Success Stories: Gene Therapy with Purpose: EB-2 NIW Approved for Brazilian Scientist Advancing Treatments for Neuromuscular Disorders

 

Client’s Testimonial:

“I am glad to have my petition approved, without RFEs. I appreciate your support in helping me obtain I-140 approval.”


On May 23rd, 2025, we received another EB-2 NIW (National Interest Waiver) approval for a Research Scientist in the Field of Gene Therapy (Approval Notice).


General Field: Gene Therapy

Position at the Time of Filing: Research Scientist

Country of Origin: Brazil

State of Residence at the Time of Filing: Ohio

Approval Notice Date: May 23rd, 2025

Processing Time: 1 month, 19 days (Premium Processing Requested)


Case Summary     

We proudly celebrate the EB-2 NIW (National Interest Waiver) approval of a Brazilian geneticist whose groundbreaking research in neuromuscular disease mechanisms and gene therapy is helping lay the foundation for next-generation treatments of devastating muscular disorders. With expertise in stem cell biology, RNA therapeutics, and satellite cell function, this scientist is actively addressing urgent national and global healthcare needs.

Rewriting the Code of Muscle Regeneration

The client’s proposed endeavor focuses on developing novel models of neuronal cells and applying gene therapy for the treatment of neuromuscular disorders. These disorders impose profound clinical and financial burdens, with no curative therapies currently available.

Through a series of preclinical studies and collaborative projects, she has:

  • Identified cell-type-specific disease signatures
  • Evaluated AAV-based gene therapies
  • Investigated satellite cell behavior across multiple dystrophic conditions

Her work demonstrates how precise gene targeting can enhance muscle regeneration, restore function, and improve outcomes in affected patients.

A Track Record of Influence and Impact

The client has authored 15 peer-reviewed journal articles, 17 abstracts, and 3 book chapters, with 360 total citations and multiple highly cited works:

  • A 2016 article in Stem Cells International has received 79 citations, placing it in the top 20% most cited for molecular biology and genetics that year.
  • A 2019 study in Scientific Reports has received 75 citations, placing it in the top 10% most cited.
  • A 2022 publication in Molecular Therapy – Methods & Clinical Development has received 24 citations, also ranking in the top 10% most cited.

Her articles appear in top-tier journals such as The FASEB Journal, PLOS One, and Frontiers in Cell and Developmental Biology. These venues consistently rank among the most influential in cell biology and genetics.

Endorsed by Experts in the Field

In a letter of recommendation, a senior researcher at a University emphasized:

“Her research into satellite cell behavior in neuromuscular diseases and muscle regeneration frameworks is foundational. These findings offer targeted therapeutic strategies that could shift treatment paradigms for DMD and CMD in the U.S.”

The letter also noted her expertise in comparative transcriptomics, CRISPR-adapted gene editing, and advanced AAV delivery systems, all of which contribute directly to the field’s growing therapeutic pipeline.

Aligned with U.S. Scientific and Health Priorities

Neuromuscular disorders collectively cost the U.S. healthcare system billions annually. DMD alone affects approximately 1 in 5,000 boys, with an annual cost of $50,000 per patient. MS and ALS add over $86 billion in total national burden. The client’s research directly addresses this challenge through biologically precise, scalable, and increasingly affordable gene therapy modalities.

Her work also supports several Critical and Emerging Technologies (CETs) outlined by the National Science and Technology Council, including:

  • Advanced biotechnology
  • Synthetic biology and nucleic acid engineering
  • Therapeutic genome editing
  • Viral delivery systems

Fast Approval Reflects Strong National Interest

Filed under premium processing on April 4, 2025, and approved on May 23, 2025, this petition met all three prongs of the Matter of Dhanasar framework:

  • Substantial merit and national importance
  • Well-positioned to advance the proposed endeavor

Benefits outweigh the need for labor certification

We’re proud to support scientists like this client, whose work brings hope to families affected by genetic diseases and ensures the U.S. remains at the forefront of therapeutic innovation. Her story is a powerful example of how gene therapy is not just a technology—it’s a path toward healing.


Histórias de Sucesso: Terapia Gênica com Propósito — EB-2 NIW Aprovado para Cientista Brasileira que Avança Tratamentos para Doenças Neuromusculares

Depoimento da Cliente:

“Estou feliz por ter minha petição aprovada, sem RFEs. Agradeço o apoio de vocês em me ajudar a obter a aprovação do I-140.”

Em 23 de maio de 2025, recebemos mais uma aprovação de EB-2 NIW (National Interest Waiver) para uma Cientista Pesquisadora na área de Terapia Gênica (Aviso de Aprovação).

