Program Leader, Rare Disease Programs (MMA and PA), Investigational Medicines Office

The Role:

The Investigational Medicines Office at Moderna is responsible for leadership and management of the clinical-stage programs from IND to licensure. Moderna is seeking a motivated and energetic Program Leader for the MMA (mRNA-3704) and PA (mRNA-3927) rare disease programs to drive the program strategy and maintain accountability of program execution. The successful candidate will work in close coordination with the program manager to enable the execution of the set timelines. The program leader will lead a cross-functional team across clinical operations, regulatory affairs, clinical pharmacology, biomarkers, CMC, non-clinical, and medical to deliver against the program strategy. The successful candidate will have experience in late-stage readiness and development of rare disease programs.

Here's What You'll Do:

The Program Leader will be involved in high-level supervision of the programs:

• Lead the cross-functional teams for MMA and PA rare disease programs
• Represent the program status, scenarios, and trade-offs for MMA and PA with senior leadership, internal stakeholders, and external stakeholders
• Align on a program direction in coordination with senior functional leaders and program representatives
• Support development of regulatory documents and HA interactions
• Engage external stakeholders including KOLs and key PIs; engage advisory boards in order to develop an effective program strategy
• Accountable for the integrated development plan for MMA and PA programs
• Develop a program budget in coordination with the program manager, finance and functional representatives; identify trade-offs
• Work with senior leadership to support late-stage development activities including endpoints for registration, patient reported outcomes, and health-economics
• Work with corporate affairs to continue patient advocacy efforts for both MMA and PA

About mRNA-3704 (Methylmalonic Acidemia program)

The MMA program has open clinical sites for a Phase 1/2 clinical trial which is an open-label, multi-center, dose escalation study of multiple ascending doses of mRNA-3704 in pediatric patients with isolated MMA due to MUT enzyme deficiency. The objectives of the study are to evaluate safety and tolerability. mRNA-3704 has received Rare Pediatric Disease Designation by the FDA and Orphan Drug Designation by both the FDA and the European Medicines Agency.
About Methylmalonic Acidemia
Methylmalonic acidemia, or MMA, is a rare, autosomal recessive organic acidemia/aciduria, most commonly (approximately 60% of cases) caused by a deficiency of the enzyme methylmalonic CoA mutase (MUT), due to a defective or missing MUT protein.

MMA is primarily a pediatric disease with onset in early infancy. The majority of patients with MMA have no functional MUT enzyme, which disrupts the metabolic pathway and leads to a build-up of toxic methylmalonic acid in the blood and urine. This acid accumulation causes, on average, three life-threatening metabolic crises per year. As a result, MMA is associated with significant mortality and morbidity, and there are no approved therapies. Standard of care includes dietary and palliative measures. Currently, liver and/or kidney transplant is the only effective treatment.

About mRNA-3927 (Propionic Acidemia program)

The FDA has completed its review of the IND for mRNA-3927 Phase 1/2 trial. mRNA-3927 is designed to instruct the body to restore the missing or dysfunctional proteins that cause PA. mRNA-3927 contains two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC), encapsulated within Moderna's proprietary lipid nanoparticle (LNP). mRNA-3927 is intended to treat patients with PA regardless of whether they are missing the alpha or beta subunits.

About Propionic Acidemia

PA is a rare, life-threatening, inherited metabolic disorder that is the result of a deficiency in PCC that is an enzyme critical for metabolism. This deficiency can lead to a toxic buildup of acids in the body. Symptoms of PA typically become apparent during infancy and may include weak muscle tone, poor feeding, vomiting and lack of energy. More severe health problems can also occur, including heart abnormalities, seizures and coma.

The only effective treatment for severely affected individuals is liver transplant, which replaces the deficient PCC enzyme. Currently there are no approved therapies to treat the underlying cause of PA, including no enzyme replacement therapy, due to the complexity of the PCC enzyme that requires mitochondrial localization.

Here's What You'll Bring to the Table:

• BS/MS degree in a scientific field with 15+ years of experience or Ph.D or MD with 10-12 years of experience in a biotechnology or pharmaceutical setting
• Experience in rare disease drug development including the transition of novel candidates from discovery through licensure
• Proven track record of successful cross functional program leadership or management supporting early- and late-stage programs
• Strong ability to lead cross-functional teams; engage senior leadership, health authorities, and KOLs
• Prior experience managing or working within cross-functional project teams with timeline management and budgetary responsibility
• Must be adept at working in a fluid environment and foster a strong collaborative spirit internally and externally.
• Strong written and oral communication skills as well as demonstrated organizational aptitude

Here's What We'll Bring to the Table:

• Daily on-site catered lunches
• Company-provided iPhone
• Free parking, monthly subway pass or a subsidized commuter rail pass
• Free annual corporate membership to Bluebikes
• Highly competitive healthcare coverage including: medical offered through BCBS (HMO/PPO), dental, and vision offered through VSP
• Flexible Spending Accounts for medical expenses and dependent care expenses
• 16 weeks of 100% paid parental leave for all new parents
• 16 weeks 100% paid family caregiver leave
• 20 weeks 100% paid medical leave
• Eligible for "Moderna Month" (one month paid sabbatical after five years of service and eligible for additional one month paid sabbatical every 3 years thereafter)
• Adoption assistance and discounts to local childcare centers, as well as access to care.com
• 401k (traditional and Roth offered) with 50% match on first 6% deferred. Match is vested immediately
• A suite of Moderna paid insurance coverage, including: life insurance, short-term and long-term disability
• Voluntary legal assistance plan
• 15 days' vacation and 7 sick days per year, in addition to a discretionary winter shut down and 11 company paid holidays (includes 2 floating holidays)

Why join Moderna:

Moderna is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. mRNA medicines are designed to direct the body's cells to produce intracellular, membrane or secreted proteins that have a therapeutic or preventive benefit with the potential to address a broad spectrum of diseases. Moderna's platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, providing the Company the capability to pursue in parallel a robust pipeline of new development candidates. Moderna is developing therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases, independently and with strategic collaborators.

Headquartered in Cambridge, Mass., Moderna currently has strategic alliances for development programs with AstraZeneca, Plc. and Merck, Inc., as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense and the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS). Moderna has been named a top employer by Science for the fifth year in a row. To learn more, visit www.modernatx.com .

Our Mission:
Deliver on the promise of mRNA science to create a new generation of transformative medicines for patients.

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