Frequently Asked Questions
Who is Perlara PBC?
Perlara PBC was originally founded as Perlstein Lab, PBC in February 2014. Perlara, PBC is a biotech platform company and Public Benefit Corporation (bioPBC) based in the San Francisco Bay Area that is on a mission to discover personalized cures for diseases rare and common.
We are a demographically diverse team with expertise in genetics, pharmacology, cell biology, data science and automation. Working with families, advocacy groups and BioPharma companies, we share an enthusiasm for scientific discovery and a commitment to transparency and engagement.
Take a look at our 2017 Public Benefit Corp Report to see how our mission has provided a roadmap for our activities and accomplishments.
What is the Genetisphere?
Perlara scientists are working with highly motivated families to develop cures for rare diseases and learn how genes work across diseases to map connections to common ailments. We call these exciting journeys of discovery PerlQuests™.
Perlara Drug Discovery Platform is based on creating disease models using simple, whole animals (yeast, nematodes, fruit flies and zebrafish) that share genetic similarity with humans, allowing us to screen massive numbers of disease models and drug candidates quickly and at low cost.
We know that disease symptom patterns are shared by individual patients and disease symptom patterns are shared across diseases. PerlQuests explore the genetics that all animal life on earth shares—what we call the Genetisphere.
What is a PerlQuest?
How does a PerlQuest work?
What is the science behind PerlQuests?
What are the goals of a PerlQuest?
- To partner with families, advocacy groups and BioPharma companies to create more, better and affordable treatments that extend and improve quality of life.
- To accelerate and de-risk drug discovery for a specific rare disease to a point of partnership with a BioPharma company.
- To share the value of intellectual property with all PerlQuest collaborators.
- To share scientific learning with all PerlQuest participants and their communities, as well as the world at large for the greater good.
Who are Perlara’s partners?
How can I start a PerlQuest?
How does my family or organization qualify for a PerlQuest?
What kinds of organizations can start a PerlQuest?
Can other families and organizations join my PerlQuest?
What information or genetic samples are required to start a PerlQuest?
What is a PerlQuest Research Plan?
Once we establish a fit for a PerlQuest, we customize a plan specifying research objectives, timeline and deliverables in multiple stages. Included in the PerlQuest Research Plan is genetic information and scientific references relating to your rare disease.
What is a PerlQuest Collaboration Agreement?
Once we have agreed upon a PerlQuest Research Plan, Perlara offers a PerlQuest Collaboration Agreement that outlines the terms of the project such as the team, research program, costs, timeline, reports, publication and intellectual property (IP) rights. Once we have all reviewed and agreed upon the final terms of the agreement, it is signed by an official representative of each party engaged in the PerlQuest and becomes a legal contract.
Can I name my PerlQuest?
Absolutely! Be our guests at creating meaning, paying tribute to a loved one or expressing your intentions for the greater good. Perlara offers the opportunity to name a PerlQuest to reflect you and your family or organization. Need help in expressing your identity? Our marketing team is expert at branding and can develop a banner for your community.
How much does a PerlQuest cost?
You can start Phase I for $50,000 -$100,000 and we can quote the next two stages when customizing your PerlQuest Research Plan. Depending on the nature and complexity of your disease and desired research goals, the costs will vary.
Need help raising money to launch or continue to another PerlQuest phase? Our PerlQuest development team can help brainstorm funding options, as well as you can review fundraising resources at your leisure.
Complete the information form to initiate your PerlQuest.
Who pays for a PerlQuest?
An individual, family, organization or group pays for a PerlQuest. We’re a mix and match kind of organization who values flexibility and options. That extends to relationships. A PerlQuest can be paid in full by a single family or organization. Alternatively, different PerlQuest constituents can pay for different stages. We can help connect families and organizations to join forces to start a PerlQuest fundraising campaign.
Does my health insurance cover a PerlQuest?
A PerlQuest is a research project aimed at discovering definitive therapeutics for rare diseases. Health insurers do not fund drug discovery, so as such, do not cover PerlQuests. Join our mailing list to keep in the loop about potential changes in this area in the future.
Can I pay for a PerlQuest with my health savings account (HSA) or medical savings account (MSA)?
How can I raise funds for a PerlQuest?
Great fundraisers push the envelope. They put on their creative hats and ask how can donors or partners benefit from giving their money to a cause or project—addressing the question, “What’s in it for them?”.
