Alan Walts

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates that are designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx’s lead product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis and cystinosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx is headquartered in Waltham, MA, with R&D operations in Rehovot, Israel.

Neuroelectrics® is a digital brain health company that innovates at the intersection of neuroscience, physics, machine learning, and hardware. We have developed wireless EEG and tDCS/tACS/tRNS stimulation devices with up to 32 channels, amongst other products encompassing software and research services, with the purpose of monitoring and enhancing brain health.

At Aura Biosciences, we are developing a new class of therapies to target and destroy cancer cells selectively, while leaving surrounding tissue unharmed – an approach we call molecular surgery. By safely eliminating cancer locally, we can treat early and transform the lives of people with a wide range of cancers that are poorly managed today. Our lead program in ocular melanoma (OM), also known as choroidal or uveal melanoma, is designed to remove cancer cells in the back of the eye as a first-line therapy, while allowing for the potential of preserving patients’ vision. The goal is to treat small ocular melanomas potentially long before the disease progresses and metastasizes to the liver, where it almost always is fatal. Development of a first-in-class, non-radioactive treatment option to selectively destroy cancer cells would create the possibility to transform the treatment of this and other cancers where the disease can be detected early. By enabling physicians to treat cancer more selectively, effectively and safely than they can do today, we aim to eliminate the need for invasive procedures that carry significant morbidity, including vision loss and often do little to improve a patient’s overall survival.

Arrakis has a simple but powerful vision: To extend small-molecule medicines into new realms of biology, unlocking that biology for medicine. Our approach builds on the existing sophisticated drug discovery toolkit developed for protein targets, which we are adapting and refocusing on discovering small-molecule compounds that act directly on RNA. We are pioneering new territory, with an expedition led by a team of skilled scientists and drug development leaders who are inventive yet pragmatic. We are on a bold and exciting mission, a mission not without peril, but we have an energized, collaborative, creative and fearless team – a team that has consistently breached barriers and blazed new trails in search of solutions. We are now poised to realize the promise of bringing RNA into play for small-molecule drug discovery. Pushing the frontier of this emerging field of science is gratifying, but our mission goes beyond the thrill of scientific discovery. We believe that by targeting RNA biology in new ways, we have the potential to impact millions of patients. Our platform will allow us to reach hundreds of biological targets not currently accessible to medicine, including known targets previously considered “undruggable.” Our destination is powerful new therapies for patients everywhere, by opening up a new path for discovering RNA-targeted medicines.

We have an audacious mission to develop novel therapies for communities with high unmet needs. Where others see challenges, Amylyx sees opportunities that we pursue with urgency, rigorous science, and unwavering commitment. We explore scientific approaches that are tailored to diseases where we can make the greatest difference, agnostic of modality, in order to usher in a new era of advancements. See our community guidelines here: https://www.amylyx.com/social-media-guidelines Please be aware of potential phishing scams imitating Amylyx. All authentic communications from Amylyx will be from the Amylyx.com domain.

Artax Biopharma is a development-stage biopharmaceutical company dedicated to the development of new therapies for autoimmune and inflammatory diseases. Artax Biopharma is developing the next generation of drugs targeting the interaction between TCR and Nck, which is responsible for T cell activation. Specific control over T cells through TCR, provides the compounds with the potential to become first-in-class immunomodulators for the treatment of a wide range of autoimmune and inflammatory diseases.

Arrakis has a simple but powerful vision: To extend small-molecule medicines into new realms of biology, unlocking that biology for medicine. Our approach builds on the existing sophisticated drug discovery toolkit developed for protein targets, which we are adapting and refocusing on discovering small-molecule compounds that act directly on RNA. We are pioneering new territory, with an expedition led by a team of skilled scientists and drug development leaders who are inventive yet pragmatic. We are on a bold and exciting mission, a mission not without peril, but we have an energized, collaborative, creative and fearless team – a team that has consistently breached barriers and blazed new trails in search of solutions. We are now poised to realize the promise of bringing RNA into play for small-molecule drug discovery. Pushing the frontier of this emerging field of science is gratifying, but our mission goes beyond the thrill of scientific discovery. We believe that by targeting RNA biology in new ways, we have the potential to impact millions of patients. Our platform will allow us to reach hundreds of biological targets not currently accessible to medicine, including known targets previously considered “undruggable.” Our destination is powerful new therapies for patients everywhere, by opening up a new path for discovering RNA-targeted medicines.

