Fundraising Tips for Biotechnology Entrepreneurs

Due to the procedural rigor required by the medical and scientific communities, biotechnology companies face a unique set of challenges on the fundraising circuit. Here are three tips to help biotechnology entrepreneurs acquire funding for their business ventures. 

Focus on the science: In biotechnology, investors typically want to hear about the specific scientific proposition underlying the company. Entrepreneurs should always bring data to investor meetings and be honest about what they do and do not know. 

Balance youthful energy with experience: As with almost any other business, experienced leaders can add a great deal of value to a business. However, energy, passion, and youthful enthusiasm also plays a large role in a company’s ability to grow, recruit talent, and develop innovative solutions. Investors usually like to see both aspects in a biotechnology company.

Research your investors: In many cases, biotechnology investors like to focus their activities on specific niches, including early stage, reformulations, and single asset plays. Consequently, entrepreneurs should research their investors and avoid wasting resources on deals that are unlikely to happen.

Fundraising Tips for Biotechnology Entrepreneurs

As any entrepreneur will tell you, raising money is an indispensable part of all stages of a company’s life cycle. Due to the unique demands of biotechnology, entrepreneurs in this sector face a number of unique fundraising challenges. 

To overcome fundraising obstacles, biotechnology entrepreneurs have begun using crowdfunding more and more in recent years. A popular alternative to more traditional funding sources, such as universities, crowdfunding relies on social influence and networking to raise money. While crowdfunding is commonplace for most tech entrepreneurs, biotechnology entrepreneurs with an academic background may not feel as comfortable in this arena. Nevertheless, crowdfunding has the potential to help entrepreneurs grow their companies on their own terms.

Another useful fundraising source for biotechnology entrepreneurs involves corporate incubators, which support small biotech companies. Although some incubators ask for an ownership stake in the company, many do not require any type of equity investment. Entrepreneurs using this funding method benefit from state-of-the-art research facilities, as well as networking opportunities with fellow entrepreneurs.

An Overview of Nonspecific Immunotherapies

An innovative cancer treatment that utilizes the body’s own immune system to fight cancer cells, immunotherapy has emerged as one of the most promising treatments of the past several years. 

Unlike targeted therapies, which target a specific pathway thought to be involved in cancer growth, the goal of cancer immunotherapy is to activate the body's immune system. As they grow, many tumors gain the ability to suppress the immune system by turning off nearby T-cells. A new class of immunotherapies called checkpoint inhibitors help "release the brakes" of the immune system, by allowing T-cells to operate efficiently. A number of checkpoint inhibtors, including those against CTLA4, PD-1, and PD-L1, are now avaialble, and many others are being developed.

Another class of cancer immunotherapies being developed are the oncolytic viruses. These are viruses that have been genetically engineered to target and kill cancer cells. In addition to directly attacking cancer cells, oncolytic therapies activate the immune system by sensitizing and activating T-cells against the tumor. Imlygic, developed by BioVex and Amgen, is the first oncolytic virus to be available, approved by FDA and EMA in Q4-2015. Results have been very promising, particularly in combination with checkpoint inhibitors like CTLA-4 and PD-1.

Advancements Made in Treating Colorblindness

Scientific advancements are helping people with colorblindness to see the world more vividly.

Researchers are working on several fronts to combat color blindness, a genetic condition that primarily affects men, reducing color vision to as few as two colors. People with colorblindness typically have photopigments in their eyes that are less sensitive to certain colors and cannot translate those hues to the brain. 

Husband-and-wife researchers Jay and Maureen Neitz of the University of Washington have used retinal surgery and gene therapy to treat color blindness in squirrel monkeys. Their method, which is being developed for humans as the result of a partnership with the company Adverum (formerly Avalanche Biotechnologies), replaces a defective gene with one that functions correctly. 

Currently, there are no treatments available to help people with color blindness. Although some claim that spectacles can help, they have been tested in clinical trials, and no available data supports their use.

Gene Therapy’s Potential for Treating Neuroblastoma

A new, experimental gene therapy demonstrated promising results for the treatment of cerebral adrenoleukodystrophy (CALD). The treatment, called LENTI-D, is being developed by bluebird bio (BLUE). Results from the study, called STARBEAM, were presented at the American Academy of Neurology meeting.

The primary endpoint of the study is freedom from "major functional disabilities, " such as the losing ability to communicate or wheelchair dependence (MFD-free), and will be assessed two years following treatment. In this interim report, all 17 treated patients remained free of major functional disabilities, with three patients completing two years follow-up and the remaining 14 patients completing at least six months of follow-up. While encouraging, the results are preliminary, and more follow up is needed prior to FDA approval.

This is the second therapy based on blood stem cell modification to make news in recent weeks. The first, called Strimvelis, received a positive opinion from the European CHMP, which recommended approval to the European Commission for the treatment of severe combined immunodeficiency.

The Financial Future of the Gene Therapy Market

Gene therapy has the potential to treat or even cure many chronic diseases, and as such, it has become an increasing area of focus for medical research. Researchers around the globe are investigating the application of gene therapy to HIV, diabetes, cancer, and many other serious conditions. A recent report by the market research firm RNCOS looked at the clinical applications of these treatments and how they could change the health care sector in the coming four years.

According to the report, titled “Global Gene Therapy Market Forecast to 2020,” the majority of research on gene therapy is done in the context of oncology, especially in relation to cancers that are traditionally resistant to treatment. However, the scope of gene therapy research is broad, encompassing developments in treating conditions from inherited blindness to cardiovascular disease.

So far, the vast majority of gene therapy products are still in the research and clinical development phase, with few therapies commercially available. The majority of financing in the field comes from funding, grants, and private investors. Promising clinical data, regulatory approvals, and commercial revenue are the key ingredients to fueling additional investment and excitement in the field.

Gene Therapy Offers Hope for Patients with Eye Diseases

Ophthalmic research in gene therapy could lead to treatments for a number of diseases that have hitherto been untreatable, Avalanche Biotechnologies CEO Thomas Chalberg recently told Retina Today. The field is seeing a great deal of interest and innovation, said Chalberg, which could usher in a new generation of medical therapy and a new class of biologics.

Avalanche's current trials focus on treatment for wet age-related macular degeneration. So far, participants in the trials have not exhibited any serious adverse events related to the medication. Avalanche Biotechnologies, a leading developer of ophthalmic gene therapy, also is investigating the potential for gene therapy to treat vascular diseases such as diabetic macular edema and retinal vein occlusion.

“The idea of using DNA as a pharmaceutical is a sort of marvelous and wondrous proposition,” Chalberg said. “But why now? Because there are positive clinical data emerging that demonstrates that gene therapy is working.”

Gene therapy may not be available in clinics for a few years, but research increasingly shows that it could significantly change how retina specialists treat certain ocular diseases.