PerlQuests can improve the quality of life for rare disease patients, and potentially, for patients with common diseases like Parkinson’s, Alzheimers and Cancer. An important aspect, distinct from other drug discovery research, is that PerlQuests are funded directly by families and patient groups, who in exchange receive joint ownership of the resulting data and inventions. Consequently, a donation to your PerlQuest now can grow resources to sustain an organization in the future. For prospective partners, the benefit of shared intellectual property (IP) is attractive to signing on .
Fundraise for a PerlQuest as you would for a school, team, nonprofit and other causes. Fundraising activities include major gifts; corporate giving such as matching gifts and sponsorships; special events (galas, silent auctions, walkathons, etc); grantseeking; product sales (t-shirts, garage sales, bake sales, etc); and annual appeals.
Online fundraising has become the norm and can be an effective way of raising funds and awareness about your PerlQuest. If you’re a charitable organization, create a mobile-optimized donation page and promote it to your constituents on Facebook, Twitter, Instagram, Pinterest and other online channels, as well as through offline communications such as donation letters, postcards and flyers. Set up a “Donate Now” call-to-action button for use on your Facebook company page and in linked ads. Reach existing and attract new donors though the power of peer-to-peer fundraising (also known as Crowdfunding). Mobile charitable giving, including text-to-give and mobile friendly email marketing, has proven to be very successful in raising funds for nonprofits.
These articles are chockfull of ideas to rev up your fundraising activities:
Remember that fundraising for your PerlQuest raises awareness about the 1 in 10 families affected by rare disease and the need for research into treatments to improve quality of life.
We are happy to share more fundraising information and support. Simply fill out the PerlQuest form and provide some information about your disease and fundraising activities.
How can my community share in the cost of my PerlQuest?
Extended family, friends, neighbors, colleagues, other families who have a loved one with the same disease, or a community at large can contribute to funding your PerlQuest. Equity partners in your PerlQuest share ownership with you in accordance with the amount of funds contributed. Similar to funding a cause or nonprofit, donors give you money to advance your PerlQuest without the expectation of a financial return. The joy of giving is in bringing the PerlQuest to fruition for the benefit of orphan disease patients and their families. Another approach is debt financing where your community loans you funds which have to be repaid at a future date without taking an ownership stake.
The number of partners, donors and debtors ranges from one to many. Cost-sharing approaches may require fundraising prior to starting, and in some cases, during a PerlQuest. Crowdfunding, or peer-to-peer fundraising, is ideal for large communities.
Like any financial arrangement, we recommend consulting a lawyer in setting up a community sharing agreement that works for all involved.
Are there grants to support a PerlQuest?
At this time, there are no specific grants to support PerlQuests. Government, corporate and family foundations that support scientific research, especially those focused on rare diseases and translational research (from rare to common), such as National Institute for Health, Rare Disease Foundation, National Organization for Rare Disorders (NORD) and Rare Genomics Institute may be interested in funding a PerlQuest. Search the Foundation Center’s Foundation Directory Online to find current grant opportunities.
An increasing number of foundations are making grants to collaborative initiatives such as PerlQuests. Reach out to these foundations and share your desire to start a PerlQuest. We are happy to partner with you on grant applications. Simply fill out the PerlQuest form and share some information about your disease and grantseeking activities.
Join our mailing list to keep in the loop about future grants for PerlQuests.
How can I crowdfund a PerlQuest?
We love crowdfunding! When Perlara was starting out, crowdfunding is how we raised funds to launch the company.
Crowdfunding is the process of raising money for a project or venture through a large number of potential backers. It has been used to fund a wide range of ventures including artistic and creative projects, scientific research, medical expenses and community-oriented social enterprises.” PerlQuests are a perfect fit for crowdfunding and will allow your community to rally together to fund much needed research for orphan diseases.
There are different types of crowdfunding that can match your PerlQuest community-ownership structure. The two primary types are Rewards-based and Equity. Donation-based and Debt-based may also be suitable for a PerlQuest.
Crowdfunding is typically done through an online platform, such as Kickstarter, Indiegogo and Crowdfunder, that allows the fundraiser to set up a public campaign for accepting funds. Other sites to explore for your peer-to-peer fundraising campaign are GiveForward, GoFundMe, YouCaring, CrowdRise MedStartr and Experiment. It is best to review and compare crowdfunding sites for the platform that is right for your PerlQuest.
To get some help creating a crowdfunding campaign, fill out the brief PerlQuest form and provide some information about your disease, community and peer-to-peer fundraising goals.
Empower your community to give money to start your PerlQuest now!
What are the stages of a PerlQuest Research Plan?
