Cure Rare Disease is a clinical-stage nonprofit biotechnology reimagining how rare disease therapeutics are developed and administered to patients. Through a collaboration of leading researchers, clinicians, regulatory experts and more, Cure Rare Disease advances life-saving therapeutics for patients impacted by rare neuromuscular diseases who lack an effective treatment or a cure. Cure Rare Disease is searching for a Chief Science Officer to advance our scientific mission forward—and ultimately help cure patients—by advancing drug development efforts in partnership with academic, CRO and CMO collaborators, technical grant writing to fund key needs, and scientific presentations at conferences and to industry to enable technology partnering.

This role is ideal for someone who is ambitious to shape the future of rare disease and wants to grow an organization that is making a real impact on patient’s lives and working toward the greater good. This position is responsible for leading the R&D function with the support of existing academic, CRO, CMO and consultant support. The position will work closely with the founder & CEO

Responsibilities & Qualifications

There are three main expectations for the applicant which include: 1) the ability to drive existing drug development efforts in collaboration with CRD’s academic, CRO/CMO and consultant groups; 2) lead technical grant and publication writing efforts in partnership with academic and consultant colleagues; 3) represent CRD in technical conferences and in biopharma partnership endeavors. This person would have the desire to see a change in outcomes within the world of fatal, rare diseases. Urgency is key.

Required Qualifications

Drug Development Expertise

• Successful track record of leading an interdisciplinary, advanced modality drug development team, from academic lab to IND approval
• 15+ years of successful experience in the biopharma industry with preference for experience in gene replacement, editing and antisense oligonucleotide modalities
• Extensive experience designing pharmacology and GLP toxicology studies with preference for experiences engaging with FDA and/or regulatory writing experience
• Engage with reimbursement work stream to drive refinement and adoption of novel financing plan for ultra-rare disease

Technical Writing

• Successful track record of technical grant writing resulting in grant funding (NIH, CIRM, foundations)
• Ability to lead collaborators through grant preparation and review process

Communications and Partnering

• Deep experiences with industry presentations especially around catalyzing research partnerships and licensing assets
• Ability to effectively present research findings at high impact conferences
• Experience leading manuscript preparation and publication process in high impact journals

Organizational

• Demonstrated ability to handle multiple projects in a fast-paced environment and perform under deadlines and changing schedule
• Develop and implement a short- and long-term organizational development plan to prioritize and advance assets into IND and early clinical trials
• Ability to develop budget to plan capital requirements for each drug development program
• Superb formal writing and organizational skills with a particular strength in relaying information in a concise and persuasive manner

Preferred Qualifications

• PhD in relevant field required (ex: genetics, immunology, muscle physiology)  
• Strong initiative and ability to self-start
• Ability to be a strong team player and willingness to work as part of a team with shared responsibility and accountability
• Desire to transform the paradigm of therapeutic development for rare diseases
• Ability to operate in a small, fast-paced, remote team

Other relevant information:

• Job Type: full time
• Education: PhD (Required)
• Work Location: Remote
• Salary: $200,000 – 225,000 + health benefits, 2-weeks’ vacation, performance bonus
• Travel: as needed, no more than 20% of time

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