joined when the company only had 15 employees, so I was able to wear a lot of different hats and be extremely involved in clinical development, the regulatory process, clinical application, manufacturing, and the CMC. I definitely wouldn't have had the opportunity to do so if I had gone into a bigger corporate structure. In my 15 years at Inovio, I rose from entry-level scientist to Senior Vice President of Research, saw two products go through phase three clinical trials, and was able to see how our development work at the lab bench directly impacted patients. Reflecting, one of the most rewarding elements of my career was visiting clinical trial sites to speak with doctors and nurses, and then explain our technology to the patients. I give patients so much credit for enrolling in clinical trials – they show such passion, interest, and enthusiasm for therapeutic advancements that likely won’t make a direct difference to their lives but will for patients in the future. After my 15-year tenure, I joined Maravai to lead the research and development team. I saw this move as an exciting challenge to continue leading innovation and research but doing so for a different technology set – RNA rather than DNA. What challenges did you face along the way and what is the most valuable lesson you have learned? What were some key lessons you learned from your experience supporting the global response to the COVID-19 pandemic? ​ I can’t emphasize enough the importance of doing work outside of your comfort zone. In my early days as a bench scientist, I was asked to support clinical development. Despite my lack of experience, rolling up my sleeves and learning on the spot helped expand my capabilities and allowed me to be the leader I am today. When you get an opportunity to learn something that isn't necessarily in your area of knowledge, jump at it! One of the most pivotal learning moments of my career was my dual role in the global response to the COVID-19 pandemic. When the first cases were reported, I and a tour de force of scientists with completely different backgrounds worked together around the clock to create a safe, effective vaccine. In addition to that work, I was later requested to be a spokesperson responsible for communicating the COVID-19 vaccine progress and its science to the masses. My background is in infectious disease, not ​ mass communications – this was completely out of my comfort zone. I spoke at the World Health Organization COVID-19 response meetings to relay clinical development updates and answer any questions. This experience proved how important it is for scientists to clearly communicate 1. what R&D teams are doing, 2. What mechanisms are involved, and 3. why we're doing it in specific ways. The majority of the population understandably doesn’t have 20 years of experience in life sciences. Making sure the advancements within our industry are available and easily interpreted by the wider population is the first step to the global understanding of the importance of science and therapeutic research and development. What is your current work ethos/style? ​ Throughout my career, my work ethos has been rooted in communication. The continuous articulation and emphasis of the vision and value of a company’s work helps those from multifactorial backgrounds collectively push toward the same goal. At TriLink and Maravai, the R&D scientists are clearly aware of the internal product development and advances in the wider field. A crucial aspect of my role is distilling those updates to the majority of the team with less visibility, making sure we understand and leverage this excitement for mRNA advancements beyond COVID-19 vaccines. If your team can believe in the company’s work, they’ll be excited to come to work every day to push these new, exciting medicines forward. What ignites your passion in your current role? ​ Right now, the future use of mRNA ignites my passion. A silver lining of the pandemic was the general public saw the power and potential of mRNA through the success of the COVID-19 vaccination. Now, we are able to expand into other capabilities. The standard of care for cancer treatment has been chemotherapy for a long time. There have been some recent immunotherapy advancements, but many patients will tell you it’s a painful treatment to experience. We are already seeing some impressive results from clinical trials for personalized mRNA cancer vaccines. The data we are seeing could entirely shift the paradigm of future cancer treatment. I’m incredibly excited about the CRISPR approvals at the end of last year in the U.S. and Europe. Before then, there were no treatment options for thousands of monogenic diseases – only symptom management. This approval will open up the doors for the treatment of other devastating diseases such as Huntington's disease, cystic fibrosis, and more. What most excites you about the future of this field? What roadblocks do you foresee? ​ In the next 15 years, medicine will evolve into a more personalized approach. With that said, there are challenges to face in the field of mRNA. For instance, the mRNA COVID-19 vaccine is delivered successfully in the body with a lipid nanoparticle (LNP), but that molecule targets only the liver. There are many diseases for which we want to target the heart, brain, skin, etc. Many companies are working to develop the delivery molecules for those specific areas. Another roadblock is the instability of mRNA as a molecule. During the COVID-19 pandemic, there was a rush to get freezers in all doctor’s offices because the vaccines needed to be kept at a specific temperature. In medical centers and large metropolitan areas, we can have big -80 freezers. However, in a rural clinic or developing nations, accessibility becomes an issue. We need to increase mRNA’s stability, so it can be accessible beyond urban areas and developed countries. Could you share some advice for young women starting to develop an interest in science or wanting to pursue a career like yours? ​ In 2024, women are almost 60% of life science graduates but only 20% of CEOs in life science companies. This lack of women rising to leadership positions is frustrating. As a mother of a young daughter, it's incredible that to this day, it's common that I'm the only woman in the room at a scientific meeting, and certainly the only female executive. I could bend your ear about how many times I've been the only woman in a room, and they’ve mistaken me as somebody who's there to take notes. I try to take it all with a sense of humor – getting angry won’t benefit me – and I make it my goal to prove my worth in that room. I do believe that over time we will see increased diversity within those settings. If you are a young woman in STEM, continue to fill your network with mentors, both men and women, who have been in your shoes and can be sounding boards for your ideas. If I could give advice to women, I would encourage them to pursue a career in STEM if it interests them. I'm quite actively involved in STEM for girls mentoring programs, and I’m humbled to work to ensure that those young women view STEM as a valid career path and that when they enter into the field, they have the support to learn and progress forward to the positions they want.