There are three stages of a PerlQuest Research Plan. Stage 1 is a natural history study (NHS) of your disease in model small animals (yeast, nematodes, fruit flies and zebrafish) using our Perlara Drug Discovery Platform. Stage 2 is a drug-repurposing screen. Stage 3 is a high-throughput drug screening campaign to discover novel compounds suitable for preclinical validation in mice.
What is my role as a PerlQuest collaborator?
To begin a PerlQuest, families and organizations provide genetic genetic and disease information. When funds need to be raised for a PerlQuest, fundraising can become an important activity. You can reach out to families with a similar disease and supporting patient organizations who could potentially become collaborators. Perlara can assist with making connections to facilitate developing relationships that are mutually beneficial. Once a PerlQuest launches, you become a champion to those in your community who may wish to follow your path of discovery. Your community becomes activated and energized as more join and the PerlQuest journey unfolds.
What happens after a PerlQuest is completed?
The participants in a PerlQuest discuss and agree upon next steps in the research path to the clinic, such as working with existing BioPharma company collaborators on larger animal studies. When a new BioPharma partner is needed, Perlara will negotiate a partnership on behalf of the PerlQuest team.
How will you provide status updates on my PerlQuest?
When will you provide status updates on my PerlQuest?
The PerlQuest project manager will send a progress report to you bi-monthly. In between reports, feel free to get in touch with questions. Keep checking in on our blog and follow @PerlaraPBC on Twitter to see real time activity of your PerlQuest. For example, see the blog posts on the Niemann-Pick C PerlQuest.
How do I communicate with Perlara during a PerlQuest?
Perlara is committed to opening and maintaining channels of communication with rare diseases families, patient advocacy organizations and our partners. We value hearing from you and other PerlQuest collaborators with whatever is on your mind.
Send an email or call the PerlQuest project manager with questions, ideas or information at your leisure. The manager will get back to you within 1 – 2 workdays. If the email address isn’t handy, use our easy contact form or [email protected]. A Twitter mention or direct message to @PerlaraPBC will also get our attention. If you would like to chat with principal investigators, let the project manager know, so that they can get in touch with you directly.
In the future, you’ll be able to set up a private account to communicate about and store information related to your PerlQuest, as well as send inquiries to the PerlQuest project manager and principal investigator. Join our mailing list for progress updates.
How can I follow a PerlQuest?
How can my community follow my PerlQuest?
Who is involved in a PerlQuest?
Who manages a PerlQuest?
How can I invite others to participate in a PerlQuest?
The more champions of PerlQuests, the merrier! Flying the banner of a PerlQuest to others not only increases awareness of your PerlQuest, but also seeds the rare disease community so that other PerlQuests can grow.
Members of your community can join as co-investors or follow the progress of your PerlQuest. Either way, advocating for a PerlQuest increases the likelihood of improving the lives of your loved ones as well as other rare disease patients.
Inviting others is simple. Call, email, text or reach out to your community through social media channels like Facebook, Twitter, YouTube and Instagram. Use the #PerlQuest hashtag, so that others following the effort can. We can help with outreach, whether it be crafting a joint email, posting to Facebook, Tweeting or pitching an article to the media. Get in touch with the PerlQuest manager or our marketing department to explore partnerships and outreach ideas.
How long will a PerlQuest take?
PerlQuests are designed to be efficient and effective, as well as fluid and flexible. A PerlQuest can take 6 to 18 months in-house at Perlara and 3-5 years collaborating with our BioPharma partners to develop a FDA approved treatment, depending on the direction a PerlQuest goes. When the science is more complex, a PerlQuest can take longer to complete. Rapid advances, breakthroughs and automation in genetics, data modeling and chemical biology could speed up the projected timeline of drug discovery and development.
Where do PerlQuests take place?
We are located at 6000 Shoreline Court, Suite 204, South San Francisco, California 94080.
Perlara just moved from its home of the last three years in the biotech incubator [email protected] to a newly constructed, 13,000+ sq. ft. lab and office space in South San Francisco, the birthplace of biotechnology. We love our new home!
How can I visit Perlara to see my PerlQuest?
Come on over! We love showing off our world-class scientific equipment and Noah’s Ark of simple animal models (yeast, nematodes, fruit flies and zebrafish). Our scientists are proud to share the latest discoveries.
To set up a lab tour to see the Perlara Drug Discovery Platform, shoot the PerlQuest manager an email. If you’re new to Perlara, fill out the contact form and state your interest and desired dates for a tour. We’ll get back with our availability and tour information.
