Answered prayers for help in seeking cures of deadly childhood diseases?

Recently blessed for its work by Pope Francis, Bamboo Therapeutics, a young life sciences venture focusing on gene therapy to treat deadly childhood neurological disorders, has raised nearly $50 million from investors.

CureDuchenne Ventures LLC, a venture philanthropy organization, is among the backers.

The funding of $49,467,472 was disclosed in a securities filing on Thursday.

In January, Bamboo announced an undisclosed funding investment from CureDuchenne, a California-based firm seeking a cure for Duchenne, a childhood disease. The amount of the investment was not disclosed, but the venture firm did receive a board seat.

Bamboo, co-founded by entrepreneurs Sheila Mikhail and R. Jude Samulski, Ph.D., director of the Gene Therapy Center at the University of North Carolina at Chapel Hill, said six investors participated in the funding. The minimum investment was $300,000.

Investors in California, Delaware, New York, North Carolina and Texas participated.

Bamboo executives will receive $840,000 from the investment.

Wall Street financial services firm Piper Jaffray will receive a $3 million sales commission for helping raise the money, according to the filing.

Last November, Samulski and a scientific collaborator, Paola Leone, a gene therapy researcher at Rowan University in Glassboro, N.J., were invited to join a group of childhood disease patients and families for a private meeting with Pope Francis at the Vatican in Rome.

“It was very special experience,” Samulski told Barry Teater in a story written for the N.C. Biotechnology Center. “The families and I were touched that the Pope would take the time to meet with us.”

(Read the full story about Bamboo at )

The Pope blessed the audience and wished the researchers success.

CureDuchenne’s backing

CureDuchenne’s focus is on an emerging gene therapy called BMB-D001 that has shown positive effects in Duchenne animal models. Life span , skeletal and cardiac muscle function in both mice and rats were improved. “In a canine Duchenne animal model, long-term dystrophin production resulted in life extension and maintenance of ambulation up to eight years post-treatment,” Bamboo said in the January announcement.

“We have dedicated our careers to harnessing the power of gene therapy to correct devastating genetic diseases,” said Samulski. “We are excited to collaborate with CureDuchenne as we move our therapeutic into the clinic.”

Duchenne is a fatal genetic disease that causes muscles to degenerate and affects some 1 in 3,500 boys. “Those with Duchenne are usually diagnosed by age 5, lose their ability to walk by 12 and most don’t survive their mid-20s. There is currently no approved treatment or cure for Duchenne,” Bamboo notes.