We are a commercial-stage clinical biopharmaceutical company driven to improve the lives of people with rare diseases of the immune system and few to no treatment options. We have successfully developed mavorixafor, receiving FDA approval for XOLREMDI® (mavorixafor) in its first indication, and are currently executing our US launch. We are also advancing mavorixafor for additional rare disease patient populations, and expect to initiate a pivotal, global Phase 3 clinical trial in people with certain chronic neutropenic disorders. With a focus on CXCR4 and immune system biology, we continue to leverage our expertise both at our corporate headquarters in Boston, Massachusetts and at our research center of excellence in Vienna, Austria to further advance our patient-centric mission.

PIC Therapeutics is a biotechnology company focused on fundamentally changing how we treat cancer by developing a new generation of therapeutics based on the modulation of RNA translation. PIC Therapeutics was founded on the seminal work of Dr. Gerhard Wagner, a professor at Harvard Medical School world-renowned for his scientific expertise in protein structure and his research on interactions of eukaryotic translation initiation factors (eIFs). PIC Therapeutics, led by President and CEO Dr. Kathy Bowdish, was launched to translate these discoveries into small molecule precision therapeutics to benefit cancer patients. Our precision-based therapeutics target the “master switch” of cancer signaling pathways, selectively blocking oncogene protein production by modulating the Pre-Initiation Complex (PIC) that drives their mRNA translation. Our selective approach has the potential to simultaneously modulate multiple oncogenic drivers leading to a powerful new generation of cancer-treating therapeutics.

We recognize that most children with rare genetic diseases will never see a treatment, not because the science doesnt exist, but because our system refuses to adopt a new sustainable business model. AlphaRose Therapeutics is a new precision medicine model for genetic treatments. We are built on the shoulder of giants like Henri Termeer, Emil Kakkis, and Dr. Tim Yu. We are born from the incredible stories of precision medicine for children, like Rose McPherson. We exist to bring treatments globally to suffering children with autism, special needs, and other neuro-genetic diseases.

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates that are designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx’s lead product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis and cystinosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx is headquartered in Waltham, MA, with R&D operations in Rehovot, Israel.

Neuroelectrics® is a digital brain health company that innovates at the intersection of neuroscience, physics, machine learning, and hardware. We have developed wireless EEG and tDCS/tACS/tRNS stimulation devices with up to 32 channels, amongst other products encompassing software and research services, with the purpose of monitoring and enhancing brain health.

At Aura Biosciences, we are developing a new class of therapies to target and destroy cancer cells selectively, while leaving surrounding tissue unharmed – an approach we call molecular surgery. By safely eliminating cancer locally, we can treat early and transform the lives of people with a wide range of cancers that are poorly managed today. Our lead program in ocular melanoma (OM), also known as choroidal or uveal melanoma, is designed to remove cancer cells in the back of the eye as a first-line therapy, while allowing for the potential of preserving patients’ vision. The goal is to treat small ocular melanomas potentially long before the disease progresses and metastasizes to the liver, where it almost always is fatal. Development of a first-in-class, non-radioactive treatment option to selectively destroy cancer cells would create the possibility to transform the treatment of this and other cancers where the disease can be detected early. By enabling physicians to treat cancer more selectively, effectively and safely than they can do today, we aim to eliminate the need for invasive procedures that carry significant morbidity, including vision loss and often do little to improve a patient’s overall survival.

Arrakis has a simple but powerful vision: To extend small-molecule medicines into new realms of biology, unlocking that biology for medicine. Our approach builds on the existing sophisticated drug discovery toolkit developed for protein targets, which we are adapting and refocusing on discovering small-molecule compounds that act directly on RNA. We are pioneering new territory, with an expedition led by a team of skilled scientists and drug development leaders who are inventive yet pragmatic. We are on a bold and exciting mission, a mission not without peril, but we have an energized, collaborative, creative and fearless team – a team that has consistently breached barriers and blazed new trails in search of solutions. We are now poised to realize the promise of bringing RNA into play for small-molecule drug discovery. Pushing the frontier of this emerging field of science is gratifying, but our mission goes beyond the thrill of scientific discovery. We believe that by targeting RNA biology in new ways, we have the potential to impact millions of patients. Our platform will allow us to reach hundreds of biological targets not currently accessible to medicine, including known targets previously considered “undruggable.” Our destination is powerful new therapies for patients everywhere, by opening up a new path for discovering RNA-targeted medicines.