Want to get a leg up on the tour? Read about the Echo 550 acoustic liquid handling technology used for high throughput screening (HTS), an essential in the process of finding a potential drug candidate.
What legal considerations are involved in a PerlQuest?
Legal considerations are stated in the PerlQuest Collaboration Agreement. They include the research plan (e.g. performance, objectives, principal investigator, period and costs), intellectual property (IP) rights, reports, confidentiality, termination, notice, publicity, indemnity, warranties and liability limits, compliance and general provisions (e.g. dispute resolution, assignment, severalbility, governing law, non-discrimination, etc). We will discuss with you and your legal representative other legal issues of relevance to your specific PerlQuest and document agreed upon terms in the PerlQuest Collaboration Agreement.
Are PerlQuests FDA approved?
No. PerlQuests are rapid, lean drug discovery programs on the path to an FDA approved treatment. To get FDA approval, drug manufacturers must conduct lab, animal, and human clinical testing and submit their data to the FDA.
Who owns the intellectual property (IP) of a PerlQuest?
The partners of a PerlQuest share all patents, inventions, and other intellectual property (IP) rights developed during a PerlQuest. Patent filing and expenses are jointly shared. All intellectual property developed prior to or outside of the Perlara Research Program and the PerlQuest Collaboration Agreement (link to research plan question) shall remain the property of its owner.
How is a PerlQuest’s intellectual property (IP) shared between multiple families and organizations participating in a PerlQuest?
Intellectual property (IP) rights between multiple families and organizations are negotiated on a case by case basis. Common splits are based on percent (%) of funds and time committed to a PerlQuest.
How does the 21st Century Cures Act affect PerlQuests?
The 21st Century Cures Act’s focus on the funding and fast track approval of drugs and devices, precision medicine and patient empowerment and information validates the Perlara Drug Discovery Platform and positions PerlQuests for growth.
The Act is a first-of-its-kind, bipartisan bill by Congress that aims to “help modernize and personalize health care, encourage greater innovation, support research and streamline the system,” according to the act’s mission statement. The sweeping biomedical research funding bill accelerates and expands the Precision Medicine Initiative, Patient-Focused Drug Development Initiative, BRAIN Initiative, and the Cancer Moonshot, eases the development and approval of experimental treatments, reforms federal policy on mental health care and unleashes the power of digital medicine and social media at the treatment delivery phase. The bill includes many provisions that will improve the discovery, development, and delivery of orphan therapies for rare disease patients.
Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD) champions, “The 21st Century Cures Act is a landmark medical innovation package that has the potential to help nearly every American family, including the 1 in 10 Americans with rare diseases who desperately need treatments and cures.”
What privacy controls are in place to insure that my genetic and health information is private?
Is a PerlQuest HIPAA compliant?
The Health Insurance Portability and Accountability Act (HIPAA) of 1996 mandates the adoption of Federal privacy protections for individually identifiable health information. The Privacy Rule sets national standards for the protection of health information, as applied to the three types of covered entities: health plans, health care clearinghouses, and health care providers who conduct certain health care transactions electronically. Not only are PerlQuests scientific research or non-medical procedures, but also Perlara is not a covered entity and therefore not under the legal requirements of HIPAA.
How safe is a PerlQuest?
Your family and organization will be completely safe from physical harm during a PerlQuest. PerlQuests do not involve clinical testing where there is a chance of pain from a procedure or side-effects affecting a research participant.
When a potential drug candidate is identified by a PerlQuest, our Perlara Drug Discovery Platform uses a whole model organism approach that increases the likelihood of developing safe, more precise treatments for rare and common diseases.
Will any animals be harmed in a PerlQuest?
The ethics of animal experimentation is of high importance to our team. We follow the principles of the “Middle Ground” out of a concern for animal welfare and a belief that the good arising out of experimentation does outweigh harm to the animal. The three guiding principles are 1) use of less complex animals such as yeast, nematodes, fruit flies and zebrafish, 2) conducting well-designed, data-driven studies that contribute to new rather than existing knowledge in the scientific community, and 3) reducing pain and suffering as much as possible.
Is Perlara a safe place for employees?
Perlara values its employees and their safety, comfort and wellbeing while at work. Perlara’s corporate office and laboratory adheres to Occupational Safety and Health Administration (OSHA) safety standards for laboratory environments. The company consults with a specialty trained, laboratory facility consultant for guidance with OSHA standards and regular inspections to insure against potential harm from chemical, biological, physical and safety hazards.