Área Geral: Terapia Gênica
Cargo no Momento da Petição: Cientista Pesquisadora
País de Origem: Brasil
Estado de Residência no Momento da Petição: Ohio
Data do Aviso de Aprovação: 23 de maio de 2025
Tempo de Processamento: 1 mês e 19 dias (com Premium Processing)


Resumo do Caso

Celebramos com orgulho a aprovação do EB-2 NIW de uma geneticista brasileira cujo trabalho inovador em mecanismos de doenças neuromusculares e terapia gênica está ajudando a construir as bases para tratamentos de próxima geração contra distúrbios musculares devastadores. Com expertise em biologia de células-tronco, terapias baseadas em RNA e função de células satélites, esta cientista contribui ativamente para enfrentar necessidades urgentes de saúde nos EUA e no mundo.


Reescrevendo o Código da Regeneração Muscular

O projeto proposto pela cliente se concentra no desenvolvimento de novos modelos de células neuronais e na aplicação de terapia gênica para o tratamento de distúrbios neuromusculares. Esses distúrbios trazem impactos clínicos e financeiros profundos, e atualmente não existem terapias curativas.

Por meio de estudos pré-clínicos e projetos colaborativos, ela:

  • Identificou assinaturas de doenças específicas por tipo celular

  • Avaliou terapias gênicas baseadas em AAV

  • Investigou o comportamento de células satélites em múltiplas condições distróficas

Seu trabalho demonstra como o direcionamento gênico preciso pode ampliar a regeneração muscular, restaurar funções e melhorar os desfechos em pacientes afetados.


Um Histórico de Influência e Impacto

A cliente é autora de 15 artigos revisados por pares, 17 resumos e 3 capítulos de livros, acumulando 360 citações e diversos trabalhos altamente citados:

  • Um artigo de 2016, publicado na Stem Cells International, recebeu 79 citações, situando-se entre os 20% mais citados em biologia molecular e genética naquele ano.

  • Um estudo de 2019 na Scientific Reports recebeu 75 citações, ficando entre os 10% mais citados.

  • Uma publicação de 2022 na Molecular Therapy – Methods & Clinical Development recebeu 24 citações, também entre os 10% mais citados.

Seus artigos aparecem em periódicos de alto prestígio, como The FASEB Journal, PLOS One e Frontiers in Cell and Developmental Biology, todos amplamente reconhecidos por sua influência em biologia celular e genética.


Apoiada por Especialistas da Área

Em uma carta de recomendação, um pesquisador sênior de uma universidade destacou:

“Suas pesquisas sobre o comportamento de células satélites em doenças neuromusculares e arcabouços de regeneração muscular são fundamentais. Esses achados oferecem estratégias terapêuticas direcionadas que podem transformar paradigmas de tratamento para DMD e CMD nos Estados Unidos.”

A carta também ressalta sua expertise em transcriptômica comparativa, edição gênica adaptada ao CRISPR e sistemas avançados de entrega viral AAV, todas tecnologias essenciais para o desenvolvimento de novas terapias.


Alinhada às Prioridades Científicas e de Saúde dos EUA

Os distúrbios neuromusculares geram custos anuais de bilhões ao sistema de saúde dos EUA. A DMD, sozinha, afeta cerca de 1 em cada 5.000 meninos e possui custo anual de aproximadamente US$ 50.000 por paciente. Condições como MS e ALS somam mais de US$ 86 bilhões ao impacto nacional.

A pesquisa da cliente aborda diretamente esse desafio por meio de terapias gênicas biologicamente precisas, escaláveis e cada vez mais acessíveis.

Seu trabalho também apoia diversas Tecnologias Críticas e Emergentes (CETs) definidas pelo National Science and Technology Council, incluindo:

  • Biotecnologia avançada

  • Biologia sintética e engenharia de ácidos nucleicos

  • Edição terapêutica do genoma

  • Sistemas de entrega viral


A Aprovação Rápida Reflete Forte Interesse Nacional

Protocolada com premium processing em 4 de abril de 2025 e aprovada em 23 de maio de 2025, a petição atendeu plenamente aos três critérios do Matter of Dhanasar:

  1. Mérito substancial e importância nacional

  2. Boa posição da requerente para avançar o projeto proposto

  3. Benefícios que superam a necessidade de certificação trabalhista

Estamos orgulhosos de apoiar cientistas como esta cliente, cujo trabalho traz esperança para famílias afetadas por doenças genéticas e contribui para manter os EUA na vanguarda da inovação terapêutica. Sua história é um exemplo poderoso de como a terapia gênica vai além da tecnologia — ela abre caminhos para a cura.

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